The present invention relates to a method of patterning and chopping 3D organoids prepared from human pluripotent stem cells, culturing the stem cells or progenitor cells, and inducing the differentiation thereof to obtain a large amount of finally differentiated cells. Compared to cells differentiated by a conventional differentiation method, the cells obtained in a large amount exhibit remarkably superior effects in terms of reproducibility, stability, and functionality, and thus are expected to be very useful for cell therapeutic agents or for the screening of therapeutic drugs.

The present invention relates to a biomimetic nerve chip for evaluating the efficacy and toxicity of a drug, a method for evaluating the efficacy of a drug on nerve cells through astrocytes by using the biomimetic nerve chip, and a method for evaluating the toxicity of a drug on nerve cells through astrocytes by using the biomimetic nerve chip, the biomimetic nerve chip comprising: an astrocyte supply unit and a nerve cell supply unit for simulating nerve tissue; and a culture solution supply unit for supplying a culture solution to the astrocyte supply unit and the nerve cell supply unit. By using the biomimetic nerve chip for evaluating the efficacy and toxicity of a drug provided in the present invention, it is possible to overcome inaccuracies due to differences between the different species in animal experiments in the study of nerve tissues, and using a combination of astrocytes and nerve cells enables use of the nerve chip as a platform to more accurately evaluate the efficacy and toxicity of a drug under conditions similar to in vivo conditions, and the nerve chip can be applied to studies of microenvironments in nerve tissues and other organ-on-a-chip studies. Therefore, the present invention may be utilized in the development of a human-on-a-chip that can effectively analyze the efficacy and toxicity of a drug.

The present disclosure provides methods for the treatment of a mammal having a neurological condition, disease, or injury. The methods involve increasing the number of functional GABAergic interneurons at or near the site of the neurological disease, injury, or condition.

The present application provides materials and methods for treating a patient with one or more condition associated with FXN whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of FXN gene in a cell by genome editing.

The present invention relates to a method, which patterns 3D organoids prepared from human pluripotent stem cells and chops the same so as to culture oligodendrocyte progenitor cells, and induces the differentiation thereof so as to obtain a large quantity of finally differentiated oligodendrocytes. Compared to cells differentiated by a conventional differentiation method, oligodendrocytes obtained in a large quantity have the same or superior reproducibility, stability, and functionality and have remarkably shortened differentiation time, and thus are expected to be very useful for cell therapeutic agents or for screening for therapeutic drugs.

This invention relates to genetic engineering, in particular to an insertion site for a transgene, cells comprising a transgene or other modification at that insertion site, vectors for targeting that insertion site, and methods for creating transgenic cells by insertion or other modification at that site. The insertion site, or “safe harbour locus”, is identified within the SPATA13 gene on human chromosome 13q12.12. Mammalian cells comprising a genetic modification within the SPATA13 gene on chromosome 13q12.12 are described, wherein the modification may be an insertion such as an integrated transgene. Nucleic acid molecules able and adapted to guide the insertion of a transgene to that insertion site are also described. These cells or nucleic acids may be useful in therapy.

The present invention discloses, in one embodiment, a method of using human induced pluripotent stem cells to generate three-dimensional human organ tissue for therapeutic drug toxicity and discovery⋅. In one embodiment, a high throughput microtiter plate is loaded with both wild type and Rett disease 3D spheroids and exposed to a drug library, and activity is measured and analyzed for disease rescue to wild type cell behavior.

A method for producing a cell construct including, contacting Schwann cells or Schwann cell-like cells with a biocompatible matrix, and subjecting to cultivation, where the cultivation is at least partially performed by administering mechanical stimulation on the cells in contact with the biocompatible matrix. A cell construct obtained by the method.

A method for rejuvenating glial progenitor cells and rejuvenated glial progenitor cells rejuvenated by such method are disclosed. The method comprises introducing a population of genetically modified glial progenitor cells into the brain and/or brain stem of a subject, wherein the genetically modified glial progenitor cells have increased expression of one or more genes compared to the same type of glial progenitor cells that have not been genetically modified, and wherein the one or more genes are selected from the group consisting of ARX, CEBPZ, DLX1, DLX2, ELK1, ETS1, ETV4, KLF16, MYBL2, MYC, NFYB, POU3F1, SMAD1, SOX3, SP5, TCF12, TFDP1, TP53, ZIC3 and ZNF195.

A method of direct transdifferentiation of somatic cells into other somatic cells may be convenient and still have good reproducibility, excellent production efficiency, and short performed time. Methods for direct transdifferentiation of somatic cells into other somatic cells may include: (a) introducing a GLIS family gene, a mutated GLIS family gene or a gene product thereof into somatic cells; and (b) culturing the gene-introduced somatic cells in a culture medium containing a component that induces differentiation of the somatic cells or precursor cells of the somatic cells into other somatic cells.