Cell models and therapies for eye diseases

Abstract

Providing nucleic acid molecules and other components for targeting the CYP4V2 gene. [Solution] This disclosure provides nucleic acid molecules (e.g., sgRNA or gRNA molecules) for targeting the CYP4V2 gene encoding the cytochrome P450, family 4, subfamily V, polypeptide 2 protein, and donor nucleic acid molecules specific to the CYP4V2 gene. The use of these for targeting, disrupting and / or modifying mutations (e.g., c.802-8_810del17insGC mutation) of the human CYP4V2 gene, and for the treatment of crystallin retinopathy (BCD), is also provided.

Claims

[Claim 1] A vector comprising a nucleic acid molecule containing a sequence encoding a non-mutant or functional CYP4V2 protein having at least 98% sequence identity with the amino acid sequence of SEQ ID NO: 4, operably ligated to at least one control sequence.

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