Provided are a T cell deleting the Ryr2 gene and a preparation method. Also provided are an Ryr2 antagonist, an Ryr2 overexpressing T cell, a method and applications for regulating Ryr2 expression, regulating basal Ca.sup.2+ oscillation, regulating m-Calpain activity, and increasing bonding strength between T cells and DC cells, applications of a reagent implementing the functions in preparing a medicament for treating an infectious disease, inflammation, or tumor, and a method for transforming Tconv cells into being functionally similar to Treg cells.

The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the Transmembrane protease, serine 6 (TMPRSS6) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a TMPRSS6 gene and to methods of preventing and treating a TMPRSS6-associated disorder, e.g., a disorder associated with iron overload and/or a disorder of ineffective erythropoiesis, e.g., hereditary hemochromatosis, β-thalassemia (e.g., β-thalassemia major and β-thalassemia intermiedia), polycythemia vera, myelodysplastic syndrome, congenital dyserythropoietic anemias, pyruvate kinase deficiency, erythropoietic porphyria, Parkinson's Disease, Alzheimer's Disease or Friedreich's Ataxia.

Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.

This invention relates to compounds, compositions, and methods useful for reducing transthyretin (TTR) target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

The present invention relates to peptides, proteins, nucleic acids and cells for use in immunotherapeutic methods. In particular, the present invention relates to the immunotherapy of cancer. The present invention furthermore relates to tumor-associated T-cell peptide epitopes, alone or in combination with other tumor-associated peptides that can for example serve as active pharmaceutical ingredients of vaccine compositions that stimulate anti-tumor immune responses, or to stimulate T-cells ex vivo and transfer into patients. Peptides bound to molecules of the major histocompatibility complex (MHC), or peptides as such, can also be targets of antibodies, soluble T-cell receptors, and other binding molecules.

Provided are gRNA that can direct a Cas enzyme to target a CTGF gene and the use thereof, which belongs to the technical field of gene editing. The gRNA may direct the Cas enzyme to perform targeted cleavage on an SMAD binding site region of a CTGF gene promoter, or the gRNA may direct the Cas enzyme to perform targeted cleavage on a CTGF gene exon 2 region. The gRNA can reduce the overexpression of the human CTGF gene via a CRISPR-Cas gene editing system. The above-mentioned gRNA is used for preparing a drug for use against fibrotic diseases.

The invention provides compositions and methods for reducing the immunogenicity of cells for transplant including cell-based immunotherapies. Vectors encoding beta 2 microglobulin (B2M) modifying RNAs along with targeting moieties and other signaling and/or suicide genes allow for efficient production of engineered CAR T-regulatory or other therapeutic cells from any source.

The present disclosure relates to compositions of oligonucleotide (e.g., SMAD7 antisense oligonucleotide diastereomers) and methods of manufacturing, assessing efficacy, and using the compositions.

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 are methods of treating a subject with an estrogen receptor-positive (ER+) breast cancer comprising obtaining a sample of the breast cancer from the subject; determining a level of E-cadherin in the sample is reduced compared to a control; and administering a therapeutically effective amount of an IGF1R pathway inhibitor and an endocrine therapeutic. Also disclosed herein are methods to treat a cancer in a subject comprising administering a therapeutically effective amount of an IGF1R pathway inhibitor and an E-cadherin inhibitor. Also disclosed are methods to predict the likelihood a subject with a breast cancer will respond therapeutically to a treatment comprising administering an IGF1R pathway inhibitor, the method comprising obtaining a sample of the cancer from the subject; and determining a level of E-cadherin in the sample.