-
公开(公告)号：US20240156989A1
公开(公告)日：2024-05-16
申请号：US18305863
申请日：2023-04-24
CPC分类号： A61K48/0066 , A61K48/0091 , A61P21/00 , C07K14/4708 , C12N5/0696 , C12N15/113 , C12N15/907 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , C12N15/861 , C12N2310/20
摘要： The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.
-
公开(公告)号：US11779653B2
公开(公告)日：2023-10-10
申请号：US16648204
申请日：2018-09-27
发明人： Huan Meng , Melissa J. Spencer , April D. Pyle , Courtney S. Young , Xiangsheng Liu , Ying Ji , Michael Reza Emami
CPC分类号： A61K47/6951 , A61K47/60 , A61K48/0008 , A61P35/00 , C08B37/0015 , C08G83/007
摘要： In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.
-
公开(公告)号：US20190038776A1
公开(公告)日：2019-02-07
申请号：US16076616
申请日：2017-02-09
摘要： The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.
-
公开(公告)号：US20240299586A1
公开(公告)日：2024-09-12
申请号：US18289219
申请日：2022-05-05
CPC分类号： A61K48/0058 , C12N9/22 , C12N15/111 , C12N15/86 , C12N2310/20 , C12N2320/32 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2750/14152
摘要： In various embodiments, AAV serotypes capable of transducing skeletal muscle fibers and skeletal muscle stem cells (MuSC) with reduced off target tissue tropism are provided.
-
公开(公告)号：US11666666B2
公开(公告)日：2023-06-06
申请号：US16076616
申请日：2017-02-09
IPC分类号： A61K48/00 , C07K14/47 , C12N15/90 , C12N15/113 , A61P21/00 , C12N5/074 , C12N15/861
CPC分类号： A61K48/0066 , A61K48/0091 , A61P21/00 , C07K14/4708 , C12N5/0696 , C12N15/113 , C12N15/907 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , C12N15/861 , C12N2310/20
摘要： The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.
-
公开(公告)号：US20220177876A1
公开(公告)日：2022-06-09
申请号：US17441888
申请日：2020-04-07
摘要： Disclosed herein are guide sequences for modifying a dystrophin gene using CRISPR technology. Specifically, the disclosure provides a method of modifying a dystrophin gene in a cell or a subject, which comprises introducing into the cell or subject (a) a Cas protein or a nucleotide sequence encoding the Cas protein; and a single guide RNA (gRNA), or a first gRNA and a second gRNA, wherein the Cas protein is a type II CRISPR/Cas endonuclease. Further disclosed are gRNA nucleic acid sequences.
-
公开(公告)号：US20200376139A1
公开(公告)日：2020-12-03
申请号：US16648204
申请日：2018-09-27
发明人： Huan Meng , Melissa J. Spencer , April D. Pyle , Courtney S. Young , Xiangsheng Liu , Ying Ji , Michael Reza Emami
摘要： In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.
-
-
-
-
-
-
搜索结果
7 条记录 (耗时 0.026 秒)
