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Sample search results for: aav-mediated gene transfer

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1 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

 
2 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

 
3 Progress in Direct Striatal Delivery of L-Dopa via Gene Therapy for Treatment of Parkinson's Disease Using Recombinant
 

Summary:  the dose of a protein by rAAV-mediated gene transfer. We have recently constructed an rAAV-tetO7-CMV... are not practical. More- 49rAAV-MEDIATED L-DOPA GENE THERAPY FOR PD #12;over, peripheral continuous administration... .e., the gene therapy procedure and whether ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
4 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... . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 466 Progress in the therapeutic efficacy of intrastriatal rAAV-mediated l-dopa delivery... . . . . . . . . . . . . . . . . . . . . ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
5 correspondence NATURE|VOL424|17JULY2003|www.nature.com/nature 251
 

Summary:  differences between retroviral and AAV-mediated integration. First, unlike retroviral vectors, AAV-mediated... 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 ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
6 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

 
7 HUMAN GENE THERAPY 16:299306 (March 2005) Mary Ann Liebert, Inc.
 

Summary:  causing significant hepatocellular injury. Quantitative evaluation of AAV-mediated liver gene transfer... confirmed that HA infusion is equally effective for rAAV-mediated gene transduc- tion, this study... concentration of rAAV contact in the liver is an important factor for ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
8 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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
9 Capsid modifications overcome low heterogeneous expression of heparan sulfate proteoglycan that limits AAV2-mediated gene transfer and therapeutic
 

Summary:  of receptors in AAV-mediated gene transfer was assessed by competition assays. Finally, we examined the ability... of receptors in AAV-mediated gene transfer for OvCa The variable expression of HSPG on OvCa cell lines... tumors. Avariety of methods have been devised ...

  

Source: Hemminki, Akseli - Department of Biosciences, University of Helsinki

 

Collection: Biology and Medicine

 
10 Discovery Medicine, Volume 9, number 46, Pages 204-211, March 2010 Abstract: Given improvements in viral vector design,
 

Summary:  in treating a CNS disor- der with gene transfer seems inevitable. Several dif- ferent vector systems have been... ] The design, production, and efficiency of gene transfer vectors, especially for transduction of the central... such as these in the future. Gene Therapy: Basic Experimental ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
11 JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0
 

Summary:  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 whether CaPi coprecipitation would improve AAV-mediated... - ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
12 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

 
13 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... AAV is capable of transducing resident lung stem/progenitor cells. Furthermore, it is unclear whether rAAV-mediated... recombination. rAAV-mediated functional expression of cre in the adult mouse ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
14 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 Therapy Single rAAV-mediated ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
15 NATURE MEDICINE VOLUME 6 NUMBER 5 MAY 2000 595 NEW TECHNOLOGY
 

Summary:  circularized molecule. Fig. 3 Intermolecular cis activation in- creases rAAV-mediated gene transfer in fi... 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- ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
16 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... delivery method for the treatment of AD. The present study demonstrates the bioactivity of NGF following rAAV-mediated... months (18). rAAV-mediated transgene expression for up to 1 year has also been reported previously (20

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
17 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... in parkinsonian rats (Carlsson et al., 2005). ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
18 JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.15.69696976.2001
 

Summary:  uncertainties as to whether rAAV-mediated hepatic gene expression can persist lifelong after a single vector... . 4. rAAV-mediated gene expression in partially hepatecto- mized mice. The figure shows serum h... contributing to persistent gene expression in muscle (8, 46). The fact that ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
19 Current Gene Therapy, 2001, 1, 137-147 137 1566-5232/00 $20.00+.00 2001 Bentham Science Publishers Ltd.
 

