The Impact of CRISPR/Cas9-Based Genomic Engineering on Biomedical Research and Medicine.

Abstract:

:There has been prolonged and significant interest in manipulating the genome for a wide range of applications in biomedical research and medicine. An existing challenge in realizing this potential has been the inability to precisely edit specific DNA sequences. Past efforts to generate targeted double stranded DNA cleavage have fused DNA-targeting elements such as zinc fingers and DNA-binding proteins to endonucleases. However, these approaches are limited by both design complexity and inefficient, costineffective operation. The discovery of CRISPR/Cas9, a branch of the bacterial adaptive immune system, as a potential genomic editing tool holds the promise of facile targeted cleavage. Its novelty lies in its RNA-guided endonuclease activity, which enhances its efficiency, scalability, and ease of use. The only necessary components are a Cas9 endonuclease protein and an RNA molecule tailored to the gene of interest. This lowbarrier of adoption has facilitated a plethora of advances in just the past three years since its discovery. In this review, we will discuss the impact of CRISPR/Cas9 on biomedical research and its potential implications in medicine.

journal_name

Curr Mol Med

authors

Go DE,Stottmann RW

doi

10.2174/1566524016666160316150847

subject

Has Abstract

pub_date

2016-01-01 00:00:00

pages

343-52

issue

4

eissn

1566-5240

issn

1875-5666

pii

CMM-EPUB-74425

journal_volume

16

pub_type

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