The present disclosure relates to a medicament for treating cholangiocarcinoma with KRAS mutations. Specifically, the present invention relates to a method of ER inhibitors for the specific treatment of cholangiocarcinoma with KRAS mutations, and to a use of ER inhibitors in the preparation of a medicament for the treatment of cholangiocarcinoma with KRAS mutations. New therapeutic regimens for patients suffering from cholangiocarcinoma with KRAS mutations are provided to improve the possibility of therapeutic benefit for patients.

Disclosed herein is a method of treating a cancer in a subject having or suspected of having the cancer that has a mutated PREX2 expressed thereon. According to the embodiment of the present disclosure, the mutation is G258V, S1113R, E1346D or K400fs. The method includes the step of administering an effective amount of a composition to the subject, wherein the composition includes a therapeutic molecule for producing a polypeptide that exhibits a binding affinity to the mutated PREX2 protein expressed in the cancer in the subject.

The present disclosure provides a 3′UTR CRISPR-dCas13 Engineering System, and methods of using said system, that allows for the manipulation of the length of 3′untranslated regions by using gRNAs to guide catalytically dead Cas13 to sites upstream and/or downstream of the desired poly adenylation sites. One aspect of the disclosure provides a system for modifying the length a 3′ untranslated region (UTR) of an mRNA transcript.

This invention relates to a novel mRNA composition and its production method useful for developing and manufacturing RNA-based anti-viral and/or anti-cancer vaccines and medicines. This invention includes two types of mRNA constructs, namely “5′-hairpin messenger RNA (5hmRNA)” and “messenger-hairpin-messenger RNA (mhmRNA)”, respectively. Both of 5hmRNA and mhmRNA contain at least a hairpin-like stem-loop RNA structure. The 5hmRNA contains at least a stem-loop RNA structure in the 5′-UTR of a protein/peptide-coding mRNA, while the mhmRNA contains a middle stem-loop structure flanked with two protein/peptide-coding mRNA sequences on both sides. In mhmRNA, the first 5′-mRNA preferably encodes an RNA replicase, for amplifying the second 3′-mRNA in transfected cells. After transfection into target cells, 5hmRNA and mhmRNA can be further translated into at least a desired protein/peptide. To produce highly structured 5hmRNA and mhmRNA, a novel PCR-IVT methodology has been developed and used with a specially designed RNA polymerase-helicase mixture reaction

The present invention relates to pharmaceutical compositions for preventing or treating pulmonary metastasis of cancer. More specifically, the present invention relates to compositions that enhance anti-cancer immunity of the lung rather than induce death of advanced cancer, thus being effective in inhibiting, preventing or treating pulmonary metastasis of cancer.

According to an aspect, provided are: a guide RNA; a vector comprising the same; a composition for removing a nucleic acid sequence encoding a KRAS polypeptide in the genome of a cell, containing the same; a composition for preventing or treating cancer, containing the same; and a method using the same. The present invention enables the mutation of a nucleic acid sequence encoding a KRAS polypeptide in the genome of a cell or a subject and, particularly, can be usable, as personalized or precision medical care, in the prevention or treatment of cancer.

Disclosed herein are compositions and methods comprising extracellular vesicles comprising nucleic acid that target genes, leading to macrophage polarization of tumor associated macrophages. In certain embodiments, disclosed herein are methods and compositions for increasing macrophage polarization for the treatment of cancer.

The present invention relates to methods and systems for a delay in the onset of early osteogenic markers, a reduction in the hematopoietic potential to form granulocyte units, and a decrease in vascular potential and in cancer-related gene expression. The present invention is a method of down-regulation of GLI1 via CRISPR or siRNA. The present invention indicates that the GLI1 intronic region is critical for the feedback loop and that GLI1 has lineage-specific effects on hESC differentiation. The present invention documents the extent of GLI1 abrogation on early stages of human development and to show that GLI1 transcription can be altered in a therapeutically useful way.

The application relates to methods for compositions for identifying lncRNA loci associated with target genotypes or phenotypes, including desirable plant genotypes or phenotype. The application also relates to regulatory regions and genes associated with drug resistance, such as resistance to BRAF-inhibitors. Such regulatory regions and genes form the basis for methods for identifying resistance to BRAF-inhibitors, which is useful for improving disease prognosis, treatment, and likely outcomes. The regulatory regions and genes are also suitable targets for therapy in melanoma that is resistant to BRAF-inhibitors.

The present invention provides a method of treating a disease associated with elevated KRAS activity or expression in a subject, comprising suppressing KRAS expression in the subject by targeting an expression regulatory region of KRAS gene using a CRISPR-Guide Nucleotide Directed Modulation (GNDM). Also, provided is a CRISPR-GNDM system for suppressing KRAS expression comprising (a) a protein selected from the group consisting of dCas9 or dCpf1, a fusion protein of dCas9 or dCpf1 and Kruppel associated box (KRAB), and (b) a guide nucleotide targeting an expression regulatory region of KRAS gene.