公开(公告)号：US20240197789A1

公开(公告)日：2024-06-20

申请号：US18488859

申请日：2023-10-17

Applicant: CITY OF HOPE

Inventor： Yanhong SHI ,  Jianfei CHAO ,  Wendong LI

IPC: A61K35/30 ,  A61K38/00 ,  C12N5/074 ,  C12N5/0797

CPC classification number: A61K35/30 ,  C12N5/0623 ,  C12N5/0696 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K38/00 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2506/1307 ,  C12N2506/45 ,  C12N2510/00 ,  C12N2740/16043

Abstract: Disclosed herein are methods of treating Canavan disease in a subject through restoring ASPA enzymatic activities in the subject by expressing exogenous wild type ASPA gene in the brain of the subject. Also disclosed are a process of producing neural precursor cells, including NPCs, glial progenitor cells and oligodendroglial progenitor cells, which express an exogenous wild type ASPA gene and the neural precursor cells produced by this process.

公开(公告)号：US20240084265A1

公开(公告)日：2024-03-14

申请号：US18453112

申请日：2023-08-21

Applicant: The University of Hong Kong ,  Centre for Translational Stem Cell Biology Limited

Inventor： Ralf Jauch ,  Haoqing Hu ,  Mingxi Weng ,  Hoi Hang Ho

IPC: C12N5/074 ,  C12N15/86

CPC classification number: C12N5/0696 ,  C12N15/86 ,  C12N2501/60 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2506/11 ,  C12N2506/1307

Abstract: Compositions of transcription factor cocktails including engineered SOX17 factor (eSOX17), together with one or more of OCT4, KLF4, and C-MYC, and vectors carrying one or more of these transcription factors suitable for delivery to donor somatic cells for generating induced expanded potential stem cells (iEPSCs) or induced neural stem cells (iNSCs) are provided. The disclosed compositions deliver reprograming of human somatic cells into a totipotency-like, or multipotency-like state with improved efficiency. Compositions of eSOX17-derived iEPSCs and iNSCs generated according to the described methods are also described. The methods engineer somatic cells to express one or more markers of pluripotency or neural stem cells. Methods of using iEPSCs or iNSCs in studying early human development and in cell therapy are also provided.

公开(公告)号：US11898169B2

公开(公告)日：2024-02-13

申请号：US17239059

申请日：2021-04-23

Applicant: The McLean Hospital Corporation

Inventor： Kwang-Soo Kim ,  Young Cha

IPC: C12N5/074 ,  C12N15/113 ,  A61K35/12

CPC classification number: C12N5/0696 ,  A61K35/12 ,  C12N15/113 ,  C12N2310/141 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2501/65 ,  C12N2506/1307 ,  C12N2510/00 ,  C12N2799/022 ,  C12N2310/141 ,  C12N2330/10

Abstract: Disclosed herein are methods of generating induced pluripotent stem cells. The method involves providing a quantity of somatic or non-embryonic cells, contacting the contacting the somatic or non-embryonic cells with a quantity of one or more programming factors and one or more RNA molecules, and culturing the somatic or non-embryonic cells for a period of time sufficient to generate at least one induced pluripotent stem cell. Various reprogramming factors and RNA molecules for use in the methods are disclosed herein. Also disclosed are cell lines and pharmaceutical compositions generated by use of the methods.

公开(公告)号：US20240034996A1

公开(公告)日：2024-02-01

申请号：US18361712

申请日：2023-07-28

Applicant: Therapeutic Solutions International, Inc.

