| Sample search results for: aav-mediated gene therapy |
| 1 | correspondence NATURE|VOL424|17JULY2003|www.nature.com/nature 251 | ||
|
Summary: vectors Sir -- The News story "Harmful potential of viral vectors fuels doubts over gene therapy" (Nature... cause or contribute to cancer in gene therapy subjects. As authors of the paper discussed in your story... suggests that recombinant AAV vectors may pose similar risks in ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 2 | Recombinant Adeno-associated Viral Vectors as Therapeutic Agents to Treat Neurological Disorders | ||
|
Summary: of rAAV-mediated gene transfer in a CNL1-knockout mouse model [128] (see [119] for review) and one... Department of Molecular Genetics and Microbiology, 2 Powell Gene Therapy Center, and McKnight Brain Institute... , potential caveats for rAAV-based gene therapy in ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 3 | Progress and prospects: techniques for site-directed mutagenesis in animal models | ||
|
Summary: and adenoviral vectors have not worked well for applications of gene targeting. rAAV-mediated gene targeting... to efficiently target the CFTR gene in pig and ferret primary fibroblast Much has been learned from the rAAV-mediated... (Figure 1a). When this type of vector construction is ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 4 | Progress in Direct Striatal Delivery of L-Dopa via Gene Therapy for Treatment of Parkinson's Disease Using Recombinant | ||
|
Summary: are not practical. More- 49rAAV-MEDIATED L-DOPA GENE THERAPY FOR PD #12;over, peripheral continuous administration... .e., the gene therapy procedure and whether the protein functions appropriately in humans. INTRACEREBRAL rAAV-MEDIATED... AAV-MFG-hTH transduction ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 5 | Discovery Medicine, Volume 9, number 46, Pages 204-211, March 2010 Abstract: Given improvements in viral vector design, | ||
|
Summary: ), it is reasonable to expect clinical progress in gene therapy for neurologi- cal disorders. However, despite... a growing number of successful phase I CNS gene therapy clinical trials, clear clinical efficacy has been... to successfully apply gene therapy to ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 6 | Adeno-Associated Virus Gene Repair Corrects a Mouse Model of Hereditary Tyrosinemia In Vivo | ||
|
Summary: to study the clinical significance of AAV-mediated gene repair by homologous recombination. Four d3 Fah5981... of AAV-mediated gene repair frequencies in neonates. Vectors are noted below each set and were... that AAV8-mediated gene repair is superior to that with AAV2. ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 7 | NATURE BIOTECHNOLOGY VOLUME 24 NUMBER 8 AUGUST 2006 949 AAV hits the genomic bull's-eye | ||
|
Summary: at the target locus10. (b) Two potential mechanisms of AAV-mediated gene correction are recombination and repair... homologous recombination as the most likely mechanism for AAV-mediated gene correction. b Adenovirus Adeno... Engelhardt Adeno-associated virus mediates correction of genetic mutations in mouse ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 8 | MOLECULAR AND CELLULAR BIOLOGY, May 2003, p. 35583565 Vol. 23, No. 10 0270-7306/03/$08.00 0 DOI: 10.1128/MCB.23.10.35583565.2003 | ||
|
Summary: stimulates the frequency of rAAV-mediated gene targeting by over 100-fold, suggesting that the mechanism of rAAV-mediated... by activating the homology-directed repair of the DSB. To examine whether rAAV-mediated gene targeting can... targeting by a DSB is evidence that one mechanism of ... |
|||
|
Source: Baltimore, David - Division of Biology, California Institute of Technology |
|||
|
Collection: Biotechnology ; Biology and Medicine |
|||
| 9 | HUMAN GENE THERAPY 16:299306 (March 2005) Mary Ann Liebert, Inc. | ||
|
Summary: concentration of rAAV contact in the liver is an important factor for AAV-mediated liver gene therapy... ). Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy... confirmed that HA infusion is equally effective for ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 10 | NATURE BIOTECHNOLOGY VOLUME 25 NUMBER 10 OCTOBER 2007 1111 and drugs that act directly on disease genes. | ||
|
Summary: - eral diseases in animal models and is gain- ing in popularity for clinical gene-therapy trials. However... that stable rAAV- mediated transgene expression was due to proviral integration, as detected by large... Figure 1 Model of AAV-mediated oncogenesis. AVV vector transduction normally results in ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 11 | Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic | ||
|
Summary: surface receptors other than HSPG will be critical to the advancement of AAV- mediated gene therapy... ]. In an effort to determine whether AAV-mediated HSVtk gene expression in conjunction with GCV therapy would... of receptors in ... |
|||
|
Source: Hemminki, Akseli - Department of Biosciences, University of Helsinki |
|||
|
Collection: Biology and Medicine |
|||
| 12 | original article The American Society of Gene Therapy Molecular Therapy vol. 17 no. 2, 285293 feb. 2009 285 | ||
|
Summary: in the lung fol- lowing injury and normal cellular turnover.15 Although the effi- ciency of rAAV-mediated gene... original article© The American Society of Gene Therapy Molecular Therapy vol. 17 no. 2, 285293 feb... . 2009 285 Although recombinant adeno-associated virus (rAAV) has been ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 13 | Introduction Cystic fibrosis (CF) is the most common inherited dis- | ||
|
