公开(公告)号：US12059479B2

公开(公告)日：2024-08-13

申请号：US17463007

申请日：2021-08-31

申请人： CELLSCRIPT, LLC

发明人： Judith Meis ,  Anthony Person ,  Cynthia Chin ,  Jerome Jendrisak ,  Gary Dahl

IPC分类号： A61K48/00 ,  A61K31/7105 ,  A61K33/06 ,  A61K38/00 ,  C12N5/074 ,  C12N5/077 ,  C12N5/0793 ,  C12N15/10 ,  C12N15/87 ,  C12P19/34 ,  C12P19/38 ,  C12P21/02

CPC分类号： A61K48/0066 ,  A61K31/7105 ,  A61K33/06 ,  A61K38/005 ,  A61K48/0041 ,  C12N5/0619 ,  C12N5/0658 ,  C12N5/0696 ,  C12N15/1003 ,  C12N15/1096 ,  C12N15/87 ,  C12P19/34 ,  C12P19/38 ,  C12P21/02 ,  C12N2501/60 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2501/73 ,  C12N2506/1307 ,  C12N2510/00 ,  C12Y301/26003 ,  A61K33/06 ,  A61K2300/00 ,  A61K31/7105 ,  A61K2300/00

摘要： The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.

公开(公告)号：US20240254511A1

公开(公告)日：2024-08-01

申请号：US18508761

申请日：2023-11-14

申请人： Whitehead Institute of Biomedical Research

发明人： Rudolf Jaenisch ,  Bryce Woodbury Carey ,  Yaqub Hanna

IPC分类号： C12N15/86 ,  A01K67/0271 ,  C07K14/47 ,  C12N5/074 ,  G01N33/50 ,  C12N5/0735 ,  C12N15/79 ,  C12N15/85

CPC分类号： 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

摘要： 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：US12043653B2

公开(公告)日：2024-07-23

申请号：US17750530

申请日：2022-05-23

申请人： Massachusetts Eye and Ear Infirmary ,  President and Fellows of Harvard College

发明人： Zheng-Yi Chen ,  David R. Liu ,  Margie Li ,  David B. Thompson ,  John Zuris

IPC分类号： C07K14/705 ,  A61K9/00 ,  A61K9/50 ,  A61K38/00 ,  A61P27/16 ,  C07K14/435 ,  C07K14/46 ,  C07K14/47 ,  C12N5/071 ,  C12N5/0793

CPC分类号： C07K14/705 ,  A61K9/0046 ,  A61K9/5068 ,  A61P27/16 ,  C07K14/43595 ,  C07K14/463 ,  C07K14/47 ,  C12N5/062 ,  C12N5/0627 ,  A61K38/00 ,  C07K2319/00 ,  C12N2501/42 ,  C12N2501/60 ,  C12N2501/606 ,  C12N2501/998 ,  C12N2506/00

摘要： Compositions and provided to induce cells of the inner ear to renter the cell cycle and to proliferate. In particular, hair cells are induced to proliferate by administration of a composition which activates the Myc and Notch. Supporting cells are induced to transdifferentiate to hair cells by inhibition of Myc and Notch activity or the activation of Atoh1. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.

公开(公告)号：US20240228977A1

公开(公告)日：2024-07-11

申请号：US18449100

申请日：2023-08-14

申请人： FUJIFILM Cellular Dynamics, Inc.

发明人： Amanda MACK

IPC分类号： C12N5/074 ,  C12N15/85

CPC分类号： C12N5/0696 ,  C12N15/85 ,  C12N2500/98 ,  C12N2501/115 ,  C12N2501/125 ,  C12N2501/145 ,  C12N2501/15 ,  C12N2501/2303 ,  C12N2501/2306 ,  C12N2501/26 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2501/727 ,  C12N2501/998 ,  C12N2506/11 ,  C12N2510/00

摘要： Methods and compositions relating to the production of induced pluripotent stem cells (iPS cells) are disclosed. For example, induced pluripotent stem cells may be generated from peripheral blood cells, such as human blood progenitor cells, using episomal reprogramming and feeder-free or xeno-free conditions. In certain embodiments, the invention provides novel methods for improving overall reprogramming efficiency with low number of blood progenitor cells.

公开(公告)号：US20240066070A1

公开(公告)日：2024-02-29

申请号：US18272044

申请日：2022-01-11

申请人： AgeX Therapeutics, Inc.

发明人： Michael D. West

IPC分类号： A61K35/42 ,  A61K35/36 ,  A61L27/38 ,  A61L27/52 ,  C12N5/071

CPC分类号： A61K35/42 ,  A61K35/36 ,  A61L27/38 ,  A61L27/52 ,  C12N5/0625 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2510/00

摘要： Methods are provided for the ex vivo reprogramming of microbiopsies of tissue such as from the skin, wherein the cells from the tissue are reverted to a state capable of extensive expansion in volume and when transplanted in vivo, of promoting scarless tissue regeneration at the site of administration.

