KR20150029756A - 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터 - Google Patents

부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터 Download PDF

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KR20150029756A
KR20150029756A KR1020157004059A KR20157004059A KR20150029756A KR 20150029756 A KR20150029756 A KR 20150029756A KR 1020157004059 A KR1020157004059 A KR 1020157004059A KR 20157004059 A KR20157004059 A KR 20157004059A KR 20150029756 A KR20150029756 A KR 20150029756A
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vector
cells
ltr
cell
promoter
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마리아 조안 디나로
미첼 하워드 화이너
가보 버리스
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블루버드 바이오, 인코포레이티드.
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KR1020157004059A 2011-06-10 2012-06-08 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터 Withdrawn KR20150029756A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161495857P 2011-06-10 2011-06-10
US61/495,857 2011-06-10
PCT/US2012/041693 WO2012170911A2 (en) 2011-06-10 2012-06-08 Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

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KR1020147000344A Division KR101528440B1 (ko) 2011-06-10 2012-06-08 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터

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KR20150029756A true KR20150029756A (ko) 2015-03-18

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KR1020157004059A Withdrawn KR20150029756A (ko) 2011-06-10 2012-06-08 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터
KR1020147000344A Expired - Fee Related KR101528440B1 (ko) 2011-06-10 2012-06-08 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터

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US (3) US8858928B2 (cg-RX-API-DMAC7.html)
EP (2) EP3492108A1 (cg-RX-API-DMAC7.html)
JP (2) JP6143231B2 (cg-RX-API-DMAC7.html)
KR (2) KR20150029756A (cg-RX-API-DMAC7.html)
CN (1) CN103717240A (cg-RX-API-DMAC7.html)
AU (1) AU2012267512B2 (cg-RX-API-DMAC7.html)
CA (1) CA2838749A1 (cg-RX-API-DMAC7.html)
DK (1) DK2717922T3 (cg-RX-API-DMAC7.html)
ES (1) ES2710452T3 (cg-RX-API-DMAC7.html)
MX (1) MX2013014544A (cg-RX-API-DMAC7.html)
PL (1) PL2717922T3 (cg-RX-API-DMAC7.html)
PT (1) PT2717922T (cg-RX-API-DMAC7.html)
RU (1) RU2014100160A (cg-RX-API-DMAC7.html)
TR (1) TR201819015T4 (cg-RX-API-DMAC7.html)
WO (1) WO2012170911A2 (cg-RX-API-DMAC7.html)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200087573A (ko) * 2019-01-11 2020-07-21 동의대학교 산학협력단 해죽순 추출물을 포함하는 신경병증성 통증 예방 및 치료용 조성물

