Search Results - adeno-associated+virus

2 Results

Sort By:

Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies

Abstract: Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype...

Published: 4/8/2024 | Inventor(s): Anand Swaroop, Kamil Kruczek, Suja Hiriyanna, Zhijian Wu

Keywords(s): AAV, Adeno-associated Virus, Cone-rod Dystrophy, CRD, GENE THERAPY, LCA, Leber Congenital Amaurosis, Lentivirus, NEI, rare disease, Retinitis Pigmentosa, Retinopathies, RP, Swaroop

Category(s): TherapeuticArea > Opthamology, TherapeuticArea > Eye / Ear / Nose / Throat, Collaboration Sought > Licensing, Application > Therapeutics, Collaboration Sought > Collaboration

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