Precise nucleic acid editing technologies have
facilitated the research of cellular function and the
development of novel therapeutics, especially the
current programmable nucleases-based editing
tools, such as the prokaryotic clustered regularly
interspaced short palindromic repeats (CRISPR)-
associated nucleases (Cas). As CRISPR-based
therapies are advancing toward human clinical
trials, it is important to understand how natural
genetic variation in the human population may affect
the results of these trials and even patient safety.
The development of "base-editing" technique allows
the direct, stable transformation of target DNA base
into an alternative in a programmable way, without
DNA double strand cleavage or a donor template.
Genome-editing techniques hold promises for the
treatment of genetic disease at the DNA level by
blocking the sequences associated with disease
from producing disease-causing proteins. Currently,
scientists can select the gene they want to modify,
use the Cas9 as a "molecular cutter" to cut it out,
and transform it into a more desirable version. In
this review, we focus on the recent advances of
CRISPR/Cas system by outlining the evolutionary
and biotechnological implications of current
strategies for improving the specificity and accuracy
of these genome-editing technologies. |