Summary:  . transduction) is needed before AAV-mediated gene transfer becomes therapeutically useful. Although, AAV... is essential for increasing the effectiveness of AAV mediated gene transfer for the human gene therapy... ). Despite ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
20 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... for airway innate immunity in the ferret (13), as predicted for humans (14, 15). Gene transfer to ferret... demonstrated the feasibility of AAV-mediated genetic manipula- tion ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine


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21 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... but forelimb akinesia as assessed by the stepping test was unaffected. rAAV-mediated human parkin was expressed... in TH. © 2007 Elsevier Inc. All rights reserved. Keywords: Gene ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
22 Recombinant Adeno-associated Viral Vector-Mediated Glial Cell Line-Derived Neurotrophic Factor Gene Transfer Protects
 

Summary:  30, 1999 Previous work has demonstrated that viral vector mediated gene transfer of glial cell line... . The onset of rAAV-mediated GDNF transgene expression near the substantia nigra was determined to begin... a long period of time. Therefore, in vivo gene transfer has been examined as a ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
23 RNAi based gene therapy for Spinocerebellar ataxia-7 HHMI Med-Into-Grad Proposal
 

Summary:  treatment for SCA7. The retina is an ideal target for viral vector mediated gene transfer, because... : Validate use of viral vectors in patient derived cell lines In order to determine whether AAV mediated RNAi... RNAi based gene therapy for Spinocerebellar ataxia-7 Lisa Kurtz HHMI ...

  

Source: Gleeson, Joseph G. - Department of Neurosciences, University of California at San Diego

 

Collection: Biology and Medicine

 
24 Systemic but Not Intraocular Epo Gene Transfer Protects the Retina from Light-and
 

Summary:  and Characterization of Epo Expression Following AAV-Mediated Gene Transfer in Albino Rats and rds and rd10 Mice We... morphological rescue after AAV-mediated Epo gene transfer. Histological analyses at P65 in light-damaged albino... /Rd2 mice following ...

  

Source: Sandini, Giulio - Dipartimento di Informatica Sistemistica e Telematica, Università degli Studi di Genova

 

Collection: Engineering ; Computer Technologies and Information Sciences

 
25 Neurobiology of Aging 27 (2006) 459470 Hypothalamic rAAV-mediated GDNF gene delivery
 

Summary:  ] Kirik D, Rosenblad C, Bjorklund A, Mandel RJ. Long-term rAAV-mediated gene transfer of GDNF in the rat... Neurobiology of Aging 27 (2006) 459­470 Hypothalamic rAAV-mediated GDNF gene delivery ameliorates... via its effects on dopaminergic neurons in the hypothalamus. Continuous ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
26 ADVANCES IN VIRUS RESEARCH, VOL. 55 ADENO-ASSOCIATED VIRUS VECTOR-MEDIATED
 

Summary:  of a particu- lar gene in a particular anatomical region during adulthood. Because rAAV mediated transfer has... . These data demon- strate that local rAAV-mediated gene delivery in solid tissues such as muscle... . Introduction II. Recombinant Adeno-Associated ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
27 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

 
28 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... that varying genome length and AAV capsids may allow for improved expression and/or ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
29 REVIEW ARTICLE Adeno-Associated Virus Vectors and Hematology
 

Summary:  have shown the therapeutic value of AAV-mediated gene transfer in murine and/or canine hemo- philia B... % of hepatocytes were positive for AAV-mediated gene expression as determined by RNA in situ hybridization... of AAV-mediated gene expres- ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
30 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... recombination in vivo, using a trans- splicing dual vector genetic approach. Evidence suggests that AAV-mediated... to enhance in vivo gene transfer with AAV (Yan et al., 2004), ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
31 original article 2114 www.moleculartherapy.org vol.15no.12,21142123dec.2007
 

Summary:  similar, thereby suggesting that their biology is highly conserved. rAAV-mediated gene transfer to airway... reflect the effectiveness of recombinant adeno-associated virus (rAAV)-mediated gene delivery... not be the best model for preclinical testing of ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
32 JOURNAL OF VIROLOGY, 0022-538X/99/$04.00 0
 

Summary:  of rAAV-mediated gene transfer by the dynamin I mutant was not due to inhibition on viral at- tachment... 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 ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
33 Systemic Mannitol-Induced Hyperosmolality Amplifies rAAV2-Mediated Striatal Transduction to a
 

Summary:  -term rAAV mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral... impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene... (rAAV2) have been investigated as highly effective ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
34 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... animals, a summary of progress in the use of gene transfer in large animal models will be provided... involves transfer of a wild-type or minimally modified ...

