Methods and compositions for treating cone-rod retinal dystrophy
AssigneesUniversity of Florida Research Foundation, Inc., Editas Medicine, Inc.
InventorsShannon E. Boye, Sanford L. Boye, Morgan Maeder
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Abstract
Provided herein are methods and compositions for treating an eye disorder, for example CORD6. Aspects of the disclosure relate to knocking out an autosomal dominant mutant GUCY2D gene.
Claims40
A method of treating a subject having CORD6, the method comprising administering to a subject having CORD6:
- a first nucleic acid that encodes a guide RNA (gRNA) that targets a GUCY2D gene sequence, and
- a second nucleic acid that encodes an RNA-guided endonuclease.
An rAAV particle comprising an rAAV genome, wherein the rAAV genome comprises first nucleic acid that encodes a gRNA that targets a GUCY2D gene.
An rAAV particle comprising an rAAV genome, wherein the rAAV genome comprises a nucleic acid that encodes a RNA-guided endonuclease that is selective for an autosomal dominant GUCY2D gene.
An rAAV particle comprising an rAAV genome, wherein the rAAV genome comprises a nucleic acid that encodes a RNA-guided endonuclease that is selective for a GUCY2D gene, wherein the nucleic acid coding the RNA-guided endonuclease is inactivated in an inducible manner with doxycycline/tetracycline.
Dependencies are inferred from the claim text.