Summary: evaluating the effect of proteasome inhibitors on AAV-mediated gene transfer in lung, liver, and muscle... . To determine the effect of the proteasome inhibitor on AAV-mediated gene transfer in mouse lung, 6-week... on AAV-mediated gene transfer in liver, 6-week-old ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 14 | Copyright Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited. Gene therapy for hemophilia | ||
|
Summary: , Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe... . Gene therapy for hemophilia Katherine P. Ponder Purpose of review This review will highlight... the progress achieved in the past 2 years on using gene therapy to ... |
|||
|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
|||
|
Collection: Biology and Medicine |
|||
| 15 | original article The American Society of Gene & Cell Therapy Molecular Therapy vol. 17 no. 11, 18571867 nov. 2009 1857 | ||
|
Summary: nov. 2009 1859 © The American Society of Gene & Cell Therapy Single rAAV-mediated Regulated Striatal... no. 11 nov. 2009 © The American Society of Gene & Cell Therapy Single rAAV-mediated Regulated... Society of Gene & Cell ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 16 | Current Gene Therapy, 2001, 1, 137-147 137 1566-5232/00 $20.00+.00 2001 Bentham Science Publishers Ltd. | ||
|
Summary: is not understood, the manipulation of rAAV integration poses a challenge in AAV mediated gene therapy. Some studies... represents another barrier for AAV mediated gene therapy applications. Transient immunosuppression using ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 17 | JOURNAL OF VIROLOGY, Apr. 2004, p. 41654175 Vol. 78, No. 8 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.8.41654175.2004 | ||
|
Summary: and the cell cycle can affect the efficiency of rAAV-mediated gene repair. MATERIALS AND METHODS Mutant e... with various agents known to induce rAAV trans- duction before rAAV-mediated gene repair was evaluated... or cytoplasmic injection of both forms of viral DNA. Evaluation of ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 18 | Principal Investigator/Program Director (Last, first, middle): Samulski, Richard Jude BIOGRAPHICAL SKETCH | ||
|
Summary: and Broad Dispersion of AAV-Mediated Transgene Expression in Mouse Brain. Molecular Therapy, 8... . Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene (ASPA... ) to the Human Brain. Human Gene Therapy ... |
|||
|
Source: Elston, Timothy C. - Department of Pharmacology, University of North Carolina at Chapel Hill |
|||
|
Collection: Mathematics ; Biology and Medicine |
|||
| 19 | HUMAN GENE THERAPY 16:781791 (July 2005) Mary Ann Liebert, Inc. | ||
|
Summary: coupled to a minimal bidirectional tet cassette for tight regulation of rAAV-mediated gene transfer... intrastriatal delivery of L-dopa using rAAV-mediated gene transfer. Proc. Natl. Acad. Sci. U.S.A. 99, 4708... HUMAN GENE THERAPY 16:781791 (July 2005) © Mary Ann ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 20 | Evaluation of Pathological Manifestations of Disease in Mucopolysaccharidosis VII Mice after Neonatal | ||
|
Summary: with the effect of AAV-mediated gene therapy in mice [20] and with echocardiographic improvements in RV... . Similar improvements were observed in MPS VII mice after systemic neonatal AAV-mediated gene therapy [20... is a reasonable target. This is consistent with ... |
|||
|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
|||
|
Collection: Biology and Medicine |
|||
| 21 | Nerve Growth Factor Expressed in the Medial Septum Following in Vivo Gene Delivery Using a Recombinant Adeno-Associated Viral Vector | ||
|
Summary: prior to a FF lesion in rats. This rAAV-mediated NGF delivery was shown to significantly attenuate... of a control rAAV vector. 1999 Academic Press Key Words: gene therapy;Alzheimer's disease; growth factors... parenchyma and the need for more diffusion than can be achieved by local protein injection. Gene ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 22 | NATURE MEDICINE VOLUME 6 NUMBER 5 MAY 2000 595 NEW TECHNOLOGY | ||
|
Summary: with this vector system. One well-known example is rAAV-mediated gene therapy for cystic fibrosis. For this... AAV vectors. Based on this, we have developed a new approach to en- hance the efficiency of rAAV-mediated gene... circularized molecule. Fig. 3 Intermolecular cis activation in- ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 23 | original article The American Society of Gene & Cell Therapy Molecular Therapy vol. 18 no. 5, 873880 may 2010 873 | ||
|
Summary: , Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB Coy D Heldermon1,2 , Kevin K... , combining CNS-directed, AAV-mediated gene therapy with BMT resulted in dramatic synergy and signifi- cant... performance, we observed little or no ... |
|||
|
Source: Orrock, John - Department of Zoology, University of Wisconsin at Madison |
|||
|
Collection: Environmental Sciences and Ecology ; Biology and Medicine |
|||
| 24 | Systemic but Not Intraocular Epo Gene Transfer Protects the Retina from Light-and | ||
|
Summary: /Rd2 mice following AAV-mediated gene replacement therapy. J. Gene Med. 5: 757 764. 25. Auricchio, A... and Characterization of Epo Expression Following AAV-Mediated Gene Transfer in Albino Rats and rds and rd10 Mice We... morphological rescue after ... |
|||
|
Source: Sandini, Giulio - Dipartimento di Informatica Sistemistica e Telematica, Università degli Studi di Genova |
|||
|
Collection: Engineering ; Computer Technologies and Information Sciences |
|||
| 25 | The idea of gene-based therapeutics has been around for some time, but it only received serious attention | ||
|
