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AssigneeUniversity of Florida Research Foundation, Inc.
InventorsShannon Elizabeth Boye, William W. Hauswirth, Sanford Leon Boye
Estimating the term…
Disclosed are viral vector compositions comprising polynucleotide sequences that express one or more biologically-active mammalian guanylate cyclase proteins. Also disclosed are methods for their use in preventing, treating, and/or ameliorating at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from a guanylate cyclase deficiency in vivo. In particular embodiments, the use of recombinant adeno-associated viral (rAAV) vectors to treat or ameliorate symptoms of Leber's congenital amaurosis, as well as other conditions caused by an absence or reduction in the expression of a functional retinal-specific guanylate cyclase 1 (retGC1).
A recombinant adeno-associated viral (rAAV) vector comprising at least a first polynucleotide that comprises a photoreceptor-specific human rhodopsin kinase promoter or a ubiquitous truncated chimeric CMV-chicken β-actin promoter, operably linked to at least a first nucleic acid segment that encodes at least a first biologically-active, retinal-specific human guanylate cyclase protein, peptide, or polypeptide that comprises a first contiguous amino acid sequence region that is at least about 95% identical to an at least 80-contiguous-amino-acid sequence from SEQ ID NO:1.
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A method for producing a biologically-active human retGC1 polypeptide in one or more retinal cells of a human in need thereof, the method comprising:
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