KR20230079172A - 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트 - Google Patents

간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트 Download PDF

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KR20230079172A
KR20230079172A KR1020237014666A KR20237014666A KR20230079172A KR 20230079172 A KR20230079172 A KR 20230079172A KR 1020237014666 A KR1020237014666 A KR 1020237014666A KR 20237014666 A KR20237014666 A KR 20237014666A KR 20230079172 A KR20230079172 A KR 20230079172A
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aav
raav
pah
vector
capsid
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Korean (ko)
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시르카 알.엠. 쿄스티오-무어
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젠자임 코포레이션
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    • C12Y403/01—Ammonia-lyases (4.3.1)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Radiation-Therapy Devices (AREA)
KR1020237014666A 2020-10-01 2021-09-30 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트 Pending KR20230079172A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063086537P 2020-10-01 2020-10-01
US63/086,537 2020-10-01
US202063121797P 2020-12-04 2020-12-04
US63/121,797 2020-12-04
PCT/US2021/052913 WO2022072657A1 (en) 2020-10-01 2021-09-30 Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy

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KR20230079172A true KR20230079172A (ko) 2023-06-05

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KR1020237014666A Pending KR20230079172A (ko) 2020-10-01 2021-09-30 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트

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US (2) US12173308B2 (https=)
EP (1) EP4222251A1 (https=)
JP (1) JP2023544165A (https=)
KR (1) KR20230079172A (https=)
AU (1) AU2021355481A1 (https=)
BR (1) BR112023003929A2 (https=)
CA (1) CA3193866A1 (https=)
CO (1) CO2023003418A2 (https=)
IL (1) IL301677A (https=)
MX (1) MX2023003805A (https=)
TW (1) TWI900667B (https=)
WO (1) WO2022072657A1 (https=)

Families Citing this family (2)

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CN115896135B (zh) * 2022-11-02 2024-03-01 苏州诺洁贝生物技术有限公司 优化的pah基因和表达盒及其用途

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TWI900667B (zh) 2025-10-11
BR112023003929A2 (pt) 2023-04-11
US20250179522A1 (en) 2025-06-05
AU2021355481A1 (en) 2023-06-08
JP2023544165A (ja) 2023-10-20
AU2021355481A9 (en) 2024-07-25
MX2023003805A (es) 2023-04-12
IL301677A (en) 2023-05-01
US12173308B2 (en) 2024-12-24
TW202229559A (zh) 2022-08-01
CA3193866A1 (en) 2022-04-07
US20220112520A1 (en) 2022-04-14
WO2022072657A1 (en) 2022-04-07
EP4222251A1 (en) 2023-08-09
CO2023003418A2 (es) 2023-03-27

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