Summary: , and at that stage it was predicted that gene therapy would become a treatment for seri- ous diseases in just... the enthusiasm for gene therapy. More recently, some important technical barriers have been overcome to the point... into the state-of-the- art accomplishments made with ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 26 | JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0 | ||
|
Summary: - creased 100-fold. Thus, delivering AAV as a CaPi coprecipitate markedly enhanced AAV-mediated gene... gene transfer by binding virus to the cell surface, we hypothesized that AAV-mediated gene transfer... proteoglycan in order to enhance gene transfer. To determine ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 27 | RNAi based gene therapy for Spinocerebellar ataxia-7 HHMI Med-Into-Grad Proposal | ||
|
Summary: RNAi based gene therapy for Spinocerebellar ataxia-7 Lisa Kurtz HHMI Med-Into-Grad Proposal... : Validate use of viral vectors in patient derived cell lines In order to determine whether AAV mediated RNAi... dominant mutations that lead to expanded polyglutamine repeat regions in specific ... |
|||
|
Source: Gleeson, Joseph G. - Department of Neurosciences, University of California at San Diego |
|||
|
Collection: Biology and Medicine |
|||
| 28 | SHORT COMMUNICATION Comparative biology of rAAV transduction in ferret, | ||
|
Summary: - associated virus (rAAV)-mediated gene therapies to the lung for cystic fibrosis (CF). As new ferret and pig... appropriate animal model to test rAAV- mediated gene therapies for CF lung disease. The testing of gene... of ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 29 | Recombinant Adeno-associated Viral Vector-Mediated Glial Cell Line-Derived Neurotrophic Factor Gene Transfer Protects | ||
|
Summary: of Florida Brain Institute and Gene Therapy Center, University of Florida College of Medicine, PO Box 100244... considering gene therapy for Parkinson's disease (PD), vector delivery prior to the onset of neuropathology... . The onset of rAAV-mediated GDNF transgene expression near the ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 30 | JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.15.69696976.2001 | ||
|
Summary: by AAV-mediated gene therapy in liver. Mol. Ther. 1:154158. 42. Wang, L., K. Takabe, S. M. Bidlingmaier... uncertainties as to whether rAAV-mediated hepatic gene expression can persist lifelong after a single vector... . 4. rAAV-mediated gene ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 31 | REVIEW ARTICLE Adeno-Associated Virus Vectors and Hematology | ||
|
Summary: % of hepatocytes were positive for AAV-mediated gene expression as determined by RNA in situ hybridization... have shown the therapeutic value of AAV-mediated gene transfer in murine and/or canine hemo- philia B... of AAV-mediated gene expres- sion appears to be in ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 32 | This article was published in an Elsevier journal. The attached copy is furnished to the author for non-commercial research and | ||
|
Summary: ://www.elsevier.com/copyright #12;Author's personal copy rAAV-mediated nigral human parkin over-expression partially ameliorates... . Mandel a, a Department of Neuroscience, Powell Gene Therapy Center, McKnight Brain Institute, University... and Microbiology, Powell Gene Therapy Center, ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 33 | Virus Research 104 (2004) 5159 Novel approaches to augment adeno-associated virus | ||
|
Summary: al., 2002; Xiao et al., 1996). Recombinant AAV (rAAV) mediated gene delivery to certain tissues... ). In these tissues, rAAV mediates high level and long lasting gene expression. However, the transduction of other... that synergistic induction of ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 34 | Systemic Mannitol-Induced Hyperosmolality Amplifies rAAV2-Mediated Striatal Transduction to a | ||
|
Summary: impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene... -term rAAV mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral... . Mandel,2,3,* 1 Department of Molecular Genetics and Microbiology, 2 ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 35 | original article 2114 www.moleculartherapy.org vol.15no.12,21142123dec.2007 | ||
|
Summary: not be the best model for preclinical testing of rAAV-mediated gene therapy to the airway in humans. Received 9... reflect the effectiveness of recombinant adeno-associated virus (rAAV)-mediated gene delivery... similar, thereby suggesting that their biology is highly conserved. ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 36 | HUMAN GENE THERAPY 18:8187 (January 2007) Mary Ann Liebert, Inc. | ||
|
Summary: , 67166721. YAN, Z., RITCHIE, T.C., DUAN, D., and ENGELHARDT, J.F. (2002). Recombinant AAV-mediated gene... HUMAN GENE THERAPY 18:8187 (January 2007) © Mary Ann Liebert, Inc. DOI: 10.1089/hum.2006.128 Brief... ., 2003). These studies have paved the way for the use of dual-vector trans-splicing in human ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 37 | MOLECULAR THERAPY Vol. 4, No. 6, December 2001 Copyright The American Society of Gene Therapy | ||
|
Summary: cells. To evaluate the safety of in vivo recombinant adeno-asso- ciated virus (rAAV) mediated gene... early disease targets for AAV- mediated gene transfer, hemophilia and muscular dystro- phy, affect males... MOLECULAR THERAPY Vol. 4, No. 6, ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 38 | Neurobiology of Aging 27 (2006) 459470 Hypothalamic rAAV-mediated GDNF gene delivery | ||
|
Summary: Neurobiology of Aging 27 (2006) 459470 Hypothalamic rAAV-mediated GDNF gene delivery ameliorates... ] Kirik D, Rosenblad C, Bjorklund A, Mandel RJ. Long-term rAAV-mediated gene transfer of GDNF in the rat... Gene Therapy Center, University of Florida, College of ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 39 | original article 2096 www.moleculartherapy.org vol. 17 no. 12, 20962102 dec. 2009 | ||
