公开(公告)号：US11596700B2

公开(公告)日：2023-03-07

申请号：US15429559

申请日：2017-02-10

Applicant: University of Rochester

Inventor： Steven A. Goldman

IPC: A61K35/30 ,  A61K49/00 ,  A61K35/545 ,  A01K67/027 ,  C12N5/079 ,  G01N33/50 ,  C12N5/07 ,  A61K45/06

Abstract: The present application relates to a non-human mammal model of a human neurodegenerative disorder, methods of producing the non-human mammal model, and methods of using the non-human mammal model to identify agents suitable for treating a neurodegenerative disorder. The present application also relates to methods of treating neurodegenerative disorders and restoring normal brain interstitial potassium levels.

公开(公告)号：US20250019653A1

公开(公告)日：2025-01-16

申请号：US18896037

申请日：2024-09-25

Applicant: University of Rochester

Inventor： Steven A. Goldman ,  Su Wang

IPC: C12N5/079 ,  A61K9/00 ,  A61K35/30 ,  C12N5/0735

Abstract: The present disclosure relates to a preparation of CD140a/PDGFRα positive cells that comprises oligodendrocyte progenitor cells co-expressing OLIG2 and CD140a/PDGFRα. The preparation of cells is derived from pluripotent cells that were derived from skin cells, fibroblasts, umbilical cord blood, peripheral blood, bone marrow, or other somatic cells. The cell preparation has an in vivo myelination efficiency that is equal to or greater than the in vivo myelination efficiency of a preparation of A2B5+/PSA-NCAM− sorted fetal human tissue derived oligodendrocyte progenitor cells. Methods of making, isolating and using the disclosed cell preparation are also described.

公开(公告)号：US10279051B2

公开(公告)日：2019-05-07

申请号：US15429585

申请日：2017-02-10

Applicant: University of Rochester

Inventor： Steven A. Goldman

IPC: C12N5/07 ,  A61K35/30 ,  A61K45/06 ,  A61K49/00 ,  C12N5/079 ,  G01N33/50 ,  A01K67/027 ,  A61K35/545

Abstract: The present application relates to a non-human mammal model of a human neurodegenerative disorder, methods of producing the non-human mammal model, and methods of using the non-human mammal model to identify agents suitable for treating a neurodegenerative disorder. The present application also relates to methods of treating neurodegenerative disorders and restoring normal brain interstitial potassium levels.

公开(公告)号：US20250073354A1

公开(公告)日：2025-03-06

申请号：US18722693

申请日：2022-12-22

Applicant: University of Rochester

Inventor： Maiken Nedergaard ,  Steven A. Goldman

IPC: A61K48/00 ,  A61P27/16 ,  C07K14/47 ,  C12N15/86

Abstract: This disclosure relates compositions and methods to delivery of various agents to the inner ear of a subject.

公开(公告)号：US12129484B2

公开(公告)日：2024-10-29

申请号：US16659110

申请日：2019-10-21

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. Goldman ,  Su Wang

IPC: A61K35/30 ,  A61K9/00 ,  C12N5/079 ,  C12N5/0735

CPC classification number: C12N5/0622 ,  A61K9/0085 ,  A61K35/30 ,  C12N5/0606 ,  C12N2506/02 ,  C12N2506/094 ,  C12N2506/45

Abstract: The present disclosure relates to a preparation of CD140a/PDGFRα positive cells that comprises oligodendrocyte progenitor cells co-expressing OLIG2 and CD140a/PDGFRα. The preparation of cells is derived from pluripotent cells that were derived from skin cells, fibroblasts, umbilical cord blood, peripheral blood, bone marrow, or other somatic cells. The cell preparation has an in vivo myelination efficiency that is equal to or greater than the in vivo myelination efficiency of a preparation of A2B5+/PSA-NCAM− sorted fetal human tissue derived oligodendrocyte progenitor cells. Methods of making, isolating and using the disclosed cell preparation are also described.

公开(公告)号：US20230241118A1

公开(公告)日：2023-08-03

申请号：US18047831

申请日：2022-10-19

Applicant: University of Rochester ,  University of Copenhagen

Inventor： Steven A. Goldman ,  Ricardo da Costa Barbedo Vieira

IPC: A61K35/30 ,  A61P25/28 ,  C12N15/113 ,  A61K31/7088

CPC classification number: A61K35/30 ,  A61P25/28 ,  C12N15/113 ,  A61K31/7088 ,  C12N2310/141

Abstract: The present application relates to alleviating adverse effects of oligodendrocyte loss, astrocyte loss, or white matter loss, including age-related oligodendrocyte loss, age-related astrocyte loss, or age-related white matter loss, in the brain of a subject. The present application also relates to rejuvenating a glial progenitor cell or a progeny thereof, or to enhancing the development potential of a glial progenitor cell or a progeny thereof.

公开(公告)号：US10779519B2

公开(公告)日：2020-09-22

申请号：US14710144

申请日：2015-05-12

Applicant: University of Rochester

Inventor： Steven A. Goldman ,  Martha Windrem

IPC: G01N33/00 ,  A01K67/027 ,  A61K49/00 ,  G01N33/50 ,  G01N33/569 ,  C12Q1/70

Abstract: The present invention is directed to a method of assessing in vivo human glial cell response to pathogenic infection that involves providing a non-human mammal either with at least 30% of its glial cells in its corpus callosum being human glial cells and/or with at least 5% of its glial cells its brain and brain stem white matter being human glial cells, subjecting the non-human mammal to pathogenic infection and assessing the in vivo human glial cell response to pathogenic infection. A method of identifying therapeutic agents for the pathogenic infection as well as forms of the non-human mammal having a pathogenic brain infection are also disclosed.

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：US20230277600A1

公开(公告)日：2023-09-07

申请号：US18047758

申请日：2022-10-19

Applicant: University of Rochester ,  University of Copenhagen

Inventor： Steven A. Goldman ,  Ricardo da Costa Barbedo Vieira

IPC: A61K35/30 ,  A61P25/28

CPC classification number: A61K35/30 ,  A61P25/28

Abstract: The present application relates to alleviating adverse effects of oligodendrocyte loss, astrocyte loss, or white matter loss, including age-related oligodendrocyte loss, age-related astrocyte loss, or age-related white matter loss, in the brain of a subject. The present application also relates to rejuvenating a glial progenitor cell or a progeny thereof, or to enhancing the development potential of a glial progenitor cell or a progeny thereof.

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.