JP2020500562A - ムコ多糖症ｉｉ型のための遺伝子治療 - Google Patents

ムコ多糖症ｉｉ型のための遺伝子治療 Download PDF

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JP2020500562A
JP2020500562A JP2019551257A JP2019551257A JP2020500562A JP 2020500562 A JP2020500562 A JP 2020500562A JP 2019551257 A JP2019551257 A JP 2019551257A JP 2019551257 A JP2019551257 A JP 2019551257A JP 2020500562 A JP2020500562 A JP 2020500562A
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cells
promoter
polynucleotide
cell
virus
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JP2020500562A5 (enExample
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ケンドリック エー． ゴス，
ケンドリック エー． ゴス，
ジェフリー ビー． パーソンズ，
ジェフリー ビー． パーソンズ，
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ブルーバード バイオ， インコーポレイテッド
ブルーバード バイオ， インコーポレイテッド
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    • C12N2740/00—Reverse transcribing RNA viruses
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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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    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)

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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

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

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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)

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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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2014519335A (ja) * 2011-06-10 2014-08-14 ブルーバード バイオ， インコーポレイテッド 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター
US20160122727A1 (en) * 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production

Family Cites Families (2)

* 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
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2014519335A (ja) * 2011-06-10 2014-08-14 ブルーバード バイオ， インコーポレイテッド 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター
US20160122727A1 (en) * 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HUMAN GENE THERAPY, vol. 26(6), JPN6021037921, 2015, pages 357 - 366, ISSN: 0004751977 *
MOL THER., vol. 11(5), JPN6021037917, May 2005 (2005-05-01), pages 776 - 789, ISSN: 0004751978 *

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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
KR20190088554A (ko) 2019-07-26
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
EP3562937A4 (en) 2020-09-16
AU2017370673A1 (en) 2019-06-27
RU2019120663A (ru) 2021-01-11

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