Inventor： Thomas E. ICHIM ,  Timothy G. DIXON ,  Famela RAMOS ,  James VELTMEYER

IPC: C12N5/0784 ,  C12N5/0775

CPC classification number: C12N5/0639 ,  C12N5/0662 ,  C12N2506/45 ,  C12N2501/60 ,  C12N2501/603 ,  C12N2501/602 ,  C12N2501/605 ,  C12N2501/73

Abstract: Disclosed are cells and cellular compositions useful for treatment of degenerative and/or autoimmune diseases derived from gene edited/gene modified pluripotent stem cells. In one embodiment pluripotent stem cell such as inducible pluripotent stem cells are gene modified to express tissue associated transcription factors such as pdx-1 if endodermal tissue is desired and cells are differentiated into regenerative-type cells such as along the mesenchymal lineage. In one embodiment the invention teaches transfection with IL-27 to induce expression of coinhibitory molecules for suppression of autoimmunity. In some embodiments the invention provides generation of iPSC derived MSC which can not stimulate inflammation due to gene-editing based removal of inflammatory associated transcription factors.

公开(公告)号：US11866733B2

公开(公告)日：2024-01-09

申请号：US16322087

申请日：2017-07-31

Applicant: University of Pittsburgh—Of the Commonwealth System of Higher Education

Inventor： Alejandro Soto-Gutierrez ,  Alexandra Sylvie Collin de l'Hortet ,  Kan Handa ,  Jorge Guzman Lepe ,  Yang Wang ,  Kazuki Takeishi ,  Ira Jacob Fox

IPC: C12N5/074 ,  C12N15/11 ,  C12N9/22

CPC classification number: C12N5/0696 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N2310/20 ,  C12N2320/12 ,  C12N2330/51 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/608 ,  C12N2506/1307 ,  C12N2510/00

Abstract: Methods are disclosed herein for efficiently generating human induced pluripotent stem cells (iPSC) containing a nucleic acid including a doxycycline promoter operably linked to a nucleic acid encoding Cas9. These methods include transfecting a human somatic cell with a nucleic acid molecule comprising a doxycycline promoter operably linked to a nucleic acid encoding a Cas9, and constitutive promoter operably linked to a tetracycline responsive element and inducing the somatic cell to form an iPSC, thereby producing an iPSC that can undergo CRISPR/Cas9-mediated recombination at a high efficiency. The human iPSC, or a cell differentiated therefrom, is cultured in the presence of doxycycline to induce expression of the Cas9. These cells can then be used to target in any gene of interest by introducing nucleic acids encoding sgRNAs. Induced pluripotent stem cells produced by these methods are also disclosed.

公开(公告)号：US11851670B2

公开(公告)日：2023-12-26

申请号：US16438424

申请日：2019-06-11

Applicant: Whitehead Institute for Biomedical Research

Inventor： Rudolf Jaenisch ,  Bryce Woodbury Carey ,  Yaqub Hanna

IPC: C12N5/074 ,  C12N15/86 ,  G01N33/50 ,  A01K67/027 ,  C07K14/47 ,  C12N15/85 ,  C12N5/0735 ,  C12N15/79

CPC classification number: C12N15/86 ,  A01K67/0271 ,  C07K14/4705 ,  C12N5/0696 ,  G01N33/5008 ,  C12N5/0606 ,  C12N15/79 ,  C12N15/85 ,  C12N2501/60 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2506/11 ,  C12N2506/115 ,  C12N2510/00 ,  C12N2740/15041 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2799/027 ,  C12N2800/108 ,  C12N2840/203

Abstract: The disclosure relates to a method of reprogramming one or more somatic cells, e.g., partially differentiated or fully/terminally differentiated somatic cells, to a less differentiated state, e.g., a pluripotent or multipotent state. In further embodiments the invention also relates to reprogrammed somatic cells produced by methods of the invention, to chimeric animals comprising reprogrammed somatic cells of the invention, to uses of said cells, and to methods for identifying agents useful for reprogramming somatic cells.

