CN108138159A - 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 - Google Patents

经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 Download PDF

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Publication number
CN108138159A
CN108138159A CN201680043108.6A CN201680043108A CN108138159A CN 108138159 A CN108138159 A CN 108138159A CN 201680043108 A CN201680043108 A CN 201680043108A CN 108138159 A CN108138159 A CN 108138159A
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sequence
aav
nucleic acid
encoding human
gly
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凯瑟琳·A·海
泽维尔·安谷拉
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Childrens Hospital of Philadelphia CHOP
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Childrens Hospital of Philadelphia CHOP
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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CN201680043108.6A 2015-06-23 2016-06-23 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 Pending CN108138159A (zh)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201562183599P 2015-06-23 2015-06-23
US62/183,599 2015-06-23
US201662315453P 2016-03-30 2016-03-30
US62/315,453 2016-03-30
US201662338315P 2016-05-18 2016-05-18
US62/338,315 2016-05-18
US201662348781P 2016-06-10 2016-06-10
US62/348,781 2016-06-10
US201662349572P 2016-06-13 2016-06-13
US62/349,572 2016-06-13
PCT/US2016/039075 WO2016210170A1 (en) 2015-06-23 2016-06-23 Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues

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CN108138159A true CN108138159A (zh) 2018-06-08

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CN (1) CN108138159A (https=)
AU (2) AU2016282781A1 (https=)
CA (1) CA2990193A1 (https=)
CO (1) CO2018000134A2 (https=)
DK (1) DK3313991T3 (https=)
ES (1) ES2989774T3 (https=)
FI (2) FI3313991T3 (https=)
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HR (1) HRP20241180T1 (https=)
HU (2) HUE068603T2 (https=)
IL (2) IL256517B2 (https=)
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CN110205297A (zh) * 2019-05-15 2019-09-06 华北理工大学 一种重组脂肪干细胞及其重组方法
CN111647625A (zh) * 2019-12-25 2020-09-11 深圳三智医学科技有限公司 一种提高人凝血因子ix表达水平的方法
CN112739818A (zh) * 2018-08-20 2021-04-30 Ucl商业有限责任公司 编码因子ix的核苷酸
CN112955174A (zh) * 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CN113817759A (zh) * 2020-07-10 2021-12-21 南京吉迈生物技术有限公司 修饰的因子ix、组合物、方法及其在基因治疗中的应用
CN114302968A (zh) * 2019-08-29 2022-04-08 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
CN115461465A (zh) * 2020-04-07 2022-12-09 密苏里大学管理机构 用于AAV基因治疗的无CpG的ITR
WO2022268110A1 (zh) * 2021-06-23 2022-12-29 英斯培瑞有限公司 用于治疗b型血友病的组合物和方法
WO2023202469A1 (zh) * 2022-04-19 2023-10-26 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途

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