|
Summary: . We anticipate this finding may greatly enhance AAV- mediated retinal gene therapy with intravitreal... Society of Gene & Cell Therapy Adeno-associated viral gene therapy has shown great promise in treating... asso- ... |
|||
|
Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
|||
|
Collection: Materials Science ; Chemistry |
|||
| 40 | Technical advance 1578 TheJournalofClinicalInvestigation http://www.jci.org Volume 118 Number 4 April 2008 | ||
|
Summary: not previously approachable in mouse mod- els of this disease. Furthermore, the fact that AAV-mediated gene... of Anatomy and Cell Biology and 2Center for Gene Therapy, University of Iowa Carver College of Medicine, Iowa... demonstrated the feasibility of AAV-mediated genetic manipula- ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 41 | DDT Volume 10, Number 19 October 2005 ReviewsDRUGDISCOVERYTODAY | ||
|
Summary: FIGURE 3 Insertion of genes into inner ear sensory epithelia using viral vectors or transgenesis. (a) AAV-mediated... player in the intricate auditory network could enable us to employ gene- or cell-based therapy to cure... death; (2) manipulation of expressed genes by ... |
|||
|
Source: Avraham, Karen - Department of Human Genetics and Molecular Medicine, Tel Aviv University |
|||
|
Collection: Biology and Medicine |
|||
| 42 | Liver-directed Neonatal Gene Therapy Prevents Cardiac, Bone, Ear, and Eye Disease in | ||
|
Summary: ] or localized AAV-mediated gene therapy [13] in MPS VII mice. Corneal clouding also contributes to decreased... Liver-directed Neonatal Gene Therapy Prevents Cardiac, Bone, Ear, and Eye Disease... -L-iduronidase (IDUA) activity results in accumulation of glycosaminoglycans in many cells. ... |
|||
|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
|||
|
Collection: Biology and Medicine |
|||
| 43 | REVIEW ARTICLE David C. Warltier, M.D., Ph.D., Editor | ||
|
Summary: American Society of Anesthesiologists, Inc. Lippincott Williams & Wilkins, Inc. Gene Therapy... 393 approved human gene therapy pro- tocols (www.od.nih.gov/oba/documents.htm). Of these, 33... of human suffering and economic cost, there are no human gene therapy ... |
|||
|
Source: Kalil, Ronald E. - Neuroscience Training Program & W.M. Keck Laboratory for Biological Imaging, University of Wisconsin at Madison |
|||
|
Collection: Biology and Medicine |
|||
| 44 | ADVANCES IN VIRUS RESEARCH, VOL. 55 ADENO-ASSOCIATED VIRUS VECTOR-MEDIATED | ||
|
Summary: . These data demon- strate that local rAAV-mediated gene delivery in solid tissues such as muscle... of a particu- lar gene in a particular anatomical region during adulthood. Because rAAV mediated transfer has... University of Florida Brain Institute University of Florida ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 45 | JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0 | ||
|
Summary: in efficiency of transduction to various tis- sues exist for rAAV. For example, rAAV-mediated gene trans- fer... (Fig. 2B). In contrast, prior infection with Ad.LacZ had no effect on rAAV-mediated gene expression... of rAAV-mediated gene transfer by the dynamin I ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 46 | Characterization of Intrastriatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer of Human Tyrosine Hydroxylase | ||
|
Summary: tested where appropriate. RESULTS Persistence of rAAV-mediated hTH and hGTPCHI gene expression... , and the animals that survived for 1 year received 2 l of vector. Neuronal specificity of rAAV-mediated gene... . Mandel, K. G. Rendahl, S. K. Spratt, R. O. Snyder, L. K. Cohen, and S. E. Leff Department of ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 47 | JOURNAL OF VIROLOGY, Aug. 2002, p. 76517660 Vol. 76, No. 15 0022-538X/02/$04.00 0 DOI: 10.1128/JVI.76.15.76517660.2002 | ||
|
Summary: , 2002 AAV-MEDIATED TRANSDUCTION OF MURINE RETINA 7653 #12;gene expression was not evident until after... , and J. F. Engelhardt. 2002. Recombinant AAV-mediated gene delivery using dual vector heterodimerization... John F. Engelhardt,3 Robert Kotin,2 and Beverly L. Davidson4,6,7 * Program in ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 48 | 2003 NaturePublishing Group 346 | MAY 2003 | VOLUME 4 www.nature.com/reviews/genetics | ||
|
Summary: S The science of gene therapy has a turbulent history. Initially perceived as a revolutionary new technology... brought the first gene-therapy success in which three children were cured of a fatal immunodeficiency... extracted from a history of anticipation and disappointment is that the future suc- cess of ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 49 | 10.1517/14712598.7.9.1 2007 Informa UK Ltd ISSN 1471-2598 1 1. Introduction | ||
|
Summary: . Gene therapy for ovarian cancer 3. Gene therapy for other gynaecological cancers 4. Gene therapy... for benign obstetric and gynaecological disorders 5. Conclusion 6. Expert opinion Gene Therapy Gene ... |
|||
|
Source: Hemminki, Akseli - Department of Biosciences, University of Helsinki |
|||
|
Collection: Biology and Medicine |
|||
| 50 | A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Muller Cells | ||
|
Summary: (AAV) mediated ocular gene therapy holds tremendous promise for treating and potentially curing... of AAV-mediated gene delivery towards other retinal degenerative diseases. Importantly, genetic... (AMD), retinitis pigmentosa, and other blinding diseases. The ... |
|||
|
Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
|||
|
Collection: Materials Science ; Chemistry |
|||
| 51 | Neurobiology of Disease Positron Emission Tomography Imaging Demonstrates | ||
|