  

Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis

 

Collection: Biology and Medicine

 
35 MOLECULAR THERAPY Vol. 4, No. 6, December 2001 Copyright The American Society of Gene Therapy
 

Summary:  early disease targets for AAV- mediated gene transfer, hemophilia and muscular dystro- phy, affect males... cells. To evaluate the safety of in vivo recombinant adeno-asso- ciated virus (rAAV) mediated gene... ,2]. The current position of the ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
36 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

 
37 doi:10.1182/blood-2010-08-302729 Prepublished online November 24, 2010;
 

Summary:  liver-directed AAV-mediated gene transfer of the F9 gene (www. clinicaltrials.gov; identifiers NCT... -directed AAV- mediated gene transfer in canine and murine models. Of 77 dogs receiving AAV vector at doses up... ...

  

Source: Bushman, Frederic - Department of Microbiology, University of Pennsylvania

 

Collection: Biology and Medicine

 
38 original article The American Society of Gene & Cell Therapy Molecular Therapy vol. 18 no. 5, 873880 may 2010 873
 

Summary:  disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Ther 8: 1291­1298. #12;... , Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB Coy D Heldermon1,2 , Kevin K... , combining CNS-directed, ...

  

Source: Orrock, John - Department of Zoology, University of Wisconsin at Madison

 

Collection: Environmental Sciences and Ecology ; Biology and Medicine

 
39 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... HEMOSTASIS, THROMBOSIS, AND VASCULAR BIOLOGY Neonatal gene transfer with a retroviral vector... , and Katherine Parker Ponder The effect of neonatal ...

  

Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis

 

Collection: Biology and Medicine

 
40 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 therapeutic potential of ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry


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41 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... Characterization of Intrastriatal Recombinant Adeno-Associated Virus-Mediated ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
42 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... TherapyCentre,CollegeofMedicine,UniversityofFlorida,Gainesville,Florida32610,and6LundUniversity Bio-ImagingCenter,FacultyofMedicine,SE-22184Lund,Sweden In vivo gene transfer... affinity and D2 receptor ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
43 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... ;Abstract Viral vector mediated gene transfer is emerging as a novel therapeutic approach with clinical... studies, by our group and others, have shown a number of therapeutic effects of ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
44 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 results obtained after ...

  

Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis

 

Collection: Biology and Medicine

 
45 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... -associated virus serotype 2 aerosol- mediated ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
46 Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host
 

Summary:  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. AAV-mediated factor IX gene transfer to skeletal muscle... by ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
47 JOURNAL OF VIROLOGY, 0022-538X/01/$04.00 0 DOI: 10.1128/JVI.75.16.76627671.2001
 

Summary:  on the recent findings that rAAV-5 can significantly enhance rAAV-mediated gene transfer in certain cell types... . For these reasons, efforts aimed at increasing the efficacy of AAV-mediated gene delivery to muscle have... a significant impact on improving ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
48 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... Limiting Membrane Barriers to AAV-mediated Retinal Transduction From the Vitreous Deniz Dalkara1... intravit- real administration. In addition, we identified the ILM as a barrier to ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
49 Principal Investigator/Program Director (Last, first, middle): Samulski, Richard Jude BIOGRAPHICAL SKETCH
 

Summary:  , 2003. Stilwell J, Samulski RJ. Adeno-associated Virus Vectors for Therapeutic Gene Transfer... and Broad Dispersion of AAV-Mediated Transgene Expression in Mouse Brain. Molecular Therapy, 8... will transfer and express the genes for specific receptor subunit proteins in the inferior ...

  

Source: Elston, Timothy C. - Department of Pharmacology, University of North Carolina at Chapel Hill

 

Collection: Mathematics ; Biology and Medicine

 
50 NATURE BIOTECHNOLOGY VOLUME 25 NUMBER 10 OCTOBER 2007 1111 and drugs that act directly on disease genes.
 

Summary:  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 maintenance... susceptible to rAAV-mediated events that enhance the generation of tumors, and this will need to be ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
51 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... to the striatum at biologically active levels via recombinant adeno-associated viral (rAAV) gene transfer 4... primary fibroblasts implanted in hippocampus (Ferrer et al., ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
52 original article The American Society of Gene Therapy Molecular Therapy 1
 

Summary:  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 rAAV-mediated... -mail: rmandel@ufl.edu Nigrostriatal rAAV-mediated GDNF Overexpression Induces Robust Weight Loss in a Rat ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
53 evidence from Lagrangian surface drifters. Geophys. Res. Lett. [online] 30, 8024 (2003) (doi:10.1029/ 2003GL017511).
 

