KR20190088554A - Ii형 점액다당류증에 대한 유전자 요법 - Google Patents

Ii형 점액다당류증에 대한 유전자 요법 Download PDF

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KR20190088554A
KR20190088554A KR1020197019345A KR20197019345A KR20190088554A KR 20190088554 A KR20190088554 A KR 20190088554A KR 1020197019345 A KR1020197019345 A KR 1020197019345A KR 20197019345 A KR20197019345 A KR 20197019345A KR 20190088554 A KR20190088554 A KR 20190088554A
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promoter
cell
cells
polynucleotide
virus
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Korean (ko)
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켄드릭 에이. 고스
제프리 비. 파슨스
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블루버드 바이오, 인코포레이티드.
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    • C12N2740/10011—Retroviridae
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    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
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KR1020197019345A 2016-12-06 2017-12-06 Ii형 점액다당류증에 대한 유전자 요법 Ceased KR20190088554A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662430819P 2016-12-06 2016-12-06
US62/430,819 2016-12-06
PCT/US2017/064940 WO2018106821A1 (en) 2016-12-06 2017-12-06 Gene therapy for mucopolysaccharidosis, type ii

Publications (1)

Publication Number Publication Date
KR20190088554A true KR20190088554A (ko) 2019-07-26

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KR1020197019345A Ceased KR20190088554A (ko) 2016-12-06 2017-12-06 Ii형 점액다당류증에 대한 유전자 요법

Country Status (13)

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US (1) US20200071721A1 (enExample)
EP (1) EP3562937A4 (enExample)
JP (1) JP2020500562A (enExample)
KR (1) KR20190088554A (enExample)
CN (1) CN110199028A (enExample)
AU (1) AU2017370673A1 (enExample)
BR (1) BR112019011590A2 (enExample)
CA (1) CA3046080A1 (enExample)
IL (1) IL267060A (enExample)
MA (1) MA47173A (enExample)
MX (1) MX2019006551A (enExample)
RU (1) RU2019120663A (enExample)
WO (1) WO2018106821A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
WO2022212616A1 (en) * 2021-04-01 2022-10-06 The University Of North Carolina At Chapel Hill Aav-ids vectors for treatment of mucopolysaccharidosis ii
WO2024233287A1 (en) * 2023-05-05 2024-11-14 The Regents Of The University Of California Viral vectors for cell therapy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2872170B1 (fr) * 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
US20110294114A1 (en) * 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
DK2717922T3 (en) * 2011-06-10 2019-01-07 Bluebird Bio Inc GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE
EP2855684A1 (en) * 2012-05-25 2015-04-08 Commissariat à l'Énergie Atomique et aux Énergies Alternatives Vector for the selective silencing of a gene in astrocytes
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production

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Publication number Publication date
MA47173A (fr) 2019-11-06
WO2018106821A1 (en) 2018-06-14
MX2019006551A (es) 2019-10-15
EP3562937A1 (en) 2019-11-06
CN110199028A (zh) 2019-09-03
IL267060A (en) 2019-08-29
BR112019011590A2 (pt) 2019-10-22
US20200071721A1 (en) 2020-03-05
CA3046080A1 (en) 2018-06-14
JP2020500562A (ja) 2020-01-16
EP3562937A4 (en) 2020-09-16
AU2017370673A1 (en) 2019-06-27
RU2019120663A (ru) 2021-01-11

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