Summary: pool can be efficiently restored after rAAV-mediated gene therapy has not been achieved yet. The [11 C... Behavioral Recovery and Correction of Dopamine Neurotransmission after Gene Therapy Ludovic Leriche,1... ,France, ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 52 | P E R S P E C T I V E www.ScienceTranslationalMedicine.org 14 October 2009 Vol 1 Issue 2 2ps2 1 | ||
|
Summary: of a pre- clinical study on a novel gene therapy ap- proach that aims to restore the dopamine- synthesizing... versions of this gene therapy ap- proach have now reached the clinic. In an ongoing phase I/II clinical... (Fig. 1). In postmortem analyses of brains from PD M E D I C I N E ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 53 | This is an Accepted Article that has been peer-reviewed and approved for publication in the Journal of Neurochemistry, but has yet to undergo copy-editing and proof correction. Please | ||
|
Summary: supply in a study that has characterized the synthesis of DOPA after AAV-mediated gene transfer... , Lund University, Lund, Sweden 3 Department of Neuroscience, Powell Gene Therapy Center, McKnight Brain... generated after this enzyme replacement strategy using gene ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 54 | original article The American Society of Gene Therapy Molecular Therapy 1 | ||
|
Summary: original article© The American Society of Gene Therapy Molecular Therapy 1 Intraventricular... that bilateral recombinant adeno-associated virus (rAAV)-mediated overexpression of GDNF in the hypothalamus... behavior and energy homeostasis. However, hypothalamic DA levels were unaffected by long-term ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 55 | Current Gene Therapy, 2007, 7, 347-360 347 1566-5232/07 $50.00+.00 2007 Bentham Science Publishers Ltd. | ||
|
Summary: Current Gene Therapy, 2007, 7, 347-360 347 1566-5232/07 $50.00+.00 © 2007 Bentham Science... Publishers Ltd. Immune Responses to Adenovirus and Adeno-Associated Vectors Used for Gene Therapy of Brain... of Neuroscience, Powell Gene Therapy Center, McKnight Brain ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 56 | GeneTherapy Using Adenovirus-Mediated Full-length Anti-HER-2 Antibody for HER-2 Overexpression Cancers | ||
|
Summary: was able to engineer the mAb expression cassette that, in the context of AAV-mediated gene transfer... GeneTherapy Using Adenovirus-Mediated Full-length Anti-HER-2 Antibody for HER-2 Overexpression... , an adenovirus-mediated full-length antibody gene therapy was developed. Experimental ... |
|||
|
Source: Tian, Weidong - Institute of Biochemistry and Cell Biology, Shanghai Institute of Biological Sciences |
|||
|
Collection: Biology and Medicine |
|||
| 57 | doi:10.1182/blood-2010-08-302729 Prepublished online November 24, 2010; | ||
|
Summary: tumor formation. This study addresses these questions with high-dose liver-directed AAV-mediated gene... liver-directed AAV-mediated gene transfer of the F9 gene (www. clinicaltrials.gov; identifiers NCT... -directed AAV- mediated ... |
|||
|
Source: Bushman, Frederic - Department of Microbiology, University of Pennsylvania |
|||
|
Collection: Biology and Medicine |
|||
| 58 | MOLECULAR THERAPY Vol. 6, No. 3, September 2002 Copyright The American Society of Gene Therapy | ||
|
Summary: the feasibility of rAAV-mediated PDH E1 gene therapy in Ad12/SV40 (Fig. 1), adenovirus particles that contain... MOLECULAR THERAPY Vol. 6, No. 3, September 2002 Copyright © The American Society of Gene Therapy... ://www.idealibrary.com on IDEAL ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 59 | HEMOSTASIS, THROMBOSIS, AND VASCULAR BIOLOGY Neonatal gene transfer with a retroviral vector results in tolerance to human | ||
|
Summary: , Armstrong E, et al. Risk and prevention of anti-factor IX formation in AAV- mediated gene transfer... . Low-dose gene therapy or fre- quent protein injections in the neonatal period might induce tolerance... caused by deficient factor IX (FIX) activity that affects 1:30 000 males.1 ... |
|||
|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
|||
|
Collection: Biology and Medicine |
|||
| 60 | Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display | ||
|
Summary: ,2,3,y 1 Department of Molecular Genetics and Microbiology, 2 Powell Gene Therapy Center, 4 Department... MOLECULAR THERAPY Vol. 10, No. 2, August 2004 302 Copyright B The American Society of Gene Therapy 1525... tools for creating animal models of diseases [22], for ... |
|||
|
Source: Kalil, Ronald E. - Neuroscience Training Program & W.M. Keck Laboratory for Biological Imaging, University of Wisconsin at Madison; Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 61 | 64 NATURE MEDICINE VOLUME 5 NUMBER 1 JANUARY 1999 Hemophilia is an X-linked recessive bleeding disorder that | ||
|
Summary: procedures. The studies here demonstrate that rAAV-mediated gene transfer of the hFIX cDNA can correct... - portant for developing new gene therapy strategies. The liver is the native site for factor IX and VIII... is important for determining the feasibility of treating hemophilia using ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 62 | MOLECULAR THERAPY Vol. 4, No. 4, October 2001 Copyright The American Society of Gene Therapy | ||
|
Summary: MOLECULAR THERAPY Vol. 4, No. 4, October 2001 Copyright © The American Society of Gene Therapy 1525... Department of Internal Medicine, and 3 Center for Gene Therapy of Cystic Fibrosis and Other Genetic Diseases... -associated virus, rAAV, homologous recombination, circular intermediates, ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 63 | Dual Therapeutic Utility of Proteasome Modulating Agents for Pharmaco-Gene Therapy of the | ||
|
