BR0211307A - Lentivetor recombinante codificando o gene da globina humana, uso do mesmo no tratamento de hemoglobinopatias, células de mamìfero oriundas do progenitor hematopoietico e método para preparo de uma composição terapêutica para tratamento de hemoglobinopatia em um mamìfero - Google Patents

Lentivetor recombinante codificando o gene da globina humana, uso do mesmo no tratamento de hemoglobinopatias, células de mamìfero oriundas do progenitor hematopoietico e método para preparo de uma composição terapêutica para tratamento de hemoglobinopatia em um mamìfero

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Publication number
BR0211307A
BR0211307A BR0211307-4A BR0211307A BR0211307A BR 0211307 A BR0211307 A BR 0211307A BR 0211307 A BR0211307 A BR 0211307A BR 0211307 A BR0211307 A BR 0211307A
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BR
Brazil
Prior art keywords
treatment
hemoglobinopathies
mammal
hematopoietic progenitor
globin gene
Prior art date
Application number
BR0211307-4A
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English (en)
Inventor
Michel Sadelain
Chad May
Joseph Bertino
Stefano Rivella
Original Assignee
Sloan Kettering Inst Cancer
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Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=26972630&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BR0211307(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Sloan Kettering Inst Cancer filed Critical Sloan Kettering Inst Cancer
Publication of BR0211307A publication Critical patent/BR0211307A/pt

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

"LENTIVETOR RECOMBINANTE CODIFICANDO O GENE DA GLOBINA HUMANA, USO DO MESMO NO TRATAMENTO DE HEMOGLOBINOPATIAS, CéLULAS DE MAMìFERO ORIUNDAS DO PROGENITOR, HEMATOPOIETICO E MéTODO PARA PREPARO DE UMA COMPOSIçãO TERAPêUTICA PARA TRATAMENTO DE HEMOGLOBINOPATIA EM UM MAMìFERO". Vetores lentivirais recombinantes apresentando uma região codificando um gene de globina funcional; e grandes partes das regiões de controle de lócus da beta-globulina que incluem Sítios hipersensíveis a Dnase I HS2, HS3 e HS4 proporciona a expressão de beta-globina quando introduzidos em um mamífero, por exemplo um ser humano, in vivo. Opcionalmente, o vetor inclui adicionalmente uma região codificando uma dihidrofolato redutase. O vetor pode ser utilizado no tratamento de hemoglobinopatias, incluindo beta-talessemia e anemia falciforme. Por exemplo, células do progenitor hematopoietico ou células tronco podem ser transformadas ex vivo e então restituídas ao paciente. Os processos de seleção podem ser usados para aumentar o percentual de células transformadas na população restituída. Por exemplo, um marcador de seleção que torna as células transformadas mais resistentes às drogas do que células não-transformadas permite a seleção através do tratamento das células com a droga correspondente.
BR0211307-4A 2001-06-29 2002-07-01 Lentivetor recombinante codificando o gene da globina humana, uso do mesmo no tratamento de hemoglobinopatias, células de mamìfero oriundas do progenitor hematopoietico e método para preparo de uma composição terapêutica para tratamento de hemoglobinopatia em um mamìfero BR0211307A (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US30186101P 2001-06-29 2001-06-29
US30285201P 2001-07-02 2001-07-02
PCT/US2002/020988 WO2003002155A1 (en) 2001-06-29 2002-07-01 Vector encoding human globlin gene and use thereof in treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
BR0211307A true BR0211307A (pt) 2004-09-14

Family

ID=26972630

Family Applications (1)

Application Number Title Priority Date Filing Date
BR0211307-4A BR0211307A (pt) 2001-06-29 2002-07-01 Lentivetor recombinante codificando o gene da globina humana, uso do mesmo no tratamento de hemoglobinopatias, células de mamìfero oriundas do progenitor hematopoietico e método para preparo de uma composição terapêutica para tratamento de hemoglobinopatia em um mamìfero

Country Status (10)

Country Link
US (2) US7541179B2 (pt)
EP (2) EP2359868B1 (pt)
JP (1) JP2004537539A (pt)
CN (1) CN1522160A (pt)
AT (1) ATE535260T1 (pt)
BR (1) BR0211307A (pt)
CA (1) CA2451173C (pt)
ES (1) ES2582028T3 (pt)
IL (2) IL159371A0 (pt)
WO (1) WO2003002155A1 (pt)

