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, Suddhasil Mookherjee, Suja Hiriyanna
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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.
Claims25
An adeno-associated virus (AAV) vector comprising a nucleic acid comprising
- (a) a nucleotide sequence encoding RP2 or a functional fragment thereof;
- (b) a human β-globin polyadenylation signal or a functional fragment thereof;
- (c) a cytomegalovirus (CMV)/human β-globin intron;
- (d) a human rhodopsin kinase promoter comprising the nucleotide sequence of SEQ ID NO:
- 10;
- and
- (e) an AAV2 Inverted Terminal Repeat (ITR) or a functional fragment thereof;
- wherein the nucleotide sequence encoding RP2 or functional fragment thereof is under the transcriptional control of the rhodopsin kinase promoter.
An adeno-associated virus (AAV) vector comprising a nucleic acid comprising
- (a) a nucleotide sequence encoding RP2 or a functional fragment thereof;
- (b) a human β-globin polyadenylation signal or a functional fragment thereof;
- (c) a cytomegalovirus (CMV)/human β-globin intron comprising the nucleotide sequence of SEQ ID NO:
- 9;
- (d) a human rhodopsin kinase promoter;
- and
- (e) an AAV2 Inverted Terminal Repeat (ITR) or a functional fragment thereof;
- wherein the nucleotide sequence encoding RP2 or functional fragment thereof is under the transcriptional control of the rhodopsin kinase promoter.
An adeno-associated virus (AAV) vector comprising a nucleic acid comprising
- (a) a nucleotide sequence encoding RP2 or a functional fragment thereof;
- (b) a human β-globin polyadenylation signal or a functional fragment thereof;
- (c) a cytomegalovirus (CMV)/human β-globin intron;
- (d) a human rhodopsin kinase promoter;
- and
- (e) an AAV2 Inverted Terminal Repeat (ITR) or a functional fragment thereof;
- wherein the nucleotide sequence encoding RP2 or functional fragment thereof is under the transcriptional control of the rhodopsin kinase promoter and the AAV vector comprises a nucleotide sequence that has at least 90% sequence identity to SEQ ID NO:
- 14.
Dependencies are inferred from the claim text.