Abstract: A gene editing system for treating Duchenne muscular dystrophy, and a method for treating the disease using the gene editing system are disclosed. The system and method have the effects of making it possible to package the gene editing system in a single vector by editing the dystrophin gene using a CRISPR/Cas12f1 or TaRGET system, as well as making it possible to produce the dystrophin protein having a normal function by preventing the production of a stop codon of exon 51 through the skipping of exon 51, and thus can be useful for treating Duchenne muscular dystrophy.

Abstract: The present invention relates to LCA10 treatment using a CRISPR/Cas12f1(Cas14a1) system. In particular, the present invention relates to a composition comprising a CRISPR/Cas12f1(Cas14a1) system for LCA10 treatment, and to a treatment method using same. In addition, the present invention relates to a composition comprising a CRISPR/Cas12f1(Cas14a1) system for artificially manipulating the CEP290 gene, and to a method of editing the CEP290 gene.

Abstract: The present invention relates to a novel target nucleic acid editing system including a miniaturized nucleic acid editing protein and an engineered guide RNA, uses of homology-directed repair (HDR) in a target gene thereof, and the like. According to one embodiment, homology-directed repair (HDR) using the target nucleic acid editing system has high HDR efficiency compared to other CRISPR/Cas systems due to a tendency to cut the back (outside) of a target nucleic acid, and also has the effect of enabling packaging of a gene editing system including a donor nucleic acid in a single vector, even when the packaging size is very limited like adeno-associated viruses (AAV), and maximizing the HDR efficiency by adding shRNA that inhibits a non-homologous end joining (NHEJ) process.

Abstract: In the present disclosure, there is provided an engineered Cas12f1 guide RNA for increasing intracellular gene editing activity of a CRISPR/Cas12f1 system by overcoming limitations of the prior art. The engineered Cas12f1 guide RNA is an engineered form of a naturally occurring guide RNA whose structure is partially modified. In addition, the engineered Cas12f1 guide RNA is characterized in that at least a portion of the scaffold region, which serves to interact with the Cas12f1 protein, is modified. The engineered scaffold region is characterized in that it is different from a scaffold region of a naturally occurring guide RNA.

Abstract: Disclosed in the present specification are: dead Cas12f1 having the nucleic acid cleavage activity removed; and a fusion protein in which a functional domain is fused to the dead Cas12f1. The dead Cas12f1 and the dCas12f1-based fusion protein may: form a CRISPR gene-editing system together with a guide RNA; and exhibit various functions relating to a target gene such as base editing and expression control.

Abstract: A CRISPR regulatory system is disclosed. More particularly, the CRISPR regulatory system is useful for effectively regulating expression of a target gene and contains a Cas12f1 fusion protein and an engineered Cas12f1 guide RNA Uses of the CRISPR regulatory system are also disclosed. A method of regulating expression of a target gene by using a CRISPR regulatory system containing a Cas12f1 fusion protein and an engineered Cas12f1 guide RNA is also disclosed.

Abstract: Provided is an engineered CRISPR/Cas12f1 (Cas14a1) system. Provided are an engineered guide RNA for more effectively performing cleavage, editing, or modifying of a target nucleic acid, and an engineered CRISPR/Cas12f1 (Cas14a1) system containing the engineered guide RNA. In addition, provided is a method of performing cleavage, editing, or modifying of a target nucleic acid by using the engineered guide RNA and the engineered CRISPR/Cas12f1 (Cas14a1) system including the engineered guide RNA.

Abstract: In the present disclosure, there is provided an engineered Cas12f1 guide RNA for increasing intracellular gene editing activity of a CRISPR/Cas12f1 system by overcoming limitations of the prior art. The engineered Cas12f1 guide RNA is an engineered form of a naturally occurring Cas12f1 guide RNA to which a new component is added and also in which a part of its structure is modified. The engineered Cas12f1 guide RNA is characterized by comprising a U-rich tail, which is a new component, at the 3? end. In addition, the engineered Cas12f1 guide RNA is characterized in that at least a portion of the scaffold region, which serves to interact with the Cas12f1 protein, is modified.