CN106456802B - 一种改进的用于镰状细胞疾病基因校正的胎儿血红蛋白 - Google Patents

一种改进的用于镰状细胞疾病基因校正的胎儿血红蛋白 Download PDF

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CN106456802B
CN106456802B CN201580018389.5A CN201580018389A CN106456802B CN 106456802 B CN106456802 B CN 106456802B CN 201580018389 A CN201580018389 A CN 201580018389A CN 106456802 B CN106456802 B CN 106456802B
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CN106456802A (zh
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P·马利克
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Cincinnati Children's Hospital Medical Center
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    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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CN201580018389.5A 2014-01-30 2015-01-30 一种改进的用于镰状细胞疾病基因校正的胎儿血红蛋白 Active CN106456802B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461933788P 2014-01-30 2014-01-30
US61/933,788 2014-01-30
PCT/US2015/013960 WO2015117027A1 (en) 2014-01-30 2015-01-30 An improved fetal hemoglobin for genetic correction of sickle cell disease

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CN106456802A CN106456802A (zh) 2017-02-22
CN106456802B true CN106456802B (zh) 2019-10-18

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US (2) US10072067B2 (enExample)
EP (1) EP3099334B1 (enExample)
JP (1) JP6509884B2 (enExample)
CN (1) CN106456802B (enExample)
AU (2) AU2015210775B2 (enExample)
CA (1) CA2938419C (enExample)
ES (1) ES2747817T3 (enExample)
WO (1) WO2015117027A1 (enExample)

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US10072067B2 (en) 2014-01-30 2018-09-11 Children's Hospital Medical Center Fetal hemoglobin for genetic correction of sickle cell disease
BR112017004349A2 (pt) * 2014-09-04 2017-12-05 Memorial Sloan Kettering Cancer Center terapia com gene de globina para o tratamento de hemoglobinopatias
DK3368052T3 (da) 2015-10-27 2022-03-14 Childrens Hospital Med Ct Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation
CN106497976A (zh) * 2016-10-31 2017-03-15 广东铱科基因科技有限公司 一种用于矫正重症β‑地中海贫血患者自体造血干细胞的HBB基因试剂盒
CA3070242A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
US11970707B2 (en) 2017-09-18 2024-04-30 Children's Hospital Medical Center Strong insulator and uses thereof in gene delivery
CN109722414A (zh) * 2017-10-27 2019-05-07 博雅辑因（北京）生物科技有限公司 一种高效制备成熟红细胞的方法以及用于制备成熟红细胞的培养基
JP7470119B2 (ja) * 2018-08-24 2024-04-17 シーエスエル ベーリング ジーン セラピー インコーポレイテッド 無血清培地におけるベクター産生
WO2020091797A1 (en) * 2018-11-01 2020-05-07 Children's Hospital Medical Center Reduced intensity conditioning with melphalan
WO2020209934A1 (en) 2019-04-11 2020-10-15 Massachusetts Institute Of Technology Novel chimeric antigen receptors and libraries
US20220243224A1 (en) * 2019-04-15 2022-08-04 Children's Hospital Medical Center Viral vector manufacturing methods
CN114127305A (zh) 2019-05-23 2022-03-01 麻省理工学院 经由逆转录病毒表面展示的配体发现和基因递送
EP3790545A4 (en) 2019-07-17 2021-07-28 Emmaus Medical, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF SICKLE CLEANEMIA AND THALASSEMIA
CA3212006A1 (en) 2021-02-26 2022-09-01 Kelonia Therapeutics, Inc. Lymphocyte targeted lentiviral vectors
WO2024050450A1 (en) 2022-08-31 2024-03-07 Gigamune, Inc. Engineered enveloped vectors and methods of use thereof
EP4784626A1 (en) 2023-09-25 2026-08-05 Kelonia Therapeutics, Inc. Antigen binding polypeptides
IL327310A (en) 2023-09-25 2026-05-01 Kelonia Therapeutics Inc Cancer treatment vehicles
WO2025151744A1 (en) * 2024-01-10 2025-07-17 University Of Tennessee Research Foundation Improved globin and other lentiviral vectors for gene therapy

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US5827693A (en) * 1990-04-16 1998-10-27 Apex Bioscience, Inc. Expression of recombinant hemoglobin and hemoglobin variants in yeast

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JP2004537539A (ja) * 2001-06-29 2004-12-16 スローン − ケッタリング インスティチュート フォー キャンサー リサーチ ヒトグロビン遺伝子をコードするベクターとその異常ヘモグロビン血症の治療におけるその使用
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WO2004083383A2 (en) * 2003-03-14 2004-09-30 Albert Einstein College Of Medicine Of Yeshiva University, A Division Of Yeshiva University Globin variant gene methods and compositions
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WO2015117027A1 (en) 2015-08-06
CN106456802A (zh) 2017-02-22
EP3099334B1 (en) 2019-07-31
US20170145077A1 (en) 2017-05-25
ES2747817T3 (es) 2020-03-11
US10072067B2 (en) 2018-09-11
CA2938419A1 (en) 2015-08-06
US20180346552A1 (en) 2018-12-06
JP2017505626A (ja) 2017-02-23
EP3099334A1 (en) 2016-12-07
US11345740B2 (en) 2022-05-31
AU2020244473A1 (en) 2020-10-29
CA2938419C (en) 2024-07-02
EP3099334A4 (en) 2017-06-21
AU2015210775B2 (en) 2020-07-02
JP6509884B2 (ja) 2019-05-08
AU2015210775A1 (en) 2016-08-18
AU2020244473B2 (en) 2024-03-07

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