公开(公告)号：US20230159890A1

公开(公告)日：2023-05-25

申请号：US18046928

申请日：2022-10-16

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  John Mariani

IPC: C12N5/079 ,  A61P25/28 ,  C12N5/0797

CPC classification number: C12N5/0622 ,  A61P25/28 ,  C12N5/0623 ,  C12N2510/00

Abstract: A genetically modified glial cells, and use of such cells for rejuvenating glial cell population or treating glial cell-related disorders are disclosed. A method of treating a disorder of the brain and/or brain stem in a subject by introducing a population of genetically modified glial progenitor cells into the brain and/or brain stem of the 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, wherein said increased expression of the one or more genes in the genetically modified glial progenitor cells confer competitive advantage over native or already resident glial progenitor cells in the subject.

公开(公告)号：US20250152738A1

公开(公告)日：2025-05-15

申请号：US18834928

申请日：2023-02-01

Applicant: University of Rochester

Inventor： Steven A. Goldman ,  Abdellatif Benraiss ,  John Mariani

IPC: A61K48/00 ,  C07K14/47 ,  C07K14/705 ,  C12N15/86

Abstract: The present disclosure relates to a method of treating a disease or disorder in a subject in need thereof. This method involves providing a recombinant genetic construct comprising: (i) a G-protein coupled receptor (GPR17) promoter-inclusive regulatory element sequence having a 5′ and a 3′ end and (ii) a nucleic acid sequence encoding a molecule of interest, wherein said nucleic acid sequence is heterologous to the GPR17 promoter-inclusive regulatory element and wherein said nucleic acid sequence is positioned 3′ to the GPR17 promoter-inclusive regulatory element sequence; an expression vector comprising the recombinant genetic construct; a pharmaceutical composition comprising the recombinant genetic construct or the expression vector; or a preparation of cells comprising the recombinant genetic construct or the expression vector. This method further involves administering, to the subject in need, an effective amount of the recombinant genetic construct, the expression vector, the pharmaceutical composition, or the preparation of cells.

公开(公告)号：US20230270818A1

公开(公告)日：2023-08-31

申请号：US18051653

申请日：2022-11-01

Applicant: University of Rochester

Inventor： Steven A. Goldman ,  Abdellatif Benriass ,  John Mariani

IPC: A61K38/17 ,  C12N5/079 ,  A61K48/00 ,  A61P25/28

CPC classification number: A61K38/1709 ,  C12N5/0622 ,  A61K48/0058 ,  A61P25/28 ,  C12N2506/08

Abstract: The present application relates to a method of treating a subject having a condition mediated by a deficiency in myelin. This method involves selecting a subject having a condition mediated by a deficiency in myelin and expressing a transcription factor 7-like 2 (TCF7L2) protein in the selected subject under conditions effective to treat the condition. Also disclosed is a method of increasing oligodendrocyte production from glial progenitor cells. This method involves providing a population of glial progenitor cells and expressing a TCF7L2 protein in the provided population of glial progenitor cells under conditions effective to increase oligodendrocyte production compared to oligodendrocyte production absent said administering. Also disclosed is a genetic construct suitable, inter alia, for carrying out these methods.