Journal article

The use of CRISPR/Cas associated technologies for cell transplant applications

Peter J Cowan

CURRENT OPINION IN ORGAN TRANSPLANTATION | LIPPINCOTT WILLIAMS & WILKINS | Published : 2016

Abstract

PURPOSE OF REVIEW: In this review, I will summarize recent developments in the use of the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated 9 (Cas9) genome editing system for cell transplant applications, ranging from transplantation of corrected autologous patient stem cells to treat inherited diseases, to the tailoring of donor pigs for cell xenotransplantation. Rational engineering of the Cas9 nuclease to improve its specificity will also be discussed. RECENT FINDINGS: Over the past year, CRISPR/Cas9 has been used in preclinical studies to correct mutations in a rapidly increasing spectrum of diseases including hematological, neuromuscular, and respirato..

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