Improved therapeutic method for rare ocular diseases by gene replacement
AssigneesUniversite Paris-Saclay, Centre National De La Recherche Scientifique, Retina France, Variant Corporation
InventorsJérôme ROGER, Elodie Kim GRELLIER
Life of this patent
Estimating the term…
Abstract
A recombinant adeno-associated virus (AAV) vector that carries a nucleic acid sequence encoding the retinal transcription factor cone-rod homeobox (CRX) for its use in treating CRX-associated IRDs in a subject in need thereof or for use in treating inherited retinal dystrophies caused by hypomorphic mutations in the CRX target genes.
Claims16
1-13. (canceled)
A method for treating a CRX-associated inherited retinal disease (IRD) in a subject in need thereof, comprising administering to the subject a recombinant adeno-associated virus (AAV) vector comprising a polynucleotide encoding the retinal transcription factor cone-rod homeobox (CRX).
Dependencies are inferred from the claim text.