Dual aav vectors for treating stargardt disease
AssigneesUniversity of Florida Research Foundation, Inc., Atsena Therapeutics, Inc.
InventorsSanford L. Boye, Shannon E. Boye, Kaitlyn Calabro
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Abstract
Disclosed herein are compositions and methods for treating diseases of the mammalian eye, and in particular, complications associated with Stargardt Disease. The disclosure provides AAV-based, dual vector systems that facilitate the expression of full-length proteins whose coding sequences exceed that of the polynucleotide packaging capacity of an individual AAV vector. In particular, methods and compositions relating to the expression of full-length ABCA4 using an AAV-based, dual vector system is described herein.
Claims33
A polynucleotide vector system for providing an ABCA4 gene, comprising
- a. a first AAV vector polynucleotide comprising a first ABCA4 sequence of the ABCA4 gene, and
- b. a second AAV vector polynucleotide comprising a second ABCA4 sequence of the ABCA4 gene,
- wherein the ABCA4 gene encodes an ABCA4 protein at least 95% identical to SEQ ID NO:
- 2;
- the first ABCA4 sequence comprises exon 1 to exon 20 of the ABCA4 gene, and the second ABCA4 sequence comprises exon 21 to exon 50 of the ABCA4 gene.
Dependencies are inferred from the claim text.