公开(公告)号：US20230057355A1

公开(公告)日：2023-02-23

申请号：US17430768

申请日：2020-02-11

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  John MARIANI

IPC: A61K35/30 ,  A61K45/06 ,  C12N5/079 ,  A61P25/00

Abstract: The present disclosure relates to methods of treating a human subject having a condition mediated by a deficiency in myelin and methods of increasing oligodendrocyte production from human glial progenitor cells. These methods involve selecting a human subject having a condition mediated by a deficiency in myelin or providing a population of human glial progenitor cells and administering to the subject or the population of human glial progenitor cells one or modulators of a cell signaling pathway selected from the group consisting of Notch signaling, cAMP signaling, CIP2A signaling, RXRA signaling, TCF7L2 signaling, and combinations thereof under conditions effective to treat the condition or increase oligodendrocyte production.

公开(公告)号：US20230226116A1

公开(公告)日：2023-07-20

申请号：US18046930

申请日：2022-10-16

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  John MARIANI ,  Nguyen P.T. HUYNH

IPC: A61K35/30 ,  C12N5/079 ,  C12N15/90 ,  C12N15/11 ,  C12N9/22 ,  A61P25/28

CPC classification number: A61K35/30 ,  C12N5/0622 ,  C12N15/907 ,  C12N15/11 ,  C12N9/22 ,  A61P25/28 ,  C12N2506/02 ,  C12N2310/20 ,  C12N2800/80

Abstract: 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.

公开(公告)号：US20240165269A1

公开(公告)日：2024-05-23

申请号：US18487740

申请日：2023-10-16

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  John MARIANI ,  Abdellatif BENRAISS ,  Benjamin MANSKY

IPC: A61K48/00 ,  A61K38/17 ,  A61P25/28 ,  C12N15/86

CPC classification number: A61K48/0066 ,  A61K38/1709 ,  A61K48/0033 ,  A61P25/28 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2750/14143 ,  C12N2830/003 ,  C12N2830/008

Abstract: The present disclosure relates to methods for inducing rejuvenation in a population of glial progenitor cells and methods for treating a subject having a glial cell-related disorder. Methods include administering, to the population of adult glial progenitor cells, an effective amount of an expression vector comprising a nucleotide sequence encoding B-cell lymphoma/leukemia 11A (BCL11A) and a regulatory element operably linked to the nucleotide sequence.