公开(公告)号：US20240018485A1

公开(公告)日：2024-01-18

申请号：US18182258

申请日：2023-03-10

申请人： Memorial Sloan-Kettering Cancer Center

发明人： Kitai KIM ,  Maria SKAMAGKI ,  Yildirim DOGAN

IPC分类号： C12N5/074 ,  C07K14/47 ,  C12N5/10 ,  C12N15/85

CPC分类号： C12N5/0696 ,  C07K14/4702 ,  C12N5/10 ,  C12N15/85 ,  C12N2501/50 ,  C12N2501/602 ,  C12N2510/00 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2501/71 ,  C12N2501/998 ,  C12N2501/603

摘要： Disclosed are findings that: (a) induced pluripotent stem cells derived from aged donors (A-iPSC) show increased genomic instability, a defect in apoptosis, a defect in glucose metabolism, and a blunted DNA damage response are compared to those derived from young donors (Y-iPSC); and (b) inhibition of excessive glutathione-mediated H202 scavenging activity, found to be associated with A-iPSC and in turn inhibiting DNA damage response and apoptosis, substantially rescues these defects and reduces the oncogenic potential of A-iPSC. Supplementation of pluripotency factor ZSCAN 10 (shown to be poorly activated in A-iPSC and to act upstream of glutathione involvement), e.g., by expression as an adjunct to the four Yamanaka iPSC reprogramming factors, led to substantial recovery of genomic stability, DNA damage response, and apoptosis in A-iPSC through enhancing GLUT3 and normalizing homeostasis of glutathione/H202; GLUT3 (a pluripotent stem cell-specific glucose transporter acting upstream of glutathione and also poorly activated in A-iPSC) has similar effects, indicating that inhibition of glutathione/H202 notably through delivery of ZSCAN 10 and/or GLUT3 and/or an exosome subunit will be clinically useful, resulting in A-iPSC of improved properties and reduced oncogenic potential.

公开(公告)号：US20230313148A1

公开(公告)日：2023-10-05

申请号：US17995969

申请日：2021-04-16

申请人： Roslin Technologies Limited

发明人： Patrick Joe Mee ,  Melany Jackson ,  Ryan Taylor

IPC分类号： C12N5/074 ,  C12N15/86

CPC分类号： C12N5/0696 ,  C12N15/86 ,  C12N2506/08 ,  C12N2501/603 ,  C12N2501/606 ,  C12N2710/24143

摘要： A method of inducing pluripotency in somatic cells derived from a non-human domestic animal or farm animal comprises culturing neural stem cells (NSCs) in the presence of vectors that express one or more reprogramming factors. Canine, porcine and bovine iPSCs are obtained with distinct genetic marker profiles.

公开(公告)号：US11739300B2

公开(公告)日：2023-08-29

申请号：US16827098

申请日：2020-03-23

申请人： The Trustees of the University of Pennsylvania

发明人： Katalin Kariko ,  Drew Weissman ,  Gary Dahl ,  Anthony Person ,  Judith Meis ,  Jerome Jendrisak

IPC分类号： C12N5/074 ,  C12N15/87 ,  C12N15/85 ,  A61K38/17 ,  A61K38/18 ,  A61K38/44 ,  A61K38/46 ,  A61K38/50 ,  A61K48/00 ,  C07K14/47 ,  C07K14/505 ,  C12N9/02 ,  C12N9/22 ,  C12N9/78 ,  C12N15/117

CPC分类号： C12N5/0696 ,  A61K38/1709 ,  A61K38/1816 ,  A61K38/44 ,  A61K38/465 ,  A61K38/50 ,  A61K48/005 ,  A61K48/0075 ,  C07K14/47 ,  C07K14/4712 ,  C07K14/505 ,  C12N9/0075 ,  C12N9/22 ,  C12N9/78 ,  C12N15/117 ,  C12N15/85 ,  C12N15/87 ,  C12N2310/17 ,  C12N2310/335 ,  C12N2320/30 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/605 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2506/02 ,  C12Y114/13039 ,  C12Y301/04012 ,  C12Y305/04004

摘要： The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and/or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and/or single-stranded run-on mRNAs.

公开(公告)号：US20190225944A1

公开(公告)日：2019-07-25

申请号：US16374482

申请日：2019-04-03

申请人： Factor Bioscience Inc.

发明人： Matthew Angel ,  Christopher Rohde

IPC分类号： C12N5/074 ,  C12N5/071 ,  C12N5/077 ,  C12N5/0789 ,  C12N5/095

CPC分类号： C12N5/0696 ,  C12N5/0018 ,  C12N5/0625 ,  C12N5/0647 ,  C12N5/0656 ,  C12N5/0657 ,  C12N5/0676 ,  C12N5/0695 ,  C12N2500/25 ,  C12N2500/33 ,  C12N2500/36 ,  C12N2500/84 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2501/998 ,  C12N2506/09 ,  C12N2506/45

摘要： The present invention relates in part to methods for producing tissue-specific cells from patient samples, and to tissue-specific cells produced using these methods. Methods for reprogramming cells using RNA are disclosed. Therapeutics comprising cells produced using these methods are also disclosed.

公开(公告)号：US20180327720A1

公开(公告)日：2018-11-15

申请号：US16037597

申请日：2018-07-17

申请人： Factor Bioscience Inc.

发明人： Matthew ANGEL ,  Christopher ROHDE

IPC分类号： C12N5/074 ,  C12N5/071 ,  C12N5/0789 ,  C12N5/077 ,  C12N5/095 ,  C12N5/00

CPC分类号： C12N5/0696 ,  C12N5/0018 ,  C12N5/0625 ,  C12N5/0647 ,  C12N5/0656 ,  C12N5/0657 ,  C12N5/0676 ,  C12N5/0695 ,  C12N2500/25 ,  C12N2500/33 ,  C12N2500/36 ,  C12N2500/84 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2501/998 ,  C12N2506/09 ,  C12N2506/45

摘要： The present invention relates in part to methods for producing tissue-specific cells from patient samples, and to tissue-specific cells produced using these methods. Methods for reprogramming cells using RNA are disclosed. Therapeutics comprising cells produced using these methods are also disclosed.