Cellular models of and therapies for ocular diseases
AssigneeReflection Biotechnologies Limited
InventorsRichard R. Yang, Stephen H Tsang
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Abstract
Provided herein are methods for treating or preventing diseases of the eye such as Bietti's Crystalline Dystrophy (BCD) using, for example, vectors for delivering a gene encoding a CYP4V2 protein to the retina (for example, to the retinal pigment epithelial (RPE) cells) or using cells comprising such vectors.
Claims34
A vector comprising a nucleic acid molecule comprising a sequence encoding a non-mutant or functional cytochrome P450, family 4, subfamily V, polypeptide 2 (CYP4V2) protein or a functional variant thereof having at least 99% sequence identity with the amino acid sequence of SEQ ID NO:
- 4 operably linked to at least one regulatory sequence, wherein the vector is a recombinant adeno-associated virus (rAAV) vector, wherein any part of the vector shares at least 60% sequence identity with the entirety of any one of the following sequences:
- SEQ ID NO:60;
- SEQ ID NO:61;
- SEQ ID NO:62;
- SEQ ID NO:63;
- SEQ ID NO:64;
- nucleotide (nt) 237-nt 3579 of SEQ ID NO 60;
- nt 166-nt 3515 of SEQ ID NO 61;
- nt 166-nt 3515 of SEQ ID NO 62;
- nt 166-nt 3515 of SEQ ID NO 63;
- or nt 130-nt 2097 of SEQ ID NO 64.
A vector comprising a nucleic acid molecule comprising a sequence encoding a non-mutant or functional cytochrome P450, family 4, subfamily V, polypeptide 2 (CYP4V2) protein or a functional variant thereof having at least 99% sequence identity with the amino acid sequence of SEQ ID NO:4 operably linked to at least one regulatory sequence, wherein the vector is a recombinant adeno-associated virus (rAAV) vector, wherein the sequence encoding the non-mutant or functional CYP4V2 protein or a functional variant thereof shares at least 80% sequence identity with SEQ ID NO:2.
Dependencies are inferred from the claim text.