Summary: to rAAV-mediated gene deliv- ery of CFTR include the limited packaging capacity of this vector (~5 kb... Dual Therapeutic Utility of Proteasome Modulating Agents for Pharmaco-Gene Therapy of the Cystic... of Anatomy and Cell Biology, 2 Department of Internal Medicine, and 5 Center for ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 64 | Proc. Natl. Acad. Sci. USA Vol. 94, pp. 1408314088, December 1997 | ||
|
Summary: . MANDEL*, S. K. SPRATT, R. O. SNYDER, AND S. E. LEFF Department of Gene Therapy Applications, Cell Genesys... were performed to evaluate the time course of expression of rAAV-mediated GDNF protein expression... therapy, or the ability to locally express or overexpress a de novo gene by ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 65 | This article appeared in a journal published by Elsevier. The attached copy is furnished to the author for internal non-commercial research | ||
|
Summary: , Powell Gene Therapy Center, McKnight Brain Institute, University of Florida College of Medicine, P.O. Box... of recombinant lentiviral vectors (rLV) is emerging as a viable candidate for clinical gene therapy... ; however, other members of the lentiviral family have been developed for ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 66 | JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.16.76627671.2001 | ||
|
Summary: a significant impact on improving rAAV-mediated gene therapy in muscle. Muscle-based gene therapy protocols have... . For these reasons, efforts aimed at increasing the efficacy of AAV-mediated gene delivery to muscle have... on the recent ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 67 | ISSN: 1524-4539 Copyright 2009 American Heart Association. All rights reserved. Print ISSN: 0009-7322. Online | ||
|
Summary: Minicircle Vector for Gene Therapy in Murine Myocardial Infarction http... .ahajournals.orgDownloaded from #12;Novel Minicircle Vector for Gene Therapy in Murine Myocardial Infarction Mei Huang, PhD; Zhi... ; Mark A. Kay, MD, PhD; Joseph C. Wu, MD, PhD Background--Conventional plasmids for ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 68 | Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host | ||
|
Summary: by AAV-mediated gene therapy in liver. Mol. Ther. 1, 154158 (2000). 6. Kay, M.A. et al. Evidence... and the lack of strong inflammatory responses to AAV- mediated gene transfer are all attractive features... . Genet. 24, 257261 (2000). 7. Manno, C.S. et al. ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 69 | Silencing of Hepatic Fatty Acid Transporter Protein 5 in Vivo Reverses Diet-induced Non-alcoholic Fatty Liver Disease and | ||
|
Summary: -consuming. Further, major advantages of AAV-mediated gene knockdown over previously reported methods aiming at gene... , these results demonstrate that a single sdsAAV-mediated delivery of shRNA expression constructs results... in phenotypes that are highly comparable with that of genetically engineered ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 70 | Current Gene Therapy, 2009, 9, 375-388 375 1566-5232/09 $55.00+.00 2009 Bentham Science Publishers Ltd. | ||
|
Summary: Current Gene Therapy, 2009, 9, 375-388 375 1566-5232/09 $55.00+.00 © 2009 Bentham Science... Publishers Ltd. Gene Therapy for Neurological Disorders: Challenges and Future Pros- pects for the Use... and Ronald J. Mandel1,* 1 Department of Neuroscience, Powell Gene ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 71 | RNA knockdown as a potential therapeutic strategy in Parkinson's disease | ||
|
Summary: College of Medicine, Gainesville, FL, USA; 2 The Powell Gene Therapy Center, University of Florida College... and caveats are discussed in this review. Gene Therapy advance online publication, 3 November 2005; doi:10... , PO Box 100244, Gainesville, FL 32610-0024, USA. E-mail: rmandel@mbi.ufl.edu ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 72 | NATURE MEDICINE VOLUME 7 NUMBER 1 JANUARY 2001 33 Converting a virus into a vector | ||
|
Summary: therapy vectors, the viral particles encapsulate a modified genome carrying a therapeutic gene cassette... effi- cacy, toxicity and immunogenicity. Properties of vectors for gene therapy For gene therapy... its suitability for specific gene ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 73 | original article The American Society of Gene & Cell Therapy Molecular Therapy 1 | ||
|
Summary: original article© The American Society of Gene & Cell Therapy Molecular Therapy 1 Two small... and Microbiology College of Medicine UF Genetics Institute, Gene Therapy Center Cancer/Genetics Research Complex... Institute, Gene Therapy Center ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 74 | JOURNAL OF VIROLOGY, June 2004, p. 63446359 Vol. 78, No. 12 0022-538X/04/$08.00 0 DOI: 10.1128/JVI.78.12.63446359.2004 | ||
|
Summary: Genetics and Microbiology,4 Powell Gene Therapy Center,2 and McKnight Brain Institute,3 University... infection. Regardless, the data show that the presence of high levels of NAb against wt AAV can reduce rAAV-mediated... as a vector for human gene therapy applications. The most com- ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 75 | GENE THERAPY AAV-mediated factor IX gene transfer to skeletal muscle in patients with | ||
|
Summary: GENE THERAPY AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe... . This requirement rests on 3 distinct features of AAV-mediated gene transfer and F.IX expression in skeletal muscle... studies, we demonstrated that either skeletal muscle ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 76 | Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using | ||
|
