Abstract:
The present invention provides a method for inducing an inversion of normal blood coagulation factor VIII (F8) gene, a method for correcting an inversion of blood coagulation factor VIII gene in which the inversion has occurred, and a Hemophilia A patient-derived induced pluripotent stem cell in which the inversion is corrected, constructed using the same. The method of the present invention effectively reproduces the inversion of intron 1 and intron 22 of the F8 gene, which is responsible for the majority of severe hemophilia A, and thereby may be effectively used for studying the development mechanism of hemophilia A and as a research tool for screening therapeutic agents. The inversion-corrected induced pluripotent stem cell constructed according the method of the present invention enables an efficient and fundamental treatment for hemophilia A by restoring a genotype in which mutation has occurred to a wild type-like state, without limitation via normal gene or protein delivery.
Abstract:
The present invention relates to method for preparing a plant from a protoplast comprising knocking-out or knocking-in one or more the endogenous gene of the protoplast, and the plant regenerated from a genome-modified protoplast prepared by the above method.
Abstract:
The present disclosure relates to a method for detecting off-target sites of a programmable nuclease in a genome, and specifically, to a method for detecting off-target sites through data analysis by subjecting the genome isolated in vitro to programmable nucleases to cleave the genome and then performing whole genome sequencing or deep sequencing, and to a method for selecting on-target sites of a programmable nuclease, which minimizes the off-target effect, using this method. The Digenome-seq of the present disclosure can detect the off-target sites of a programmable nuclease on the genomic scale at a high degree of reproducibility, and thus can be used in the manufacture of programmable nucleases having high target specificity and the study thereof.
Abstract:
The present invention relates to a method of increasing the production efficiency of gene-edited plants, regenerated from plant protoplasts, by use of a Cas protein-guide RNA ribonucleoprotein (RNP). According to the present invention, the method of increasing the production efficiency of gene-edited plants makes it possible to efficiently produce target gene-mutated plants and to minimize the introduction of foreign DNA into plants. Thus, the present invention can be very advantageously used in a wide variety of fields, including agriculture, food and biotechnology.
Abstract:
The present invention relates to a method for analyzing a genotype using a target-specific nuclease and, specifically, to a method for diagnosing cancer or analyzing a genotype by removing wild type DNA or particular genotype DNA using a target-specific nuclease or a variant thereof to amplify or concentrate only a small amount of DNA which has a difference in variation, such as a mutation, or genotype, and to a method for separating target DNA sesusing a target-specific nuclease or a variant thereof. Such methods are novel paradigm methods contrary to existing simple target-specific nucleases for post-PCR recognition of normal genotype and carcinogenic genotype, and can be favorably used in the early diagnosis of cancer or analysis of similar genotypes.
Abstract:
The present invention provides a method for inducing an inversion of normal blood coagulation factor VIII (F8) gene, a method for correcting an inversion of blood coagulation factor VIII gene in which the inversion has occurred, and a Hemophilia A patient-derived induced pluripotent stem cell in which the inversion is corrected, constructed using the same. The method of the present invention effectively reproduces the inversion of intron 1 and intron 22 of the F8 gene, which is responsible for the majority of severe hemophilia A, and thereby may be effectively used for studying the development mechanism of hemophilia A and as a research tool for screening therapeutic agents. The inversion-corrected induced pluripotent stem cell constructed according the method of the present invention enables an efficient and fundamental treatment for hemophilia A by restoring a genotype in which mutation has occurred to a wild type-like state, without limitation via normal gene or protein delivery.
Abstract:
The present invention relates to a method for regulating gene expression, comprising introducing into a cell each of a recombinant vector which expresses a first domain comprising N-terminus of a Cas9 protein, and a recombinant vector which expresses a second domain comprising C-terminus of a Cas9 protein, a composition comprising the recombinant vectors, a kit for regulating gene expression, and a method for intracellular production of Cas9 protein. Moreover, the present invention relates to a transformed cell introduced with a viral vector which packages the first domain, and a viral vector which packages the second domain, and to a composition comprising a virus produced therefrom.
Abstract:
The present invention relates to a method for producing immune-compatible cells or a cell population which comprises a step of editing one or two alleles of one or more immune-compatible antigen genes by gene deletion or modification in an isolated cell comprising at least one of the immune-compatible antigen genes selected from HLA (human leukocyte antigen)-A, HLA-B and HLA-DR, to immune-compatible cells produced by the method, and to a cell population comprising the immune-compatible cells produced by the method.
Abstract:
The present invention relates to a method for regulating gene expression, comprising introducing into a cell each of a recombinant vector which expresses a first domain comprising N-terminus of a Cas9 protein, and a recombinant vector which expresses a second domain comprising C-terminus of a Cas9 protein, a composition comprising the recombinant vectors, a kit for regulating gene expression, and a method for intracellular production of Cas9 protein. Moreover, the present invention relates to a transformed cell introduced with a viral vector which packages the first domain, and a viral vector which packages the second domain, and to a composition comprising a virus produced therefrom.