Methods and compositions for gene correction

摘要

Disclosed herein are methods and compositions for correction and/or mutation of genes associated with Parkinson's Disease as well as clones and animals derived therefrom.

主权项

1. A zinc finger nuclease (ZFN) fusion protein comprising a cleavage domain and a zinc-finger protein (ZFP) that binds to a target site in a leucine rich repeat kinase 2 (LRRK2) or α-synuclein (SCNA) gene in a genome, wherein the ZFP comprises 5 or 6 engineered zinc-finger binding domains, each zinc finger domain comprising a recognition helix region designated F1 to F5 or F1 to F6, wherein the zinc finger protein comprises the recognition helix regions as shown in a single row of Table 2 or Table 4 and further wherein the target site is selected from the group consisting of SEQ ID NO:17, SEQ ID NO:18, SEQ ID NO:46, SEQ ID NO:36 and SEQ ID NO:37.