Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases
AssigneePresident and Fellows of Harvard College
InventorsYunlu Xue, Constance L. Cepko
Life of this patent
Estimating the term…
Abstract
The present invention provides compositions, e.g., pharmaceutical compositions, which include a recombinant adeno-associated viral (AAV) expression construct, AAV vectors, AAV particles, and methods of treating a subject having a degenerative ocular disorder, e.g., retinitis pigmentosa.
Claims29
A composition, comprising an adeno-associated virus (AAV) expression cassette, the expression cassette comprising a photoreceptor-specific (PR-specific) promoter and a nucleic acid molecule encoding a C247S variant thioredoxin-interacting 5 protein (TXNIP).
4-10. (canceled)
12-15. (canceled)
(canceled)
A composition, comprising an adeno-associated virus (AAV) expression cassette, the expression cassette comprising a photoreceptor-specific (PR- specific) promoter and a nucleic acid molecule encoding a dominant negative variant of hypoxia inducible factor 1 subunit alpha (HIF1α).
23-30. (canceled)
33-38. (canceled)
40-42. (canceled)
(canceled)
(canceled)
50-54. (canceled)
Dependencies are inferred from the claim text.