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.

A method of treating a subject with dry-form age-related macular degeneration (AMD) is disclosed. The method comprises administering into the subretina of the subject a therapeutically effective amount of a pharmaceutical composition comprising human RPE cells, wherein at least 95% of the cells thereof co-express premelanosome protein (PMEL17) and cellular retinaldehyde binding protein (CRALBP), and wherein the trans-epithelial electrical resistance of the cells is greater than 100 ohms to the subject, thereby treating the subject.

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.

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.

Disclosed herein are methods and compositions for gene therapy, in particular, methods and compositions related to alterations in a capsid protein sequence for altering permissiveness of a promoter within a cell when the promoter and the capsid protein are present within the cell and the capsid protein and the promoter are in the context of a recombinant adeno-associated virus (AAV) particle.

The present invention relates to a differentiation method for obtaining a large quantity of microglia by patterning, proliferating, culturing, and inducing the differentiation of yolk sac-mimic 3D organoids prepared from human pluripotent stem cells, wherein the microglia thus obtained in a large quantity exhibit significantly superior effects in terms of yield, purity, and storage stability compared to cells differentiated by existing differentiation methods, and thus may be utilized in research on lesions and therapeutic mechanisms of brain diseases, and drug screening platforms.

The present disclosure concerns a cell or tissue culture system comprising a solid support for the culture of adherent cells or adherent tissues and a plurality of cationic dendrimers associated to the surface of the solid support. Each cationic dendrimer includes one or more functional amine group. The cationic dendrimer is protonated at physiological pH. The cell or tissue culture system can be used for the culture of adherent cells or tissues and be used for the differentiation of stem cells.

Disclosed are methods and compositions useful for treatment of blindness or dry macular degeneration. In one embodiment, retinal pigmented epithelial cells are generated from fibroblasts through induction of differentiation, and/or transdifferentiation. In another embodiment, fibroblast-derived products, such as differentiated retinal pigmented epithelial cells, are provided to subjects in a therapeutically effective amount.