Summary:  by AAV-mediated gene transfer (V1aR-vp). b, Meadow vole infused with the AAV control vector expressing... by using viral vector V1aR gene transfer into the ventral forebrain. We show that a change... vector-mediated gene transfer to overexpress ...

  

Source: Contreras, Robert J. - Department of Psychology, Florida State University; Wang, Zuoxin - Department of Psychology, Florida State University

 

Collection: Biology and Medicine

 
54 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... gene ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
55 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... 2 to 10 months after vector ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
56 This article appeared in a journal published by Elsevier. The attached copy is furnished to the author for internal non-commercial research
 

Summary:  's personal copy Commentary The development of flexible lentiviral vectors for gene transfer in the CNS... , clinical trials in the CNS using viral gene transfer have almost exclusively utilized recombinant adeno... AAV, especiallywith regardtoresearchwhichshouldeventuallytranslatetothe clinic. For viral ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
57 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:  )-Mediated, but Not rAAV5-Mediated, Gene Transfer in the Brain Carmen S. Peden,1,2,3 Corinna Burger,2... infection. Regardless, the data show that the presence of high levels of NAb against wt AAV can reduce rAAV-mediated... for the possibility that rAAV-mediated GDNF expression levels increase over time. ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
58 The idea of gene-based therapeutics has been around for some time, but it only received serious attention
 

Summary:  successes (such as higher gene transfer efficiency and lower immunological responses) and the areas in which... -of-the-art gene-based therapies: the road ahead Mark A. Kay Abstract | Improvements in the gene transfer vectors... The initial creation of gene ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
59 From disease genes to cellular pathways: a progress report
 

Summary:  cells using viral-based vectors (Bennett et al 1998, Cheng et al 2002). However, gene transfer... 2002 Human immunode¢ciency virus type 2 (HIV-2) vector-mediated in vivo gene transfer into adult rabbit... of multiple response elements. J Biol Chem 276:34999^35007 Liang FQ, Dejneka NS, Cohen DR et al 2001 ...

  

Source: Hero, Alfred O. - Department of Electrical Engineering and Computer Science & Psychology, University of Michigan

 

Collection: Engineering

 
60 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... of long-term hepatic gene transfer of factor IX genes into the livers of hemophilia B animal models... of the vector genome over time after ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine


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61 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... of viral and nonviral gene transfer vectors have been developed for implementation of gene therapy... with advances in gene ...

  

Source: Avraham, Karen - Department of Human Genetics and Molecular Medicine, Tel Aviv University

 

Collection: Biology and Medicine

 
62 JOURNAL OF VIROLOGY, July 2003, p. 76897695 Vol. 77, No. 13 0022-538X/03/$08.00 0 DOI: 10.1128/JVI.77.13.76897695.2003
 

Summary:  -mediated gene expression and genomes slowly declined by about 95% over a period of a year, AAV-mediated hepatic... % reduction in episomal plasmid or recombinant adeno-associated virus (AAV)-mediated transgene expression... gene transfer vector. Hum. Gene Ther. ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
63 Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene
 

Summary:  of Gene Transfer Vectors Edited by: J. M. Le Doux © Humana Press, Totowa, NJ 161 #12;162 Koerber... -facilitated transfer of a unique drug-resistance gene from a donor plasmid to a random location in an acceptor plasmid... in the presence of a transposase, resulting in the transfer of ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
64 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... -mediated gene targeting and somatic cell nuclear ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
65 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:  muscles, and brain. Thus, rAAV8 is a robust vector for gene transfer to the liver and provides a promising... for vector spillover and tight control of tissue-specific gene expression. Liver-directed gene transfer... , including hemo- philia B (22). Despite such recent advances, ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
66 Current Gene Therapy, 2009, 9, 375-388 375 1566-5232/09 $55.00+.00 2009 Bentham Science Publishers Ltd.
 

