RP2 and RPGR vectors for treating X-linked retinitis pigmentosa
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsZhijian Wu, Anand Swaroop, Suja Hiriyanna, Tiansen Li
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Abstract
Disclosed are adeno-associated virus (AAV) vectors comprising a nucleotide sequence encoding RP2 or RPGR-ORF15 and related pharmaceutical compositions. Also disclosed are methods of treating or preventing X-linked retinitis pigmentosa, increasing photoreceptor number in a retina of a mammal, and increasing visual acuity of a mammal using the vectors and pharmaceutical compositions.
Claims19
An AAV vector comprising a nucleic acid comprising a nucleotide sequence encoding RPGR-ORF15 or a functional fragment or a functional variant thereof, wherein:
- (i) the vector further comprises a CMV/human β-globin intron and/or a human β-globin polyadenylation signal;
- and
- (ii) the nucleotide sequence encoding RPGR-ORF15 or a functional fragment or a functional variant thereof is under the transcriptional control of a rhodopsin kinase promoter, wherein the rhodopsin kinase promoter comprises a nucleotide sequence that is at least 99% identical to SEQ ID NO:
- 10.
An AAV vector comprising a nucleic acid comprising a nucleotide sequence encoding RPGR-ORF15 or a functional fragment or a functional variant thereof, wherein:
- (i) the vector further comprises a CMV/human β-globin intron, wherein the CMV/human β-globin intron comprises a nucleotide sequence that is at least 90% identical to SEQ ID NO:
- 8, optionally wherein the vector further comprises a human β-globin polyadenylation signal;
- and
- (ii) the nucleotide sequence encoding RPGR-ORF15 or a functional fragment or a functional variant thereof is optionally under the transcriptional control of a rhodopsin kinase promoter.
Dependencies are inferred from the claim text.