Methods and compositions for gene correction

Patent metadata

Field	Value
Publication number	US-9267123-B2
Application number	US-201113327558-A
Country	US
Kind code	B2
Filing date	Dec 15, 2011
Priority date	Jan 5, 2011
Publication date	Feb 23, 2016
Grant date	Feb 23, 2016

Abstract

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.

First claim

What is claimed is:

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.

2. A kit comprising a ZFN according to claim 1 and instructions for cleaving a LRRK2 or a SCNA gene.

Assignees

• Jaenisch Rudolf
• Laganiere Josee
• Soldner Frank
• Zhang Lei
• Sangamo Biosciences Inc
• Whitehead Biomedical Inst

Inventors

• Jaenisch Rudolf
• Laganiere Josee
• Soldner Frank
• Zhang Lei

Classifications

• C12N9/22 Primary

  Ribonucleases {[RNase]; Deoxyribonucleases [DNase]} · CPC title

• C07K2319/81

  containing a Zn-finger domain for DNA binding · CPC title

• C07K2319/00

  Fusion polypeptide · CPC title

• C12N15/8509

  for producing genetically modified animals, e.g. transgenic · CPC title

• C12N15/62

  DNA sequences coding for fusion proteins · CPC title