Summary:  delivery method such as gene transfer. However, if earlier stage patients are to be en- rolled in GDFL gene... stated that gene transfer would likely be a potentially better alternative delivery method for GDNF [39... , thereby requiring less vector to transduce a unit area. Moreover, at ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
67 JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0
 

Summary:  that the transgene product is made in about 5% of hepatocytes, suggesting that most rAAV-mediated gene expression... -directed gene transfer results in a slow rise in transgene expres- sion, reaching steady-state levels over... and may be useful for improving liver gene ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
68 Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display
 

Summary:  been used successfully to transfer genes to a variety of tissues in adult animals [1­10]. These vectors... ,2,3,y 1 Department of Molecular Genetics and Microbiology, 2 Powell Gene Therapy Center, 4 Department... @ufl.edu. Available online 1 July 2004 Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver ...

  

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

 
69 Long-Term rAAV-Mediated Gene Transfer of GDNF in the Rat Parkinson's Model: Intrastriatal But Not Intranigral Transduction
 

Summary:  Long-Term rAAV-Mediated Gene Transfer of GDNF in the Rat Parkinson's Model: Intrastriatal... for the nigral DA neurons and their targets raises the possibility that rAAV-mediated gene transfer can be used... /or substantia nigra by rAAV- ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
70 ISSN: 1524-4539 Copyright 2009 American Heart Association. All rights reserved. Print ISSN: 0009-7322. Online
 

Summary:  and efficient systems for gene transfer in eukaryotic cells. There are 2 strategies to provide target genes... for gene transfer: viral-based and nonviral-based systems.1 Although viral-based systems have shown high... , and nitric oxide synthase, among others.9 With the use of various ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
71 ORAL PRESENTATION Open Access Immunosuppressive role of fibrinogen-like
 

Summary:  antibodies [3] and can be mimicked by FGL2 protein alone. Moreover, AAV-mediated FGL2 overexpression in rat... the graft. Splenocytes are transferred to sublethaly irradiated rats by i.v injection the day before... is improved when i.v injected. Furthermore, adoptive transfer of splenocytes from an AAVFGL2-trea- ted

  

Source: Paris-Sud XI, Université de - Institut d'Optique, Laboratoire Charles-Fabry, Groupe d'Optique Atomique

 

Collection: Physics

 
72 Technical advance TheJournalofClinicalInvestigation http://www.jci.org Volume 118 Number 4 April 2008 1571
 

Summary:  these approaches. We then investigated adeno-asso- ciated virus­mediated (AAV-mediated) gene targeting, which has... these possibilities. A better understanding of the factors that influence AAV-mediated gene targeting in pri- mary... ­mediated gene targeting and somatic cell nuclear ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
73 Current Gene Therapy, 2007, 7, 347-360 347 1566-5232/07 $50.00+.00 2007 Bentham Science Publishers Ltd.
 

Summary:  -clinical and clinical gene transfer studies using recombinant viral vectors derived from a wide range of pathogenic... neutralizing antibodies can affect intrac- erebral rAAV-mediated transduction. Moreover, when rAAV2 pre... careful analysis of rAAV-mediated immune responses in the brain, similar to the ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
74 JOURNAL OF VIROLOGY, Jan. 2006, p. 426439 Vol. 80, No. 1 0022-538X/06/$08.00 0 doi:10.1128/JVI.80.1.426439.2006
 

Summary:  2005/Accepted 8 October 2005 We and others have recently reported highly efficient liver gene transfer... and provides useful information for the future design and application of recombinant AAV. Gene transfer vectors... and persistent gene transfer, they appear as ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
75 HUMAN GENE THERAPY 15:405413 (April 2004) Mary Ann Liebert, Inc.
 

Summary:  of these. Viral-mediated gene delivery is currently the most efficient gene transfer method... in humans and could be adapted for deliv- ery of gene transfer vectors. This in combination with the im... ., PAUL, R., FENG, T., and HE, T.C. (2001). Adenoviral vector-mediated ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
76 MOLECULAR THERAPY Vol. 4, No. 4, October 2001 Copyright The American Society of Gene Therapy
 

Summary:  as the predominant pathway for augmentation of rAAV- mediated transduction by adenoviral proteins [17,18]. A second... ). Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector... : 599­602. 9. Nakai, H., Storm, T. A., and Kay, M. A. (2000). Increasing the ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
77 original article The American Society of Gene & Cell Therapy Molecular Therapy 1
 