Summary: -dopa using rAAV-mediated gene transfer Deniz Kirik*, Biljana Georgievska*, Corinna Burger , Christian Winkler... . Second, we show that this critical L-dopa level can be achieved by rAAV-mediated TH gene transfer... Box 100244, Gainesville, FL, 32610; and Powell Gene ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 77 | HUMAN GENE THERAPY 14:12551264 (September 1, 2003) Mary Ann Liebert, Inc. | ||
|
Summary: -inducible enhancers. Gene Ther. 7, 896902. NAKAI, H., STORM, T.A., and KAY, M.A. (2000). Increasing the size of rAAV-mediated... HUMAN GENE THERAPY 14:12551264 (September 1, 2003) © Mary Ann Liebert, Inc. System... , PETER D. PENG, and MARK A. KAY ABSTRACT Gene ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 78 | HUMAN GENE THERAPY 18:871880 (October 2007) Mary Ann Liebert, Inc. | ||
|
Summary: HUMAN GENE THERAPY 18:871880 (October 2007) © Mary Ann Liebert, Inc. DOI: 10.1089/hum.2007... therapy of dominantly active mutant alleles, and to the investigation of normal gene function in animal... delivery of RNAi constructs offers a powerful and versatile approach for both gene ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 79 | Ovarian cancer gene therapy strategies Parameshwar J. Mahasreshti, Manjula Kataram, Ronald D. | ||
|
Summary: Chapter 1 Ovarian cancer gene therapy strategies Parameshwar J. Mahasreshti, Manjula Kataram... . Genes targeted/employed for ovarian cancer gene therapy 10 Table 2. Vectors for gene therapy of ovarian... cancer 30 Table 3. Promoters for ... |
|||
|
Source: Groningen, Rijksuniversiteit - Centre for Ecological and Evolutionary Studies, Department of Marine Benthic Ecology and Evolution, |
|||
|
Collection: Biology and Medicine ; Environmental Sciences and Ecology |
|||
| 80 | From disease genes to cellular pathways: a progress report | ||
|
Summary: to gene therapy. Wiley, Chichester (Novartis Foundation Symposium 255) p 147^164 Background and basic... of gene-based therapies; nonetheless, all of these diseases result in the same fate, i.e. the death... to photoreceptor degeneration is inescapable. Gene-based ... |
|||
|
Source: Hero, Alfred O. - Department of Electrical Engineering and Computer Science & Psychology, University of Michigan |
|||
|
Collection: Engineering |
|||
| 81 | Linear DNAs Concatemerize in Vivo and Result in Sustained Transgene Expression | ||
|
Summary: ;in stable rAAV-mediated gene expression in liver (8, 9) and muscle (1012), we specifically compared... expression remains a major obstacle for the implementation of nonviral DNA vectors in clinical gene therapy... . Key Words: gene therapy; liver; hemophilia; factor ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 82 | HUMAN GENE THERAPY 15:405413 (April 2004) Mary Ann Liebert, Inc. | ||
|
Summary: HUMAN GENE THERAPY 15:405413 (April 2004) © Mary Ann Liebert, Inc. Brief Report Comparison... . PENG,2 ANJA EHRHARDT,2 THERESA A. STORM,2 and MARK A. KAY2 ABSTRACT Although effective gene therapy... therapy. Curr. Gene Ther. 1, 149162. CARTER, P.J., and SAMULSKI, ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 83 | Free DNA Ends Are Essential for Concatemerization of Synthetic Double-Stranded Adeno-Associated | ||
|
Summary: concerns exist as to how far our system repre- sents rAAV- mediated gene delivery in vivo, these findings... an important role in rAAV vector genome concatemerization. Key Words: adeno-associated virus, gene therapy... AAV vectors for in vivo gene ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 84 | The American Society of Gene Therapy original article | ||
|
Summary: © The American Society of Gene Therapy original article Molecular Therapy vol. 15 no. 5, 889... of glycosaminoglycans and multisystemic disease. Gene therapy could program cells to secrete mannose 6-phosphate... )mediated gene therapy has been ... |
|||
|
Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis |
|||
|
Collection: Biology and Medicine |
|||
| 85 | HUMAN GENE THERAPY 16:11161123 (September 2005) Mary Ann Liebert, Inc. | ||
|
Summary: RT repair of F508 CFTR pre-mRNA. DISCUSSION Gene therapy for CF lung disease, using rAAV-mediated CFTR c... HUMAN GENE THERAPY 16:11161123 (September 2005) © Mary Ann Liebert, Inc. Brief Report Spliceosome... for CF gene therapy require further ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 86 | JOURNAL OF VIROLOGY, Jan. 2005, p. 214224 Vol. 79, No. 1 0022-538X/05/$08.00 0 doi:10.1128/JVI.79.1.214224.2005 | ||
|
Summary: -mediated gene therapy, several clinical trials were ini- tiated for the treatment of inherited diseases... transduction has become imperative for successful gene therapy. The other breakthrough, packaging of double... muscles, and brain. Thus, rAAV8 is a robust vector for gene transfer ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 87 | Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene | ||
|
Summary: with an AAV vector. Nat. Genet. 24, 257261 (2000). 4. Manno, C.S. et al. AAV-mediated factor IX gene transfer... , Cambridge, Massachusetts 02139, USA. 3Department of Gene Therapy and Division of Molecular Medicine... in approximately 2 weeks. INTRODUCTION Gene ... |
|||
|
Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
|||
|
Collection: Materials Science ; Chemistry |
|||
| 88 | Long-term Skeletal Muscle Protection After Gene Transfer in a Mouse Model of LGMD-2D | ||
|
Summary: . The ability of recombinant AAVmediated gene delivery to restore normal muscle mechanical properties in sgca... be prevented in a mouse model of LGMD-2D using an AAV-mediated gene delivery technique. We found... ,2 , Cathryn S Mah1,2,4 , Kevin P Campbell5 and Barry J Byrne1,2 1 Powell ... |
|||
|
Source: Campbell, Kevin P. - Department of Physiology and Biophysics, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||
