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US 12,702,721 B2Granted
Gene therapy for macular degeneration
AssigneeUniversity of Utah Research Foundation
InventorsGregory Scott Hageman, Burt Timothy Richards
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Abstract
The invention provides compositions and methods for treatment of age-related macular degeneration, including gene therapy employing vectors and transgenes expressing protective CFH polypeptide and CFHT polypeptide sequences.
Claims16
A viral vector comprising:
- (i) a recombinant polynucleotide transgene comprising a polynucleotide sequence that has at least 98% identity to SEQ ID NO:3, wherein the polynucleotide sequence encodes a truncated complement factor H (CFHT) polypeptide, wherein the CFHT polypeptide comprises isoleucine (I) at position 62 and tyrosine (Y) at position 402 a;
- (ii) a CBA promoter operably linked to the polynucleotide transgene, wherein the CBA promoter comprises a polynucleotide sequence having at least 98% identity to any of SEQ ID NO:
- 12-14;
- (iii) a Bovine Growth Factor (bGH) polyadenylation sequence;
- and
- (iv) left and right inverted terminal repeat (ITR) sequences,
- wherein the viral vector is an adeno-associated virus 2 (AAV2) vector, and
- wherein introduction of the polynucleotide transgene into a mammalian cell results in expression of the CFHT polypeptide.
5 more elements
Dependencies are inferred from the claim text.