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CN101348786B (zh) * 2007-07-20 2011-05-11 谭孟群 一种人β珠蛋白基因及其重组腺相关病毒载体
US20090156534A1 (en) * 2007-09-13 2009-06-18 Memorial Sloan-Kettering Cancer Center Globin lentiviral vectors for treatment of disease
GB0812191D0 (en) * 2008-07-03 2008-08-13 Greater Glasgow Health Board Modified cells and methods of monitoring their viability
CN101475936B (zh) * 2008-10-17 2011-04-06 滨州医学院 一种edrf基因片段
ITRM20100418A1 (it) * 2010-07-28 2012-01-29 Santa Anna Acuto Vettore lentivirale ricombinante per la beta-globina e suoi usi in campo medico.
KR101958612B1 (ko) * 2011-09-23 2019-03-14 블루버드 바이오, 인코포레이티드. 개선된 유전자 치료 방법
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US20160039910A1 (en) * 2013-03-15 2016-02-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Compositions and methods for the treatment of carboxyhemoglobinemia
US10072067B2 (en) * 2014-01-30 2018-09-11 Children's Hospital Medical Center Fetal hemoglobin for genetic correction of sickle cell disease
KR102500531B1 (ko) 2014-09-04 2023-02-17 메모리얼 슬로안 케터링 캔서 센터 헤모글로빈병증 치료용 글로빈 유전자 치료법
CA2977785A1 (en) * 2015-01-21 2016-07-28 Cornell University Viral vectors for prophylaxis and therapy of hemoglobinopathies
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CN106978443B (zh) * 2017-05-04 2020-01-14 济南赛尔生物科技股份有限公司 一种β-珠蛋白重组慢病毒载体及其应用
MX2020000676A (es) 2017-07-18 2021-05-14 Csl Behring Gene Therapy Inc Composiciones y metodos para tratar beta-hemoglobinopatias.
WO2019113321A1 (en) 2017-12-06 2019-06-13 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
EP3511412A1 (en) * 2018-01-12 2019-07-17 Genethon Genetically engineered hematopoietic stem cell as a platform for systemic protein expression
EP3788070A4 (en) * 2018-04-30 2022-03-09 The Children's Hospital Of Philadelphia COMPOSITIONS AND METHODS OF HEMOGLOBIN PRODUCTION
WO2020056400A1 (en) * 2018-09-14 2020-03-19 The Children's Hospital Of Philadelphia Compositions and methods for hemoglobin production
EP3924376A4 (en) * 2019-02-14 2022-12-14 The Regents Of The University Of California OPTIMIZED LENTIVIRAL VECTOR COMPRISING MINIMUM ENABLING SEQUENCE ELEMENTS FOR HEMOGLOBINOPATHY STEM CELL GENE THERAPY
CN110699381A (zh) * 2019-09-17 2020-01-17 合肥瑞灵生物科技有限公司 地中海贫血病基因治疗载体构建方法及其用途
CN114480293B (zh) * 2022-04-15 2022-07-12 山东兴瑞生物科技有限公司 一种hbb融合基因修饰的自体造血干细胞、制备方法及其应用
WO2024006388A1 (en) * 2022-06-29 2024-01-04 The Regents Of The University Of California Lentiviral vectors expressing alpha-glob in genes for gene therapy of alpha thalassemia

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Also Published As

Publication number Publication date
ATE535260T1 (de) 2011-12-15
US20030022303A1 (en) 2003-01-30
US7541179B2 (en) 2009-06-02
EP1409025A1 (en) 2004-04-21
IL159371A0 (en) 2004-06-01
IL159371A (en) 2010-05-31
WO2003002155B1 (en) 2004-05-27
US8058061B2 (en) 2011-11-15
EP1409025B1 (en) 2011-11-30
EP2359868B1 (en) 2016-05-04
ES2582028T3 (es) 2016-09-08
EP2359868A1 (en) 2011-08-24
CN1522160A (zh) 2004-08-18
CA2451173A1 (en) 2003-01-09
WO2003002155A1 (en) 2003-01-09
JP2004537539A (ja) 2004-12-16
US20090274671A1 (en) 2009-11-05
CA2451173C (en) 2012-12-18
EP1409025A4 (en) 2005-10-19

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