Summary:  of -syn could have a therapeutic effect. Hayashito-Kinoh et al.15 used AAV-mediated expression of -syn... and Kirik, D (2009). Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal... original article© The American Society of Gene & Cell Therapy Molecular Therapy 1 Two small

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
78 RNA knockdown as a potential therapeutic strategy in Parkinson's disease
 

Summary:  ) expression in the SNc of mice using rAAV-mediated expression. TH is the rate limiting enzyme... K, Lewin AS, Mandel RJ. Intrastriatal rAAV-mediated delivery of anti-hun- tingtin shRNAs induces... therapy for Parkinson's disease: use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
79 HUMAN GENE THERAPY 18:871880 (October 2007) Mary Ann Liebert, Inc.
 

Summary:  in the outer retina in vivo. The advantages of AAV as a gene transfer vector include its lack of pathogenicity... ., HAUSWIRTH, W.W., and JACOBSON, S.G. (2005). Long-term restoration of rod and cone vision by single dose rAAV-mediated... gene transfer to the retina in a canine model of ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
80 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... transfer properties of these viruses are needed. AAV vectors have proven to be safe and efficient gene... , M.A. et al. Evidence for gene ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry


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81 P E R S P E C T I V E www.ScienceTranslationalMedicine.org 14 October 2009 Vol 1 Issue 2 2ps2 1
 

Summary:  that encode TH and AADC into the brain (a process referred to as gene transfer) as a tool for obtaining... has begun to fail (Fig. 2A). The attraction of this "pro-drug" gene transfer approach... gene from the vector construct. In such a two-enzyme approach, perform- ing ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
82 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... : (352) 846-2739. E-mail: flotttr@gtc.ufl.edu. To determine the feasibility of gene transfer to correct... to transduce neurons within the rat striatum. Gene ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
83 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:  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, vector... 2004 Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
84 2003 NaturePublishing Group 346 | MAY 2003 | VOLUME 4 www.nature.com/reviews/genetics
 

Summary:  been made in the development of gene-transfer technologies over recent years. The message we have... that are potentially amenable to gene transfer,it has become clear that there can be no single... ) might require cell cycling for efficient gene transfer in vivo16 . Vector TROPISM, the ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
85 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... rhodopsin expression in vivo by AAV mediated siRNA delivery . Vision Res . 47 : 1202 - 1208 37... , Maclaren RE , Duran Y , Balaggan KS , MacNeil A , Schlichtenbrede FC . 2006 ; In contrast to ...

  

Source: Ecole Polytechnique, Centre de mathématiques

 

Collection: Mathematics

 
86 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... -Associated Viral Vectors for Enhanced Gene Transfer Inchan Kwon1 and David V. Schaffer1,2 Received July 4, 2007... vector transduc- tion to liver. Based on the observed ...

  

Source: Long, Jeffrey R.- Department of Chemistry, University of California at Berkeley

 

Collection: Materials Science ; Chemistry

 
87 NATURE MEDICINE VOLUME 7 NUMBER 1 JANUARY 2001 33 Converting a virus into a vector
 

Summary:  , and in the release of excess defective vector particles that not only are incapable of gene transfer but often... of inactivated tumor suppressor genes may require gene transfer into a large fraction of the abnormal cells. Gene... be achieved even when targeting ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
88 FF Principal Investigator/Program Director (Last, First, Middle): Breese, George R PHS 398 (Rev. 4/98 facsimile)GRBGABAs (Form Page 6) Page FF
 

Summary:  .R., and Samulski, R.J. Differential and persistent expression patterns of CNS Gene Transfer by an adeno... distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther. 8

  

Source: Elston, Timothy C. - Department of Pharmacology, University of North Carolina at Chapel Hill

 

Collection: Mathematics ; Biology and Medicine

 
89 Hydroxylation of 5-Methylcytosine by TET1 Promotes Active DNA
 

Summary:  increased the 5hmC level by 43% (Figure 6A). On the other hand, AAV-mediated 428 Cell 145, 423­434, April 29... in the adult dentate granule cells (Ma et al., 2009b). AAV- mediated overexpression of TET1 or AID, but not TET... after AAV-mediated expression (Figure S7D and Table S1). Interestingly, knockdown ...

