RU2019120663A - Генная терапия для лечения мукополисахаридоза ii типа - Google Patents

Генная терапия для лечения мукополисахаридоза ii типа Download PDF

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RU2019120663A
RU2019120663A RU2019120663A RU2019120663A RU2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A RU 2019120663 A RU2019120663 A RU 2019120663A
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Russia
Prior art keywords
polynucleotide
promoter
ltr
hiv
polypeptide
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RU2019120663A
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English (en)
Russian (ru)
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Кендрик А. ГОСС
Джеффри Б. ПАРСОНС
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Блубёрд Био, Инк.
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15034—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
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    • 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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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—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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RU2019120663A 2016-12-06 2017-12-06 Генная терапия для лечения мукополисахаридоза ii типа RU2019120663A (ru)

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)

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RU2019120663A true RU2019120663A (ru) 2021-01-11

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RU2019120663A RU2019120663A (ru) 2016-12-06 2017-12-06 Генная терапия для лечения мукополисахаридоза ii типа

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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)

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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

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* Cited by examiner, † Cited by third party
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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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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
JP2020500562A (ja) 2020-01-16
EP3562937A4 (en) 2020-09-16
AU2017370673A1 (en) 2019-06-27

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