在生物医学研究和医学以CRISPR /Cas9为基础的基因组工程

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

Volume: 16 Issue: 4

Author(s): D.E. Go, R.W. Stottmann

Affiliation:

关键词: 生物医学研究，CRISPR/Cas9，基因治疗，基因工程、药物基因组编辑。

摘要: 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.

Cite this article as:

D.E. Go, R.W. Stottmann , The Impact of CRISPR/Cas9-Based Genomic Engineering on Biomedical Research and Medicine, Current Molecular Medicine 2016; 16 (4) . https://dx.doi.org/10.2174/1566524016666160316150847