JP6509884B2 - 鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン - Google Patents

鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン Download PDF

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JP6509884B2
JP6509884B2 JP2016549495A JP2016549495A JP6509884B2 JP 6509884 B2 JP6509884 B2 JP 6509884B2 JP 2016549495 A JP2016549495 A JP 2016549495A JP 2016549495 A JP2016549495 A JP 2016549495A JP 6509884 B2 JP6509884 B2 JP 6509884B2
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globin
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プナム マーリク
プナム マーリク
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Cincinnati Children's Hospital Medical Center
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
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    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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  • Chemical & Material Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Virology (AREA)
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  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2016549495A 2014-01-30 2015-01-30 鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン Active JP6509884B2 (ja)

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

Publications (3)

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JP2017505626A JP2017505626A (ja) 2017-02-23
JP2017505626A5 JP2017505626A5 (enExample) 2018-03-08
JP6509884B2 true JP6509884B2 (ja) 2019-05-08

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JP2016549495A Active JP6509884B2 (ja) 2014-01-30 2015-01-30 鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン

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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

Family Cites Families (14)

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Publication number Priority date Publication date Assignee Title
US6172039B1 (en) 1990-04-16 2001-01-09 Apex Bioscience, Inc. Expression of recombinant hemoglobin and hemoglobin variants in yeast
CA2080589A1 (en) * 1990-04-16 1991-10-17 Joseph De Angelo Expression of recombinant hemoglobin in yeast
CA2246005A1 (en) 1998-10-01 2000-04-01 Hsc Research And Development Limited Partnership Hybrid genes for gene therapy in erythroid cells
EP1852511B1 (en) * 1999-12-14 2010-02-17 Novartis AG Bovine immunodeficiency virus (biv) based vectors
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
CA2506798C (en) 2002-11-22 2013-01-22 Institut Clayton De La Recherche Compositions and systems for the regulation of genes
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
WO2005030999A1 (en) 2003-09-25 2005-04-07 Dana-Farber Cancer Institute, Inc Methods to detect lineage-specific cells
US7276340B1 (en) * 2004-09-22 2007-10-02 University Of Central Florida Research Foundation, Inc. Age determination from biological stains using messenger RNA profiling analysis
US20090156534A1 (en) * 2007-09-13 2009-06-18 Memorial Sloan-Kettering Cancer Center Globin lentiviral vectors for treatment of disease
WO2010127195A2 (en) 2009-05-01 2010-11-04 Curna, Inc. Antisense oligonucleotides of hemoglobins
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
AU2011377617B2 (en) * 2011-09-23 2018-03-08 Bluebird Bio, Inc. Improved gene therapy methods
US10072067B2 (en) 2014-01-30 2018-09-11 Children's Hospital Medical Center Fetal hemoglobin for genetic correction of sickle cell disease

Also Published As

Publication number Publication date
CN106456802B (zh) 2019-10-18
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
AU2015210775A1 (en) 2016-08-18
AU2020244473B2 (en) 2024-03-07

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