Search Results - retinopathies

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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