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Title: Methods of treating Parkinson's disease using viral vectors

Patent ·
OSTI ID:1080363
;

Methods of delivering viral vectors, particularly recombinant AAV virions, to the central nervous system (CNS) are provided for the treatment of CNS disorders, particularly those disorders which involve the neurotransmitter dopamine. The methods entail providing rAAV virions that comprise a transgene encoding aromatic amino acid decarboxylase (AADC) and administering the virions to the brain of a mammal using a non-manual pump.

Research Organization:
Univ. of California, Oakland, CA (United States)
Sponsoring Organization:
USDOE
DOE Contract Number:
AC02-05CH11231
Assignee:
Genzyme Corporation (Framingham, MA); The Regents of the University of California (Oakland, CA)
Patent Number(s):
8,309,355
Application Number:
12/384,935
OSTI ID:
1080363
Country of Publication:
United States
Language:
English

References (38)

Application of Gene Therapy for Parkinson's Disease: Nonhuman Primate Experience book January 1997
Variables affecting convection-enhanced delivery to the striatum: a systematic examination of rate of infusion, cannula size, infusate concentration, and tissue—cannula sealing time journal February 1999
Ligand-targeted receptor-mediated vectors for gene delivery journal January 1998
Increasing Volume of Distribution to the Brain with Interstiti; Infusion: Dose, Rather Than Convection, Might Be the Most Important Factor journal April 1996
Mechanism of MPTP toxicity: more answers, more questions journal January 1985
Characteristics of a Human Cell Line Transformed by DNA from Human Adenovirus Type 5 journal July 1977
A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. journal January 1987
In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector journal June 1998
Gene Transfer by Adeno-Associated Virus Vectors into the Central Nervous System journal March 1997
l-Dopa and Dopamine-Producing Gene Cassettes for Gene Therapy Approaches to Parkinson's Disease journal March 1997
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain journal October 1994
Long-term restoration of striatal l-aromatic amino acid decarboxylase activity using recombinant adeno-associated viral vector gene transfer in a rodent model of Parkinson's disease journal August 1999
Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats journal December 1997
Erratum journal January 1989
Adeno-associated virus vectors journal October 1992
Viral Gene Delivery Selectively Restores Feeding and Prevents Lethality of Dopamine-Deficient Mice journal January 1999
Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. journal May 1994
Characterization of Intrastriatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer of Human Tyrosine Hydroxylase and Human GTP-Cyclohydrolase I in a Rat Model of Parkinson’s Disease journal June 1998
Effect of ischemic cerebral volume changes on behavior journal August 1997
Prospects for the Use of Adeno-Associated Virus as a Vector for Human Gene Therapy journal July 1994
Convection-Enhanced Delivery of Adeno-Associated Virus Type 2 (AAV2) into the Striatum and Transport of AAV2 Within Monkey Brain journal March 2006
Adeno-associated Virus Vector Containing the Herpes Simplex Virus Thymidine Kinase Gene Causes Complete Regression of Intracerebrally Implanted Human Gliomas in Mice, in Conjunction with Ganciclovir Administration journal January 1998
Adeno-associated virus vectors can be efficiently produced without helper virus journal July 1998
Adeno-associated virus vector-mediated transduction in the cat brain journal September 2003
Use of Adeno-Associated Virus as a General Transduction Vector for Mammalian Cells book January 1992
Practical Aspects of the Development ofex Vivoandin VivoGene Therapy for Parkinson's Disease journal March 1997
Gene Delivery to the Mouse Brain with Adeno-Associated Virus book January 2004
Gene Therapy in the United States: A Five-Year Status Report journal September 1996
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector journal January 1999
Transfer and Expression of Potentially Therapeutic Genes into the Mammalian Central Nervous System in Vivo Using Adeno-Associated Viral Vectors book January 1995
Behavioral Recovery in 6-Hydroxydopamine-Lesioned Rats by Cotransduction of Striatum with Tyrosine Hydroxylase and Aromatic l -Amino Acid Decarboxylase Genes Using Two Separate Adeno-Associated Virus Vectors journal November 1998
Prospects and problems of gene therapy: an update journal October 2001
Immunological Instability of Persistent Adenovirus Vectors in the Brain: Peripheral Exposure to Vector Leads to Renewed Inflammation, Reduced Gene Expression, and Demyelination journal May 1996
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. journal November 1993
Surface properties, more than size, limiting convective distribution of virus-sized particles and viruses in the central nervous system journal August 2005
A novel MPTP primate model of Parkinson's disease: neurochemical and clinical changes journal September 1998
Total synthesis of a human leukocyte interferon gene journal August 1981
Gene therapy - promises, problems and prospects journal September 1997

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