Korea Research Institute of Bioscience and Biotechnology

Binna Seol

Yee Sook Cho

Han-Seop Kim

Jae Yun Kim

Mi Young Son

In Pyo Choi

Hye Jin Jeon

Jae Eun Kwak

The present invention relates to: a method for producing immunocytes, specifically induced natural killer T (iNKT) cells that are induced by direct reprogramming of isolated somatic cells, and chimeric antigen receptor (CAR)-iNKT cells into which a CAR gene encoding a CAR is introduced; iNKT cells produced by the method; and a cell therapy composition and a pharmaceutical composition for preventing or treating cancer, comprising the iNKT cells. The method according to the present invention can produce, through direct reprogramming, iNKT cells or iNKT cells into which a CAR gene is introduced, from isolated cells so as to simplify the production process and shorten production time, thereby reducing costs, to have excellent NKT cell production efficiency, and to ensure safety according to the production without passing through induced pluripotent stem cells, thereby having an excellent NKT cell production effect distinguished from that of a conventional reprogramming technique. In addition, the iNKT cells or iNKT cells into which a CAR gene is introduced, which are produced by the method, have an excellent cancer cell killing ability, and thus can be effectively used as a cell therapy composition or a pharmaceutical composition for preventing or treating cancer.

Method for producing immune cells and use thereof

The present invention relates to a method for producing natural killer cells using direct reprogramming, natural killer cells produced thereby, a biomarker specific to the natural killer cells, a cell therapeutic agent comprising the natural killer cells, a composition for treatment and prevention of cancer, a cryopreservation cell vial for storing the natural killer cells, and a medium kit for inducing the direct reprogramming. Exhibiting excellent proliferative potential and cancer cell killing potential, the natural killer cells produced by the production method can be effectively utilized for mass production and in a composition for treatment and prevention of cancer.

Method for producing natural killer cell and use thereof

The present invention relates to a method for preparing a GM1 gangliosidosis human cell model based on induced pluripotent stem cells (iPSCs) and iPSCs originated neural progenitor cells, and a use of the GM1 model above for the development of a GM1 gangliosidosis treating agent. The iPSCs originated from GM1 patient fibroblasts can be differentiated into neural progenitor cells (NPCs) and neurosphere cells that can emulate the characteristics shown in GM1 patient, so that the said cells can be efficiently used for the investigation of intracellular GM1 symptoms such as the GM1 gangliosidosis and lysosome accumulation and the gene expression pattern change. So, the GM1 cell model of the present invention can be efficiently used for the study of GM1 development mechanism and the study for the development of a therapeutic agent for the disease. The present inventors also established the inflammasome inhibitor rhIL1RA or Z-YVAD-FMK by using the above GM1 cell model and further confirmed that it can be efficiently used as a relieving/treating agent of GM1 gangliosidosis.

Method for treatment of GM1 gangliosidosis

Growing community of inventors

Daejeon, South Korea

Binna Seol