Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
OwnerMaureen NEITZ, Jay NEITZinventors
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Abstract
Disclosed herein are nucleic acid regulatory elements, expression cassettes, expression vectors, and methods for using the nucleic acid regulatory elements, expression cassettes, expression vectors for treating cone cell disorders.
Claims29
An isolated nucleic acid comprising the sequence:
- (SEQ ID NO:
- 1)
- GGGAGGAG GAGGTCTAAG TCCCAGGCCC AATTAAGAGA
- TCAGGTAGTG TAGGGTTTGG GAGCTTTTAA GGTGAAGAGG
- CCCGGGCTGA TCCCACAGGC CAGTATAAAG CGCCGTGACC
- CTCAGGTGAC GCGCCAGGGC CGGCTGCCGT CGGGGACAGG
- GCTTTCC(X);
- wherein X is absent, or is selected from the group consisting of
- (SEQ ID NO:
- 104)
- ATAGCC
- and
- .
-4. (canceled)
-14. (canceled)
The nucleic acid expression cassette of claim 13 wherein the coding region comprises a nucleic acid encoding OPN1LW (exons 1-6) or OPN1MW (exons 1-6), and wherein:
- (a) the coding region does not include endogenous OPN1LW/MW introns;
- and
- (b) the one or more introns comprise:
- (i) a first intron comprising the sequence of SEQ ID NO:7 upstream of OPN1LW/MW exon 3, and a second intron comprising the sequence of SEQ ID NO:8 downstream of OPN1LW/MW exon 3;
- or
- (ii) a first intron comprising the sequence of SEQ ID NO:10 upstream of OPN1LW/MW exon 3, and a second intron comprising the sequence of SEQ ID NO:12 downstream of OPN1LW/MW exon 3.
-26. (canceled)
A nucleic acid expression cassette, comprising a nucleic acid that encodes an opsin polypeptide operatively linked to a promoter, wherein the nucleic acid encoding the opsin polypeptide comprises one or more introns comprising, consisting essentially of, or consisting of the nucleic acid sequence of SEQ ID NO:10 and/or SEQ ID NO:
- 12.
-38. (canceled)
-43. (canceled)
(canceled)
-48. (canceled)
-54. (canceled)
An isolated nucleic acid comprising a sequence selected from the group consisting of:
- (i) SEQ ID NO:
- 10;
- (ii) SEQ ID NO:12;
- and
- (iii) a nucleic acid sequence of the general formula A-B, wherein A encodes a polypeptide comprising, consisting essentially of, or consisting of the amino acid sequence of SEQ ID NO:21, and wherein B encodes a gene product for treating a cone cell disorder;
- or
- an isolated comprising, consisting essentially of, or consisting of the amino acid sequence of SEQ ID NO:103.
-63. (canceled)
Dependencies are inferred from the claim text.