Search Results - adeno-associated

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Novel Adeno-associated Viral (AAV) Vectors to Treat Hereditary Methylmalonic Acidemia (MMA) Caused by Methylmalonyl-coA Mutase (MMUT) Deficiency
Deficiency of the enzyme in methylmalonyl-CoA mutase (MMUT) results is a life-threatening disease, methylmalonic acidemia (MMA), that carries high rates of morbidity and mortality. NHGRI scientists have developed novel AAV vectors that combine the proprietary codon-optimized synMMUT alleles with either a liver-specific promoter from the human alpha-1...
Published: 10/28/2024   |   Inventor(s): Randy Chandler, Charles Venditti
Keywords(s): AAV, Acidemia, Adeno-associated, CAUSED, DEFICIENCY, Hereditary, Methylmalonic, Methylmalonyl-CoA, MMA, Mmut, Mutase, Novel, TREAT, vectors, viral, VPXXXX, WIXXXX, WJXXXX, XEXXXX
Category(s): TherapeuticArea > Cardiology, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, Application > Research Materials
Gene Therapy for Niemann-Pick Disease Type C
Investigators at the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) are seeking collaborators to further develop gene therapy to treat Niemann-Pick Disease Type C (NPC). NPC is a rare, autosomal recessive, neurodegenerative disease. Approximately 95% of patients with NPC have mutations in NPC1, a gene implicated...
Published: 10/28/2024   |   Inventor(s): Randy Chandler, William ("Bill") Pavan, Charles Venditti
Keywords(s): Adeno-associated, C, Disease, Gene, Listed LPM Contreras as of 4/15/2015, NIEMANN-PICK, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, THERAPY, treatment, TYPE, VEXXXX, VHXXXX, viral, WJXXXX, XEXXXX
Category(s): Collaboration Sought > Collaboration, TherapeuticArea > Endocrinology, Application > Therapeutics, TherapeuticArea > Neurology
Isolation, Cloning and Characterization of New Adeno-Associated Virus (AAV) Serotypes
Adeno-associated viruses (AAV) are used in gene delivery, but with limited success due to toxicity. The novel AAVs described in this technology may be more effective and useful in gene therapy applications. This invention relates to new adeno-associated viruses (AAV), vectors and particles derived therefrom and also provides methods for delivering...
Published: 10/28/2024   |   Inventor(s): John (Jay) Chiorini, Michael Schmidt
Keywords(s): Adeno-associated, CHARACTERIZATION, Cloning, GB2A2X, GB2AXX, GB2XXX, GBXXXX, GXXXXX, Isolation, Novel, Serotypes, virus
Category(s): Application > Therapeutics
Production of Adeno-Associated Viruses in Insect Cells
Adeno-associated virus (AAV) is being developed for gene therapy applications. This virus type presents several advantages over alternate vectors for therapeutic gene delivery. AAV is not considered pathogenic and transduces stably dividing and non-dividing cells. AAV also shows good serotype specificity to various cell types for targeted gene delivery. The...
Published: 10/28/2024   |   Inventor(s): Robert Kotin
Keywords(s): AAV, AAV1, AAV2, AAV5, Adeno-associated, CB2AXX, CB2XXX, CBXXXX, Cells, CXXXXX, Duke DNA Project, GB2A2X, GB2AXX, GB2XXX, GBXXXX, GENE THERAPY, GXXXXX, insect, production, virus
Category(s): Collaboration Sought > Licensing, TherapeuticArea > Oncology, Application > Therapeutics