Retinal protective factor 2 (rpf2) protein delivered by adeno-associated virus expression
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsJohn Ash, Clayton Pio Santiago
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Abstract
In some aspects, the disclosure relates to compositions and methods useful for maintaining or improving retinal function and/or morphology. The disclosure is based, in part, on isolated nucleic acids encoding certain neurotrophic factors (e.g., leukemia inhibitory factor (LIF), etc.) and gene therapy vectors (e.g., recombinant adeno-associated virus (rAAV) vectors) encoding the same. In some embodiments, isolated nucleic acids and gene therapy vectors described by the disclosure are useful for treatment of certain diseases or disorders of the eye, for example retinal degeneration, retinitis pigmentosa (RP), age-related macular degeneration (AMD), glaucoma, etc.
Claims18
1-36. (canceled)
A recombinant adeno-associated virus (rAAV) comprising:
- (i) an isolated nucleic acid comprising an expression cassette flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs), wherein the expression cassette encodes a fusion protein comprising a therapeutic protein, a destabilization domain (DD), and an amino acid linker comprising one or more furin cleavage sites;
- and
- (ii) one or more AAV capsid proteins.
Dependencies are inferred from the claim text.