Search Results - adeno-associated

5 Results

Sort By:

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: 7/25/2024 | 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

AAV Mediated CTLA-4 Gene Transfer to Treat Sjogren's Syndrome

Sjögren’s syndrome is an autoimmune disease that affects over 2 million Americans, primarily over the age of 40. One of the major outcomes of Sjögren's syndrome is xerostomia (dry mouth) that is caused by immune system attack on moisture producing salivary glands. Researchers at the National Institute of Dental and Craniofacial Research have developed...

Published: 7/25/2024 | Inventor(s): John (Jay) Chiorini, Hongen Yin

Keywords(s): Adeno-associated, Adeno-associated Virus, IB3XXX, IBXXXX, IXXXXX, Sjogren's, Sjogren's syndrome, Syndrome, T-LYMPHOCYTE, virus, Xerostomia

Category(s): Application > Diagnostics, Application > Research Materials, Application > Therapeutics, TherapeuticArea > Immunology

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: 7/25/2024 | 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: 7/25/2024 | 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: 7/25/2024 | 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