CN103717240A - 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体 - Google Patents

用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体 Download PDF

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CN103717240A
CN103717240A CN201280038832.1A CN201280038832A CN103717240A CN 103717240 A CN103717240 A CN 103717240A CN 201280038832 A CN201280038832 A CN 201280038832A CN 103717240 A CN103717240 A CN 103717240A
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vector
cells
ltr
promoter
cell
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玛利亚·琼恩·德纳罗
米切尔·霍华德·菲尼尔
加博尔·沃莱斯
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Bluebird Bio Inc
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CN201280038832.1A 2011-06-10 2012-06-08 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体 Pending CN103717240A (zh)

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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CN103717240A true CN103717240A (zh) 2014-04-09

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US (3) US8858928B2 (enExample)
EP (2) EP2717922B1 (enExample)
JP (2) JP6143231B2 (enExample)
KR (2) KR20150029756A (enExample)
CN (1) CN103717240A (enExample)
AU (1) AU2012267512B2 (enExample)
CA (1) CA2838749A1 (enExample)
DK (1) DK2717922T3 (enExample)
ES (1) ES2710452T3 (enExample)
MX (1) MX2013014544A (enExample)
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PT (1) PT2717922T (enExample)
RU (1) RU2014100160A (enExample)
TR (1) TR201819015T4 (enExample)
WO (1) WO2012170911A2 (enExample)
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CN104857014A (zh) * 2015-04-21 2015-08-26 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
CN108456697A (zh) * 2018-03-29 2018-08-28 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
CN108472264A (zh) * 2015-11-05 2018-08-31 通用医疗公司 编码用于治疗肾上腺脊髓神经病的abcd1的核酸序列的鞘内递送
CN109475619A (zh) * 2016-06-13 2019-03-15 蓝鸟生物公司 神经元蜡样脂褐质沉积症的基因治疗
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
CN110214182A (zh) * 2016-12-06 2019-09-06 蓝鸟生物公司 用于i型黏多糖贮积症的基因治疗
CN110520535A (zh) * 2017-04-18 2019-11-29 葛兰素史克知识产权开发有限公司 用于逆转录病毒产生的稳定细胞系
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
CN112955174A (zh) * 2018-07-09 2021-06-11 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
CN113613633A (zh) * 2018-11-14 2021-11-05 旗舰先锋创新V股份有限公司 用于cns递送的融合剂脂质体组合物
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

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AU2013352156B2 (en) 2012-11-27 2018-12-06 Children's Medical Center Corporation Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
GB201318804D0 (en) * 2013-10-24 2013-12-11 Adaptimmune Ltd Vectors for transgene expression
IL296691B2 (en) * 2014-04-25 2023-11-01 2Seventy Bio Inc MND promoter chimeric antigen receptors
EP3738597A1 (en) 2014-04-25 2020-11-18 Bluebird Bio, Inc. Improved methods for manufacturing adoptive cell therapies
ES2744186T3 (es) 2014-04-25 2020-02-24 Childrens Medical Center Composiciones y métodos para tratar las hemoglobinopatías
CN106793780B (zh) 2014-06-06 2020-05-26 蓝鸟生物公司 改善的t细胞组合物
MX2017001079A (es) 2014-07-24 2017-09-12 Bluebird Bio Inc Receptores de antígeno quiméricos de antígeno de maduración de células b (bcma).
EP3971210A1 (en) * 2014-12-12 2022-03-23 2seventy bio, Inc. Composition for use in the treatment of b cell related conditions in a subject in need thereof comprisine an effector cell comrising a bcma chimeric antigen receptors
EP3270960A4 (en) * 2015-03-20 2018-08-08 Bluebird Bio, Inc. Vector formulations
ES2835861T5 (en) 2015-05-08 2025-02-18 Childrens Medical Ct Corp 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
WO2017099712A1 (en) 2015-12-07 2017-06-15 Bluebird Bio, Inc. Improved t cell compositions
RU2021103425A (ru) 2016-02-12 2021-02-25 Блубёрд Био, Инк. Композиции, повышающие число копий вектора (чкв), и способы их применения
EP3413896B1 (en) 2016-02-12 2021-03-17 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same
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