公开(公告)号：US20230292719A1

公开(公告)日：2023-09-21

申请号：US18045148

申请日：2022-10-08

Applicant: UNIVERSITY OF ROCHESTER ,  UNIVERSITY OF COPENHAGEN

Inventor： Steven A. GOLDMAN ,  Ricardo de Costa Barbedo VIEIRA

IPC: A01K67/027

CPC classification number: A01K67/0271 ,  A01K2207/15 ,  A01K2227/105 ,  A01K2267/0318

Abstract: A chimeric non-human mammal disease model, wherein (1) at least 30% of all the glial cells in the corpus callosum of the chimeric non-human mammal are human glial cells, and/or (2) at least 5% of all of the glial cells in the white matter of the brain and/or brain stem of the chimeric non-human mammal are human glial cells, and wherein the human glial cells comprise a combination of a first group of human glial cells tagged with a first label and a second group of human glial cells tagged with a second label that is distinguishable from the first label.

公开(公告)号：US20220062378A1

公开(公告)日：2022-03-03

申请号：US17254734

申请日：2019-06-19

Applicant: UNIVERSITY OF ROCHESTER ,  UNIVERSITY OF COPENHAGEN, A DENMARK UNIVERSITY

Inventor： Steven A. GOLDMAN ,  Mikhail OSIPOVITCH

IPC: A61K38/17 ,  A61K35/30 ,  A61P25/28

Abstract: The disclosure herein relates generally to a method of treating or inhibiting onset of Huntington's disease. This method involves selecting a subject having or at risk of having Huntington's disease and administering to the subject one or modulators of one or more genes as described herein, or proteins encoded therefrom, under conditions effective to treat or inhibit onset of Huntington's disease in the subject.

公开(公告)号：US20210260002A1

公开(公告)日：2021-08-26

申请号：US17254008

申请日：2019-06-18

Applicant: UNIVERSITY OF ROCHESTER ,  UNIVERSITY OF COPENHAGEN

Inventor： Steven A. GOLDMAN ,  Zhengshan LIU ,  Mikhail OSIPOVITCH

IPC: A61K31/19 ,  A61K31/4184 ,  A61K31/5377 ,  C12N15/113 ,  A61P25/28

Abstract: The present disclosure is directed to methods of restoring glial cell K+ uptake in a subject. This method involves selecting a subject having impaired glial cell K+ uptake, and administering, to the selected subject, a RE1-Silencing Transcription factor (REST) inhibitor under conditions effective to restore glial cell K+ uptake. Subjects having impaired glial cell K+ uptake include those at risk of having or having a neuropsychiatric disease or disorder.

公开(公告)号：US20240167034A1

公开(公告)日：2024-05-23

申请号：US18467442

申请日：2023-09-14

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  John N. MARIANI ,  ABDELLATIF BENRAISS

IPC: C12N15/113 ,  A61K38/17 ,  A61K38/45 ,  A61K38/50 ,  A61K48/00 ,  A61P25/00 ,  A61P25/28 ,  C12N9/10 ,  C12N9/80 ,  C12N15/86

CPC classification number: C12N15/113 ,  A61K38/1709 ,  A61K38/45 ,  A61K38/50 ,  A61K48/0058 ,  A61K48/0066 ,  A61P25/00 ,  A61P25/28 ,  C12N9/1007 ,  C12N9/80 ,  C12N15/86 ,  C12Y305/01098 ,  C12N2310/11 ,  C12N2310/141 ,  C12N2320/35 ,  C12N2740/15032 ,  C12N2740/15043

Abstract: The present disclosure is directed to methods of inducing rejuvenation in a population of adult glial progenitor cells, and methods of treating a subject having a myelin deficiency. The method of inducing rejuvenation in a population of adult glial progenitor cells may comprise administering, to the population of adult glial progenitor cells, an effective amount of an agent that suppresses one or more transcription factors selected from the group consisting of (i) zinc finger protein 274 (ZNF274), (ii) Myc-associated factor X (MAX), (iii) E2F transcription factor 6 (E2F6), (iv) zinc finger protein Aiolos (IKZF3), and (v) signal transducer and activator of transcription 3 (STAT3).

公开(公告)号：US20230293594A1

公开(公告)日：2023-09-21

申请号：US18135543

申请日：2023-04-17

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  Maiken NEDERGAARD

IPC: A61K35/30 ,  A61P25/18 ,  A61K31/495 ,  C12N5/0735 ,  C12N5/074

CPC classification number: A61K35/30 ,  A61K31/495 ,  A61P25/18 ,  C12N5/0606 ,  C12N5/0696 ,  A61K9/0019

Abstract: The present disclosure is directed to a method of treating a neuropsychiatric disorder. This method involves selecting a subject having the neuropsychiatric disorder and administering to the selected subject a preparation of glial progenitor cells at a dosage effective to treat the neuropsychiatric disorder in the subject. Another aspect of the disclosure is directed to a method of treating a neuropsychiatric disorder that includes selecting a subject having the neuropsychiatric disorder and administering, to the selected subject, a potassium (K+) channel activator at a dosage effective to restore normal brain interstitial glial K+ levels in the selected subject and treat the neuropsychiatric disorder is also disclosed.

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：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.

公开(公告)号：US20230243811A1

公开(公告)日：2023-08-03

申请号：US17920140

申请日：2021-04-20

Applicant: UNIVERSITY OF ROCHESTER ,  UNIVERSITY OF CØPENHAGEN ,  RIGSHOPITALET (REGH)

Inventor： Steven A. GOLDMAN ,  Abdellatif BENRAISS ,  Gitte Moos KNUDSEN

IPC: G01N33/50 ,  C12N15/86

CPC classification number: G01N33/5088 ,  C12N15/86 ,  C12N2510/00

Abstract: The present disclosure is directed to systems for in vivo tracking of target cells resulting from implantation of a preparation of cells. The present disclosure is further directed to in vivo methods of tracking a preparation of cells implanted in a subject, and of preparations of cells.

公开(公告)号：US20220290099A1

公开(公告)日：2022-09-15

申请号：US17701267

申请日：2022-03-22

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  Su WANG

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

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：US20220267737A1

公开(公告)日：2022-08-25

申请号：US17627894

申请日：2020-07-20

Applicant: UNIVERSITY OF ROCHESTER

Inventor： Steven A. GOLDMAN ,  Abdellatif BENRAISS ,  Christina TROJEL-HANSEN

IPC: C12N5/074 ,  C07K14/705 ,  C12N15/113

Abstract: The present disclosure is directed to preparation of one or more cells, wherein cells of the preparation are modified to conditionally express (i) increased levels of one or more immune checkpoint proteins as compared to corresponding wild-type cells, (ii) reduced levels of one or more HLA-I proteins as compared to corresponding wild-type cells, or a combination of (i) and (ii). The present disclosure is further directed to methods and constructs for producing the cell preparations as well as methods of administering the cell preparation to a subject in need thereof.