The present invention relates to a
gene expression regulation optimization technology based on a target
DNA recognition ultra-small molecular module, and relates to an ultra-small eCWCas12f-VPR
system optimized through
engineering of a
CRISPR-Cas module, linking of activation domains, and various combinations of linkers and NLS sequences. It has been identified that effective and target-specific
gene expression regulation superior to that of a conventional CRISPRa
system can be achieved and the accuracy of the
gene expression regulation is remarkably excellent. In particular, unlike a conventional
CRISPR gene scissors technology, the
system of the present invention can accurately regulate the expression of a
target gene without
cutting or damaging
DNA, and thus is relatively safe and optimized for application in a gene therapy method, thereby exerting a great
impact, and, due to the characteristics of ultra-small size, enables all genes to be loaded in a single vector (all-in-one), and thus can be effectively used as an excellent
novel gene editing technology that compensates for the disadvantages of a conventional gene editing technology.