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.
