-
公开(公告)号：US20220282219A1
公开(公告)日：2022-09-08
申请号：US17634766
申请日：2020-08-12
IPC分类号： C12N5/0789 , C12N5/071 , C07K14/47 , A61K35/28
摘要： The present disclosure relates to methods for expanding populations of hematopoietic stem cells (HSCs) using a genetically engineered human fetal liver niche and compositions of purified ex vivo expanded HSCs. Also provided herein are methods of using such expanded HSC cell populations for clinical applications including allogeneic hematopoietic stem cell transplantation and for drug discovery and modeling human liver development.
-
公开(公告)号：US20210254012A1
公开(公告)日：2021-08-19
申请号：US16973421
申请日：2019-06-10
IPC分类号： C12N5/071 , C12N15/86 , G01N33/50 , A61K35/407 , C12N15/90
摘要： The present disclosure relates to synthetic liver organoids and methods of using such synthetic liver organoids for various applications including drug discovery and modeling human liver development. In particular, provided herein are methods of producing and using synthetic mature liver organoids comprising mature, functional cells found in the human liver.
-
公开(公告)号：US20240002878A1
公开(公告)日：2024-01-04
申请号：US18327373
申请日：2023-06-01
IPC分类号： C12N15/85 , C12N9/22 , C12N15/11 , C12N15/115 , C12N15/62
CPC分类号： C12N15/85 , C12N9/22 , C12N15/11 , C12N15/115 , C12N15/62 , C12N2310/20 , C12N2800/80 , C12N15/907
摘要： Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.
-
公开(公告)号：US11773407B2
公开(公告)日：2023-10-03
申请号：US16626021
申请日：2018-06-26
IPC分类号： C12N15/86 , C12N9/22 , C12N15/113
CPC分类号： C12N15/86 , C12N9/22 , C12N15/113 , C12N2310/20 , C12N2750/14143
摘要： Aspects of the disclosure relate to synthetic regulatory systems composed of a multifunctional Cas nuclease, at least two guide RNAs (gRNAs) configured to target distinct nucleotide sequences, and a multilayered regulatory control element comprising ribozyme-based safety switches providing spatial and temporal control over the synthetic circuit in vivo.
-
公开(公告)号：US11208640B2
公开(公告)日：2021-12-28
申请号：US16632782
申请日：2018-04-27
发明人： Radwa Ewaisha , Samira Kiani , Shayesteh Roshdi Ferdosi , Karen Anderson , Farzaneh Moghadam , Sri Krishna , Mo Reza Ebrahimkhani
IPC分类号： C12N9/22 , G01N33/573 , A61K48/00
摘要： Provided herein are methods and compositions for reducing an undesirable T cell immune response in human patients prior to and/or during gene therapy using CRISPR/Cas9-based genetic modulation.
-
6.发明授权公开(公告)号：US11041154B2
公开(公告)日：2021-06-22
申请号：US16632792
申请日：2017-09-29
发明人： Samira Kiani , Xiao Wang , David Menn , Mo Reza Ebrahimkhani
IPC分类号： C12N15/113 , C12N9/22 , C12N15/63
摘要： Provided herein are tools for understanding and engineering dynamics of synthetic genetic circuits which utilize CRISPR components. More particularly, methods, systems, and compositions for directing Cas9 activity using a fluorescent guide RNA (fgR-NA) which fluoresces in the presence of small molecules (e.g., DFHBI-IT) are described and illustrated in the present provisional application.
-
公开(公告)号：US20210154326A1
公开(公告)日：2021-05-27
申请号：US16626013
申请日：2018-06-26
IPC分类号： A61K48/00 , C12N15/113 , C12N15/86 , C12N9/22
摘要： Aspects of the disclosure relate to synthetic regulatory systems comprising a multifunctional Cas nuclease and at least two guide RNAs (gRNAs) including a truncated gRNA and an multilayered regulatory control element. The synthetic regulatory system modulates endogenous gene expression, including transcriptional repression and transcriptional activation of one or more endogenous genes of a mammalian inner ear cell with multiple safety switches. Also provided herein are methods for modulating hearing sensitivity in damaged cells of the organ of corti.
-
公开(公告)号：US20200377871A1
公开(公告)日：2020-12-03
申请号：US16632782
申请日：2018-04-27
发明人： Radwa Ewaisha , Samira Kiani , Shayesteh Roshdi Ferdosi , Karen Anderson , Farzaneh Moghadam , Sri Krishna , Mo Reza Ebrahimkhani
IPC分类号： C12N9/22 , G01N33/573
摘要： Provided herein are methods and compositions for reducing an undesirable T cell immune response in human patients prior to and/or during gene therapy using CRISPR/Cas9-based genetic modulation.
-
公开(公告)号：US12084691B2
公开(公告)日：2024-09-10
申请号：US17546810
申请日：2021-12-09
发明人： Radwa Ewaisha , Samira Kiani , Shayesteh Roshdi Ferdosi , Karen Anderson , Farzaneh Moghadam , Sri Krishna , Mo Reza Ebrahimkhani
IPC分类号： C12N9/22 , A61K48/00 , G01N33/573
CPC分类号： C12N9/22 , G01N33/573 , A61K48/00 , C12N2310/20 , C12N2800/80 , G01N2333/922
摘要： Provided herein are methods and compositions for reducing an undesirable T cell immune response in human patients prior to and/or during gene therapy using CRISPR/Cas9-based genetic modulation.
-
公开(公告)号：US12077771B2
公开(公告)日：2024-09-03
申请号：US18327373
申请日：2023-06-01
CPC分类号： C12N15/85 , C12N9/22 , C12N15/11 , C12N15/115 , C12N15/62 , C12N15/907 , C12N2310/128 , C12N2310/16 , C12N2310/20 , C12N2501/12 , C12N2501/405 , C12N2800/80
摘要： Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.
-
-
-
-
-
-
-
-
-
搜索结果
13 条记录 (耗时 0.025 秒)
