Improved aav-mediated x-linked retinoschisis therapies
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsShannon E. Boye, Sanford L. Boye
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Abstract
The present disclosure provides for improved recombinant AAV therapies for the treatment of X-linked retinoschisis (XLRS). These therapies are designed for administration to subjects, such as human subjects, including humans diagnosed with or suffering from XLRS.
Claims55
An adeno-associated virus (AAV) vector comprising a polynucleotide that comprises (i) a heterologous nucleic acid encoding a retinoschisin protein, and (ii) an intron, splice donor region, splice acceptor region, or any combination of two or more thereof.
An AAV vector comprising a polynucleotide that comprises (i) a heterologous nucleic acid encoding a retinoschisin protein and (ii) a post-transcription regulatory element.
A recombinant AAV (rAAV) vector comprising a polynucleotide that comprises a heterologous nucleic acid comprising a sequence having at least 80%, 81%, 82%, 83%, 84%, 85%, 86%, 87%, 88%, 89%, 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, or 99% identity to the nucleotide sequence of SEQ ID NO:
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Dependencies are inferred from the claim text.