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Hum Gene Ther. 2015 Jul;26(7):443-51. doi: 10.1089/hum.2015.074.

Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.

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1
Editas Medicine , Cambridge, Massachusetts.

Abstract

Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated 9 (Cas9) technology is revolutionizing the study of gene function and likely will give rise to an entire new class of therapeutics for a wide range of diseases. Achieving this goal requires not only characterization of the technology for efficacy and specificity but also optimization of its delivery to the target cells for each disease indication. In this review we survey the various methods by which the CRISPR-Cas9 components have been delivered to cells and highlight some of the more clinically relevant approaches. Additionally, we discuss the methods available for assessing the specificity of Cas9 editing; an important safety consideration for development of the technology.

PMID:
26068008
DOI:
10.1089/hum.2015.074
[Indexed for MEDLINE]

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