Therapeutic applications of CRISPR/Cas9 system in gene therapy.

Abstract:

:Gene therapy is based on the principle of the genetic manipulation of DNA or RNA for treating and preventing human diseases. The clustered regularly interspaced short palindromic repeats/CRISPR associated nuclease9 (CRISPR/Cas9) system, derived from the acquired immune system in bacteria and archaea, has provided a new tool for accurate manipulation of genomic sequence to attain a therapeutic result. The advantage of CRISPR which made it an easy and flexible tool for diverse genome editing purposes is that a single protein (Cas9) complex with 2 short RNA sequences, function as a site-specific endonuclease. Recently, application of CRISPR/Cas9 system has become popular for therapeutic aims such as gene therapy. In this article, we review the fundamental mechanisms of CRISPR-Cas9 function and summarize preclinical CRISPR-mediated gene therapy reports on a wide variety of disorders.

journal_name

Biotechnol Lett

journal_title

Biotechnology letters

authors

Mollanoori H,Teimourian S

doi

10.1007/s10529-018-2555-y

subject

Has Abstract

pub_date

2018-06-01 00:00:00

pages

907-914

issue

6

eissn

0141-5492

issn

1573-6776

pii

10.1007/s10529-018-2555-y

journal_volume

40

pub_type

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