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2717922T3 (en) 2011-06-10 2019-01-07 Bluebird Bio Inc GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE
ES2708948T3 (es) 2012-11-27 2019-04-12 Childrens Medical Ct Corp Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal
GB201318804D0 (en) † 2013-10-24 2013-12-11 Adaptimmune Ltd Vectors for transgene expression
CN106794260B (zh) 2014-04-25 2021-02-23 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
WO2015164759A2 (en) * 2014-04-25 2015-10-29 Bluebird Bio, Inc. Mnd promoter chimeric antigen receptors
CN106456670B (zh) 2014-04-25 2020-01-07 蓝鸟生物公司 制备过继性细胞疗法的改善方法
RS61406B1 (sr) 2014-06-06 2021-03-31 Bluebird Bio Inc Poboljšane kompozicije t ćelija
EP3172231B1 (en) 2014-07-24 2021-05-05 Bluebird Bio, Inc. Bcma chimeric antigen receptors
US10383929B2 (en) 2014-12-12 2019-08-20 Bluebird Bio, Inc. BCMA chimeric antigen receptors
EP3270960A4 (en) * 2015-03-20 2018-08-08 Bluebird Bio, Inc. Vector formulations
CN104857014B (zh) * 2015-04-21 2017-12-08 武汉大学 2‑羟丙基‑β‑环化糊精在制备治疗X‑连锁肾上腺脑白质退化症的药物中的应用
EP3763814A1 (en) 2015-05-08 2021-01-13 The Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
IL246634B (en) * 2015-07-23 2020-08-31 Brett Buno A method for creating a high-titer hepatitis e virus stock and detecting calibration for hepatitis e virus
PL3370705T3 (pl) * 2015-11-05 2022-08-01 The General Hospital Corporation Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii
WO2017099712A1 (en) 2015-12-07 2017-06-15 Bluebird Bio, Inc. Improved t cell compositions
CN108883136A (zh) 2016-02-12 2018-11-23 蓝鸟生物公司 Vcn增强子组合物及其使用方法
KR20250134722A (ko) 2016-02-12 2025-09-11 블루버드 바이오, 인코포레이티드. Vcn 인핸서 조성물 및 이의 사용 방법
JP7215905B2 (ja) * 2016-03-18 2023-01-31 プレシゲン，インコーポレイテッド Ｖｉｉ型コラーゲン欠損症の治療のための組成物及び方法
US20220323612A1 (en) * 2016-06-13 2022-10-13 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
US20190345450A1 (en) * 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
MX2019005277A (es) 2016-11-04 2019-09-27 Bluebird Bio Inc Composiciones de celulas t con car anti-bcma.
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
CN110199028A (zh) * 2016-12-06 2019-09-03 蓝鸟生物公司 用于ii型黏多糖贮积症的基因治疗
MX2019006552A (es) * 2016-12-06 2019-10-15 Bluebird Bio Inc Terapia génica para mucopolisacaridosis de tipo i.
SG11201908844YA (en) 2017-03-29 2019-10-30 Bluebird Bio Inc Vectors and compositions for treating hemoglobinopathies
US11261441B2 (en) * 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
GB201706121D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Stable cell lines for retroviral production
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
WO2019070740A2 (en) 2017-10-02 2019-04-11 Fred Hutchinson Cancer Research Center LUTENING HORMONE RECEPTOR BINDING AGENTS AND LUTINISING HORMONE AGONISTS FOR IDENTIFICATION, MULTIPLICATION, REMOVAL AND MODIFICATION OF PRIMITIVE STEM CELL POPULATIONS
CN108456697B (zh) * 2018-03-29 2022-05-27 成都优娃生物科技有限公司 应用慢病毒载体EF1α启动子优化表达ABCD1基因治疗肾上腺脑白质营养不良症
US12522811B2 (en) 2018-05-01 2026-01-13 The Children's Medical Center Corporation Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
CN112367973A (zh) * 2018-05-15 2021-02-12 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CA3105953A1 (en) 2018-07-09 2020-01-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
MY204234A (en) 2018-08-27 2024-08-16 Regeneron Pharma Use of raman spectroscopy in downstream purification
KR20210131991A (ko) 2018-11-14 2021-11-03 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 구획-특이적 카고 전달을 위한 조성물 및 방법
EP3880179A2 (en) * 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
JP2024532963A (ja) 2021-04-08 2024-09-11 サナ バイオテクノロジー，インコーポレイテッド Ｃｄ８特異的抗体コンストラクト及びその組成物
CN115838765A (zh) * 2021-09-18 2023-03-24 济南赛尔生物科技股份有限公司 一种治疗异染性脑白质营养不良的慢病毒载体
CN114478713B (zh) * 2022-02-21 2023-03-28 江苏蒙彼利生物科技有限公司 一种cmv包膜蛋白包装慢病毒载体及其应用
CN121620592A (zh) * 2023-05-01 2026-03-06 西雅图儿童医院d/b/a西雅图儿童研究所 表达突变体白细胞介素15的免疫细胞

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5635387A (en) 1990-04-23 1997-06-03 Cellpro, Inc. Methods and device for culturing human hematopoietic cells and their precursors
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
US5460964A (en) 1992-04-03 1995-10-24 Regents Of The University Of Minnesota Method for culturing hematopoietic cells
ATE155681T1 (de) 1992-05-18 1997-08-15 Minnesota Mining & Mfg Einrichtung zur transmucosalen wirkstoffabgabe
ES2249760T3 (es) * 1993-01-26 2006-04-01 The Trustees Of The University Of Pennsylvania (A Corporation Of Pennsylvania) Composiciones y procedimientos de administracion de materias geneticas.
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5658784A (en) * 1994-04-14 1997-08-19 Dana-Farber Cancer Institute, Inc. Nucleic acid encoding transcription factor p300 and uses of p300
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
FR2765578B1 (fr) 1997-07-03 1999-09-10 Adir Procede de preparation d'un perhydroisoindole substitue
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
FR2778670B1 (fr) * 1998-05-18 2002-12-13 Rhone Poulenc Rorer Sa Methodes et compositions pour la production de particules virales
US20030039636A1 (en) 2001-05-01 2003-02-27 Genetix Pharmaceuticals, Inc. Novel self-inactivating (SIN) lentiviral vectors
WO2002088346A2 (en) 2001-05-01 2002-11-07 National Research Council Of Canada A system for inducible expression in eukaryotic cells
ES2348868T3 (es) 2002-12-13 2010-12-16 Genetix Pharmaceuticals Inc. Vectores retrovirales terapeuticos para terapia genica.
EP2573185A3 (en) * 2005-02-16 2013-06-05 Lentigen Corporation Lentiviral vectors and their use
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
EP1835032A1 (en) * 2006-03-14 2007-09-19 Université de Liège A self-inactivating recombinant lentiviral vector for the inhibition of HIV replication
WO2008134878A1 (en) * 2007-05-04 2008-11-13 University Health Network Compositions and methods for cancer treatment
EP2020444B1 (en) 2007-08-03 2017-05-31 Institut Pasteur Defective non-integrative lentiviral transfer vectors for vaccines
CN102438658A (zh) 2009-03-13 2012-05-02 莱蒂恩公司 非整合型逆转录病毒载体疫苗
DK2717922T3 (en) 2011-06-10 2019-01-07 Bluebird Bio Inc GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200087573A (ko) * 2019-01-11 2020-07-21 동의대학교 산학협력단 해죽순 추출물을 포함하는 신경병증성 통증 예방 및 치료용 조성물

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