| 89 | Neuropathological and Behavioral Consequences of Adeno-Associated Viral Vector-Mediated | ||
|
Summary: into the rat striatum via rAAV-mediated gene transfer, lead to mild improvements of motor impairments following... , and § The Powell Gene Therapy Center, University of Florida College of Medicine, P.O. Box 100244, Gainesville... was to evaluate the effects of rAAV-mediated BDNF and NGF ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 90 | Adenovirus Transduction is Required for the Correction of Diabetes Using Pdx-1 or | ||
|
Summary: . Several strategies have been attempted to correct diabetes using gene therapy.2,3 The delivery of insulin... via gene therapy has been attempted by several groups with varying degrees of success, but the main... and pancreas differ by only the & The American Society of Gene ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 91 | Frank Reidy Research Center for Bioelectrics "The cell biology of gene delivery: what happens to DNA | ||
|
Summary: Way (near Ted Constant Center) Dr. Dean's Research: Despite all of the hype regarding gene therapy... , at present, gene therapy is a dream due to insufficient levels of gene transfer and expression at desired... into the nucleus, and localization to the appropriate subnuclear domain, no ... |
|||
|
Source: Purdue University, Combinatorial Scientific Computing and Petascale Simulations Institute |
|||
|
Collection: Computer Technologies and Information Sciences |
|||
| 92 | original article The American Society of Gene & Cell Therapy Molecular Therapy 1 | ||
|
Summary: original article© The American Society of Gene & Cell Therapy Molecular Therapy 1 Neurotrophic... with gene therapy, possess great therapeutic poten- tial for Parkinson's disease (PD). Pleiotrophin (PTN... Rapids, Michigan, USA; 4 Department of Neuroscience, Powell ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 93 | LONG-TERM RESTORATION OF STRIATAL l-AROMATIC AMINO ACID DECARBOXYLASE ACTIVITY USING RECOMBINANT | ||
|
Summary: . The question remains, however, whether rAAV-mediated delivery of AADC can be made efficacious as a gene therapy... gene therapy strategy. 1999 IBRO. Published by Elsevier Science Ltd. Key words: l-DOPA, dopamine... , nigrostriatal tract, microdialysis, gene ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 94 | GENE THERAPY Agene-deleted adenoviral vector results in phenotypic correction of canine | ||
|
Summary: of hemophilia. Gene Ther. 1998;5:40-49. 6. Manno CS, Chew AJ, Hutchison S, et al. AAV- mediated factor IX gene... GENE THERAPY Agene-deleted adenoviral vector results in phenotypic correction of canine hemophilia... therapy. As a result, ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 95 | General Introduction Cancer Gene Therapy | ||
|
Summary: 1 General Introduction #12;Chapter 1 10 Cancer Gene Therapy Cancer is still one of the major causes... severe side effects, there is a need to develop new types of therapy for cancer. Gene therapy... is a promising new type of treatment for many diseases, including cancer. In ... |
|||
|
Source: Groningen, Rijksuniversiteit - Centre for Ecological and Evolutionary Studies, Department of Marine Benthic Ecology and Evolution, |
|||
|
Collection: Biology and Medicine ; Environmental Sciences and Ecology |
|||
| 96 | JOURNAL OF VIROLOGY, Mar. 2005, p. 36063614 Vol. 79, No. 6 0022-538X/05/$08.00 0 doi:10.1128/JVI.79.6.36063614.2005 | ||
|
Summary: 2004 Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low... process. Thus, the present study provides new insights into the risk of rAAV-mediated insertional... to carefully consider rAAV-mediated insertional mutagenesis because of the following reasons. First, ... |
|||
|
Source: Kay, Mark A. - Department of Genetics, Stanford University |
|||
|
Collection: Biology and Medicine |
|||
| 97 | doi:10.1093/brain/awh374 Brain (2005), 128, 559569 Reversal of dyskinesias in an animal model of | ||
|
Summary: , 4 Powell Gene Therapy Center, McKnight Brain Institute, University of Florida College of Medicine... . Keywords: dyskinesia; gene therapy; GTP cyclohydrolase 1; Parkinson's disease; tyrosine hydroxylase... in the brain, i.e. in the DA-depleted striatum. Local DOPA delivery by in vivo ... |
|||
|
Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida |
|||
|
Collection: Biology and Medicine |
|||
| 98 | Mol Ther . Author manuscript Functional cone rescue by RdCVF protein in a dominant model of retinitis | ||
|
Summary: Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models... for corrective gene therapy . The neuroprotective effect of FGF2 in a rat model of inherited retinal[40... cell signaling to cell therapy; pre-clinical and clinical ... |
|||
|
Source: Ecole Polytechnique, Centre de mathématiques |
|||
|
Collection: Mathematics |
|||
| 99 | Expert Review Designer Gene Delivery Vectors: Molecular Engineering and Evolution | ||
|
Summary: al. AAV-mediated gene transfer for retinal diseases. Expert Opin. Biol. Ther. 6:12791294 (2006). 26... adeno- associated virus for muscle directed gene therapy. Nat. Med. 3:306312 (1997). 5. G. P. Gao, M. R... :63816388 (2004). 7. W. D. Xiao, N. Chirmule, S. C. Berta, et al. Gene ... |
|||
|
Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley |
|||
|
Collection: Materials Science ; Chemistry |
|||
| 100 | BioMed Central Page 1 of 10 | ||
|
Summary: , Hirata RK, Inoue N: Validation of AAV-mediated gene targeting. Nat Biotechnol 2002, 20:658. 11. Sun X... . Fernandez SL, Russell DW, Hurlin PJ: Development of human gene reporter cell lines using rAAV mediated... , Carver College of Medicine, University of Iowa, Iowa City, IA, USA and ... |
|||
|
Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa |
|||
|
Collection: Biology and Medicine |
|||