  

Source: Blackshaw, Seth - Department of Neuroscience, Johns Hopkins University

 

Collection: Biology and Medicine

 
90 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... ADENO-ASSOCIATED VIRAL VECTOR GENE TRANSFER IN A RODENT MODEL OF PARKINSON'S DISEASE S. E. LEFF,* S. K... -mediated gene transfer of l-aromatic amino ...

  

Source: Mandel, Ronald J. - Department of Neuroscience, University of Florida

 

Collection: Biology and Medicine

 
91 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... been shown to lead to hemangioma formation, heart failure, and mortality within weeks of gene transfer... ., BENOIST, C., ABDALLAH, B., RANGARA, R., HASSAN, A., SCHERMAN, D., and ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
92 JOURNAL OF VIROLOGY, 0022-538X/00/$04.00 0
 

Summary:  strategies for improving rAAV-mediated gene transfer in vivo. Recombinant adeno-associated virus (r... -stranded (ss) rAAV genomes. Following intraportal administration of vector into animals, rAAV-mediated gene... In Vivo HIROYUKI NAKAI, THERESA A. STORM, AND MARK A. KAY* Program in ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
93 Fax +41 61 306 12 34 E-Mail karger@karger.ch
 

Summary:  disorders. To date, different cerebellar cell types have been trans- duced in diverse gene transfer studies... on movement coordination, maintenance of equilibrium, muscle tone and motor memory. Efficient gene transfer... , oligodendrocytes, and that this gene ...

  

Source: Bongarzone, Ernesto R. - Department of Anatomy and Cell Biology, University of Illinois at Chicago

 

Collection: Biology and Medicine

 
94 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... Modulation Agents Enhance rAAV-Mediated CFTR Functional Correction in Polarized CF Airway Epithelia Current... to enhance rAAV-mediated CFTR delivery to the CF airway was the observation that these same ...

  

Source: Engelhardt, John F. - Department of Anatomy and Cell Biology, University of Iowa

 

Collection: Biology and Medicine

 
95 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... in developing gene transfer strategies into the liver for treatment of inherited and acquired diseases... in gene transfer into about 40% of hepatocytes, this ...

  

Source: Kay, Mark A. - Department of Genetics, Stanford University

 

Collection: Biology and Medicine

 
96 10.1517/14712598.7.9.1 2007 Informa UK Ltd ISSN 1471-2598 1 1. Introduction
 

Summary:  and metastases remains challenging. This review focuses on existing and developmental gene transfer applications... for gynaecological diseases. Keywords: cervical cancer, gene therapy, gene transfer, gynaecological disease, ovarian... of the molecular biology and genetics of diseases ...

  

Source: Hemminki, Akseli - Department of Biosciences, University of Helsinki

 

Collection: Biology and Medicine

 
97 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... values are shown for mice that received gene transfer as adults. (B) IDUA activity. Liver IDUA activity... , and eye disease. For the RV-treated mice, we evaluated only animals that received ...

  

Source: Ponder, Katherine P. - Departments of Medicine & Biochemistry and Molecular Biophysics, Washington University in St. Louis

 

Collection: Biology and Medicine

 
98 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... administration. Bioactive antibody macromolecules can be generated via gene transfer in vivo. All the data... of this approach by full-length antibody gene ...

  

Source: Tian, Weidong - Institute of Biochemistry and Cell Biology, Shanghai Institute of Biological Sciences

 

Collection: Biology and Medicine

 
99 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... Long-term Skeletal Muscle Protection After Gene ...

  

Source: Campbell, Kevin P. - Department of Physiology and Biophysics, University of Iowa

 

Collection: Biology and Medicine

 
100 REVIEW ARTICLE David C. Warltier, M.D., Ph.D., Editor
 

Summary:  - nologies (both viral and nonviral) available for gene therapy and transfer. Part II of the review... that somatic cell gene therapy targets somatic cells. The gene transfer and its therapeutic (or deleterious... technology. Alternative technol- ogies capable of ...

  

Source: Kalil, Ronald E. - Neuroscience Training Program & W.M. Keck Laboratory for Biological Imaging, University of Wisconsin at Madison

 

Collection: Biology and Medicine


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