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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.
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公开(公告)号:US20240084265A1
公开(公告)日:2024-03-14
申请号:US18453112
申请日:2023-08-21
Inventor: Ralf Jauch , Haoqing Hu , Mingxi Weng , Hoi Hang Ho
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.
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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.
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公开(公告)号:US11866733B2
公开(公告)日:2024-01-09
申请号:US16322087
申请日:2017-07-31
Inventor: Alejandro Soto-Gutierrez , Alexandra Sylvie Collin de l'Hortet , Kan Handa , Jorge Guzman Lepe , Yang Wang , Kazuki Takeishi , Ira Jacob Fox
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.
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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.
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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.
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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.
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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
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.
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公开(公告)号:US20180346933A1
公开(公告)日:2018-12-06
申请号:US15958965
申请日:2018-04-20
Inventor: IGOR KOGUT , DENNIS R. ROOP , GANNA BILOUSOVA , PATRICK S. MCGRATH
CPC classification number: A61K31/713 , C12N5/0696 , C12N15/113 , C12N2310/141 , C12N2330/31 , C12N2500/60 , C12N2501/115 , C12N2501/602 , C12N2501/603 , C12N2501/604 , C12N2501/605 , C12N2501/606 , C12N2501/608 , C12N2501/65 , C12N2506/1307 , C12N2510/00
Abstract: The present disclosure relates to methods and compositions for reprogramming cells to a pluripotent state. In particular, it relates to an integration- and feeder cell-free method for reprogramming primary human fibroblast cells to induced pluripotent stem cells (iPSCs).
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公开(公告)号:US20180320138A1
公开(公告)日:2018-11-08
申请号:US15773960
申请日:2016-11-07
Applicant: CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED (CIBER) , CENTRO DE INVESTIGACIONES ENERGÉTICAS, MEDIOAMBIENTALES Y TECNOLÓGICAS , FUNDACIÓN INSTITUTO DE INVESTIGACIÓ SANITARIA FUNDACIÓN JIMÉNEZ DÍAZ , CELLECTIS
Inventor: José Carlos SEGOVIA SANZ , Oscar QUINTANA BUSTAMANTE , Zita Maite GÁRATE MUTILOA , Juan Antonio BUEREN RONCERO , Brian R. DAVIS , Roman GALETTO , Agnes GOUBLE , Laurent POIROT
IPC: C12N5/0789 , C12N5/074
CPC classification number: C12N5/0647 , C12N5/0696 , C12N2500/25 , C12N2501/105 , C12N2501/113 , C12N2501/115 , C12N2501/125 , C12N2501/14 , C12N2501/145 , C12N2501/155 , C12N2501/16 , C12N2501/165 , C12N2501/22 , C12N2501/2303 , C12N2501/2311 , C12N2501/26 , C12N2501/415 , C12N2501/602 , C12N2501/603 , C12N2501/604 , C12N2501/606 , C12N2501/727 , C12N2501/91 , C12N2506/11 , C12N2506/45 , C12N2510/00
Abstract: The present invention relates to the medical field, in particular to gene editing as a therapeutic approach for the treatment of metabolic diseases affecting the erythroid lineage in a mammalian subject. In invention particular embodiment it refers to the combination of cell reprograming and gene editing for PKD correction as a first example of the potential application of these advanced technologies to metabolic diseases affecting the erythroid lineage. In this sense, PKD patient-specific iPSCs were efficiently generated from PB-MNCs (peripheral blood mononuclear cells) by a SeV non-integrative system and efficiently use to treat pyruvate kinase deficiency. The gene editing strategy for PKLR gene correction was also successfully applied directly to hematopoietic progenitors.
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