Methods and compositions for regulation of transgene expression

Patent metadata

Field	Value
Publication number	US-9150847-B2
Application number	US-201213624217-A
Country	US
Kind code	B2
Filing date	Sep 21, 2012
Priority date	Sep 21, 2011
Publication date	Oct 6, 2015
Grant date	Oct 6, 2015

Abstract

Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.

First claim

What is claimed is:

1. A non-naturally occurring fusion protein comprising
(i) a Transcription Activator Like Effector (TALE) DNA binding domain that binds to an endogenous albumin gene, wherein the TALE DNA binding protein comprises a plurality of TALE repeat units, each TALE repeat unit comprising an amino acid Repeat Variable Diresidue (RVD) that binds to a nucleotide in a target sequence in an endogenous albumin gene, wherein the TALE DNA binding domain comprises a +17 or +63 C-terminal truncation and
(ii) a cleavage domain,
wherein the non-naturally occurring fusion protein cleaves the endogenous albumin gene.

2. A kit comprising the non-naturally occurring fusion protein according to claim 1 .

Assignees

• Sangamo Biosciences Inc

Inventors

• Rebar Edward J

Classifications

• A61P37/06

  Immunosuppressants, e.g. drugs for graft rejection · CPC title

• A61P3/10

  for hyperglycaemia, e.g. antidiabetics · CPC title

• A61P7/04

  Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents · CPC title

• A61P7/02

  Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors · CPC title

• A61P37/02

  Immunomodulators · CPC title