In vivo genome editing of the albumin locus as a platform for protein replacement therapy-Reference-Cited by-同舟云学术

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Published:2015-10-08 Issue:15 Volume:126 Page:1777-1784
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Sharma Rajiv¹^ORCID,Anguela Xavier M.¹²^ORCID,Doyon Yannick³,Wechsler Thomas³,DeKelver Russell C.³,Sproul Scott³,Paschon David E.³,Miller Jeffrey C.³,Davidson Robert J.¹,Shivak David³,Zhou Shangzhen¹,Rieders Julianne¹,Gregory Philip D.³,Holmes Michael C.³,Rebar Edward J.³,High Katherine A.¹²

Affiliation:

1. Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA;

2. Howard Hughes Medical Institute, Philadelphia, PA; and

3. Sangamo BioSciences, Richmond, CA

Abstract

Key Points AAV- and ZFN-mediated targeting of the albumin locus corrects disease phenotype in mouse models of hemophilia A and B. Robust expression from the albumin locus provides a versatile platform for liver-directed protein replacement therapy.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/126/15/1777/1389342/1777.pdf

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