THERAPEUTIC AGENT FOR DISEASE CAUSED BY DOMINANT MUTANT GENE

    公开(公告)号:US20220249698A1

    公开(公告)日:2022-08-11

    申请号:US17626042

    申请日:2020-07-10

    Applicant: RIKEN

    Abstract: Provided is a novel therapeutic agent for a disease caused by a dominant gene mutation (7). A therapeutic agent of the present invention comprises a donor DNA (20) that contains a polynucleotide having the following sequences (a) to (c): (a) a normal gene (1); (b) a first reverse target sequence (2a) that is located upstream of the normal gene (1) and that is cleaved by a designer nuclease; and (c) a second reverse target sequence (2b) that is located downstream of the normal gene (1) and that is cleaved by the designer nuclease, where the reverse target sequences (2a, 2b) each mean a sequence obtained by inverting a target sequence (6) that is present in the genome and that is cleaved by the designer nuclease.

    METHOD FOR PRODUCING RETINAL TISSUES

    公开(公告)号:US20240399026A1

    公开(公告)日:2024-12-05

    申请号:US18805131

    申请日:2024-08-14

    Abstract: The present invention aims to provide a method for suppressing differentiation of ganglion cell, amacrine cell, horizontal cell and/or bipolar cell in a neural retina tissue containing photoreceptor precursor and/or photoreceptor, and the like. A method for suppressing differentiation of a ganglion cell, an amacrine cell, a horizontal cell and/or a bipolar cell in a neural retinal tissue containing a photoreceptor precursor and/or a photoreceptor, including a step of culturing a retinal tissue comprising a neural retinal progenitor cell and in any stage between a differentiation stage immediately after emergence of a ganglion cell and a differentiation stage where emergence rate of a cone photoreceptor precursor reaches maximum in a medium containing a thyroid gland hormone signal transduction pathway agonist.

Patent Agency Ranking