University of Medicine and Pharmacy, HCMC

Center for Molecular Biomedicine

Development and Applications of CRISPR-Cas9 for Genome Engineering

Recent advances in genome engineering technologies based on the CRISPR-associated
RNAguided endonuclease Cas9 are enabling the systematic interrogation of mammalian
function. Analogous to the search function in modern word processors, Cas9 can
be guided to 
specific locations within complex genomes by a short RNA search string.
Using this system, 
DNA sequences within the endogenous genome and their functional
outputs are now easily edited 
or modulated in virtually any organism of choice. Cas9-
mediated genetic perturbation is simple and 
scalable, empowering researchers to elucidate
the functional organization of the genome at the 
systems level and establish causal linkages
between genetic variations and biological phenotypes. 
In this Review, we describe the
development and applications of Cas9 for a variety of research or 
translational applications
while highlighting challenges as well as future directions. Derived from a 
remarkable microbial
defense system, Cas9 is driving innovative applications from basic biology to 
and medicine.


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