公开(公告)号：US20230392125A1

公开(公告)日：2023-12-07

申请号：US18249661

申请日：2021-10-19

Applicant: The Board of Regents of The University of Texas System

Inventor： Jun WU ,  Leqian YU

IPC: C12N5/074 ,  C12N5/0735 ,  C12N15/85

CPC classification number: C12N5/0696 ,  C12N5/0606 ,  C12N15/85 ,  C12N2501/115 ,  C12N2501/15 ,  C12N2501/415 ,  C12N2501/16 ,  C12N2501/603 ,  C12N2501/602 ,  C12N2501/235 ,  C12N2800/107

Abstract: Aspects of the present disclosure relate to methods for producing formative pluripotent stem cells (PSCs) by culturing cells in a medium that comprises a fibroblast growth factor (FGF) activator, a transforming growth factor beta (TGF-β) activator, and a WNT activator. Also provided are in vitro culture systems for producing the formative pluripotent stem cells.

公开(公告)号：US11795439B2

公开(公告)日：2023-10-24

申请号：US16752535

申请日：2020-01-24

Applicant: GENESIS TECHNOLOGIES LIMITED

Inventor： Jan-Eric Ahlfors ,  Rouwayda El-Ayoubi

IPC: C12N5/00 ,  C12N5/074 ,  C12N5/0797 ,  C12N5/0775

CPC classification number: C12N5/0696 ,  C12N5/0623 ,  C12N5/0662 ,  C12N2501/60 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2502/99 ,  C12N2506/1307 ,  C12N2506/1384

Abstract: A method of obtaining a pancreatic multipotent or unipotent cell including providing a cell of a first type which is not a pancreatic multipotent or unipotent cell; contacting the cell of a first type with an agent capable of remodeling the chromatin and/or DNA of the cell; transiently increasing expression of at least one pancreatic multipotent or unipotent gene regulator in the cell of a first type, to a level at which the at least one pancreatic multipotent or unipotent gene regulator is capable of driving transformation of the cell of a first type into the pancreatic multipotent or unipotent cell; and placing or maintaining the cell in a pancreatic cell culture medium and maintaining intracellular levels of the at least one pancreatic multipotent or unipotent gene regulator for a sufficient period of time to allow a pancreatic multipotent or unipotent cell to be obtained.

公开(公告)号：US11714081B2

公开(公告)日：2023-08-01

申请号：US16588864

申请日：2019-09-30

Applicant: The Board of Supervisors of Louisiana State University and Agricultural and Mechanical College

Inventor： Kenneth James Eilertsen ,  Jong Rim

IPC: G01N33/50 ,  C12N5/0793

CPC classification number: G01N33/5044 ,  C12N5/0619 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2506/1384 ,  C12N2510/00 ,  G01N2500/10

Abstract: Described herein are methods of transdifferentiating preadipocytes, populations of transdifferentiated preadipocytes, and methods of using the transdifferentiated preadipocytes.

公开(公告)号：US20190241874A1

公开(公告)日：2019-08-08

申请号：US16147003

申请日：2018-09-28

Applicant: Whitehead Institute for Biomedical Research

Inventor： Rudolf Jaenisch ,  Yaqub Hanna ,  Marius Wernig ,  Christopher J. Lengner ,  Alexander Meissner ,  Oliver Tobias Brambrink ,  G. Grant Welstead ,  Ruth Foreman

IPC: C12N5/074 ,  C07K14/47

CPC classification number: C12N5/0696 ,  A01K67/0273 ,  A01K67/0275 ,  A01K2217/05 ,  A01K2227/105 ,  C07K14/4702 ,  C12N15/8509 ,  C12N15/8775 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2506/11 ,  C12N2506/13 ,  C12N2800/30 ,  C12N2830/003 ,  C12N2830/006 ,  G01N33/5023 ,  G01N2333/91011

Abstract: The disclosure relates to a method of reprogramming one or more somatic cells, e.g., partially differentiated or fully/terminally differentiated somatic cells, to a less differentiated state, e.g., a pluripotent or multipotent state. In further embodiments the invention also relates to reprogrammed somatic cells produced by methods of the invention, to uses of said cells, and to methods for identifying agents useful for reprogramming somatic cells.