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
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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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

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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
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
MX2021001292A (es) * 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Metodos para la modificacion genica de celulas hematopoyeticas.
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
AU2012267512B2 (en) * 2011-06-10 2014-11-20 Bluebird Bio, Inc. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
US9695443B2 (en) * 2012-05-25 2017-07-04 Commissariat A L'energie Atomique Et Aux Energies 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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WO2018106821A1 (en) 2018-06-14
RU2019120663A (ru) 2021-01-11
US20200071721A1 (en) 2020-03-05
BR112019011590A2 (pt) 2019-10-22
JP2020500562A (ja) 2020-01-16
CN110199028A (zh) 2019-09-03
MX2019006551A (es) 2019-10-15
EP3562937A4 (en) 2020-09-16
CA3046080A1 (en) 2018-06-14
AU2017370673A1 (en) 2019-06-27
EP3562937A1 (en) 2019-11-06
MA47173A (fr) 2019-11-06
IL267060A (en) 2019-08-29

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