JP2020500562A5 - - Google Patents

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Publication number
JP2020500562A5
JP2020500562A5 JP2019551257A JP2019551257A JP2020500562A5 JP 2020500562 A5 JP2020500562 A5 JP 2020500562A5 JP 2019551257 A JP2019551257 A JP 2019551257A JP 2019551257 A JP2019551257 A JP 2019551257A JP 2020500562 A5 JP2020500562 A5 JP 2020500562A5
Authority
JP
Japan
Prior art keywords
promoter
polynucleotide
ltr
virus
hiv
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2019551257A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020500562A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2017/064940 external-priority patent/WO2018106821A1/en
Publication of JP2020500562A publication Critical patent/JP2020500562A/ja
Publication of JP2020500562A5 publication Critical patent/JP2020500562A5/ja
Pending legal-status Critical Current

Links

JP2019551257A 2016-12-06 2017-12-06 ムコ多糖症ｉｉ型のための遺伝子治療 Pending JP2020500562A (ja)

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 (2)

Publication Number Publication Date
JP2020500562A JP2020500562A (ja) 2020-01-16
JP2020500562A5 true JP2020500562A5 (enExample) 2021-01-21

Family

ID=62492351

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019551257A Pending JP2020500562A (ja) 2016-12-06 2017-12-06 ムコ多糖症ｉｉ型のための遺伝子治療

Country Status (13)

Country Link
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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