-
公开(公告)号：US11083799B2
公开(公告)日：2021-08-10
申请号：US16084531
申请日：2017-03-16
IPC分类号： A61K48/00 , C12N15/90 , C12N15/113 , C12N15/10 , A61K9/00 , C12N5/0775 , C12N5/071
摘要： Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.
-
公开(公告)号：US11866727B2
公开(公告)日：2024-01-09
申请号：US15759202
申请日：2016-11-07
CPC分类号： C12N15/907 , C12N9/16 , C12N9/22 , C12N15/11 , C12Y301/03009 , A61K38/00 , C12N2310/20 , C12N2800/80
摘要： The present application provides materials and methods for treating a patient with Glycogen Storage Disease type 1a (GSD1a) both ex vivo and in vivo. In addition, the present application provides materials and methods for modulating the expression, function, and/or activity of the glucose-6-phosphatase, catalytic (G6PC) and/or the glucose-6-phosphatase (G6Pase) protein in a cell by genome editing.
-
公开(公告)号：US11851653B2
公开(公告)日：2023-12-26
申请号：US15779836
申请日：2016-12-01
发明人： Chad Albert Cowan , Roman Lvovitch Bogorad , Jeffrey Li , Ante Sven Lundberg , Matthias Johannes John , Jeffrey William Stebbins , Thao Thi Nguyen
CPC分类号： C12N15/111 , A61K9/0019 , A61K31/7088 , C12N9/22 , C12N15/102 , A61K48/00 , C12N2310/20 , C12N2320/30 , C12N2320/32 , C12N2800/80 , C12N15/102 , C12Q2521/301
摘要： The present application provides materials and methods for treating a patient with Alpha-1 antitrypsin deficiency (AATD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing the SERPINA1 gene in a cell by genome editing.
-
公开(公告)号：US11332760B2
公开(公告)日：2022-05-17
申请号：US17178737
申请日：2021-02-18
摘要： The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA (gDNA) molecule in a cell. In some embodiments the present disclosure provides donor polynucleotides comprising two strands capable of correcting a mutation that causes Glycogen Storage Disease 1a.
-
公开(公告)号：US20190076551A1
公开(公告)日：2019-03-14
申请号：US16084531
申请日：2017-03-16
IPC分类号： A61K48/00 , A61K9/00 , C12N5/0775 , C12N5/071
摘要： Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.
-
公开(公告)号：US11827877B2
公开(公告)日：2023-11-28
申请号：US16457528
申请日：2019-06-28
CPC分类号： C12N15/907 , C12N9/22 , C12N15/11 , C12N2310/18 , C12N2310/20 , C12N2800/80
摘要： The present disclosure provides donor polynucleotides, genome editing systems, methods, pharmaceutical compositions, and kits which correct or induce a mutation that causes Glycogen Storage Disease 1a in a genomic DNA molecule in a cell. In some embodiments the present disclosure provides donor polynucleotides comprising two strands capable of correcting a mutation that causes Glycogen Storage Disease 1a.
-
公开(公告)号：US20220008558A1
公开(公告)日：2022-01-13
申请号：US17371386
申请日：2021-07-09
IPC分类号： A61K48/00 , C12N15/113 , C12N15/10 , C12N15/90 , A61K9/00 , C12N5/0775 , C12N5/071
摘要： Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) gene in a cell by genome editing.
-
-
-
-
-
-
搜索结果
7 条记录 (耗时 0.016 秒)
