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MX2019002699A - Terapia genica para pacientes con anemia de fanconi. - Google Patents

Terapia genica para pacientes con anemia de fanconi.

Info

Publication number
MX2019002699A
MX2019002699A MX2019002699A MX2019002699A MX2019002699A MX 2019002699 A MX2019002699 A MX 2019002699A MX 2019002699 A MX2019002699 A MX 2019002699A MX 2019002699 A MX2019002699 A MX 2019002699A MX 2019002699 A MX2019002699 A MX 2019002699A
Authority
MX
Mexico
Prior art keywords
gene therapy
fanconi
anemia
patients
methods
Prior art date
Application number
MX2019002699A
Other languages
English (en)
Inventor
Navarro Susana
A Bueren Juan
Sevilla Julian
Carlos Segovia Jose
Antonio Casado Jose
Guenechea Guillermo
RÍO Paula
González África
Original Assignee
Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P filed Critical Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
Publication of MX2019002699A publication Critical patent/MX2019002699A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem 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
    • 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
    • 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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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C—CHEMISTRY; METALLURGY
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1217—Phosphotransferases with a carboxyl group as acceptor (2.7.2)
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
    • C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
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    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10141—Use of virus, viral particle or viral elements as a vector
    • C12N2730/10143—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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    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10171—Demonstrated in vivo effect
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    • 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
    • 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
    • C12N2740/15011—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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    • 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
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15071—Demonstrated in vivo effect
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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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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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente invención proporciona composiciones y métodos para rescatar la expresión de FANCA en células con un producto del gen FANCA disminuido o no. En particular, se describen métodos y composiciones para la terapia génica de la anemia de Fanconi.
MX2019002699A 2016-09-08 2017-09-08 Terapia genica para pacientes con anemia de fanconi. MX2019002699A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662385185P 2016-09-08 2016-09-08
US201662412028P 2016-10-24 2016-10-24
PCT/US2017/050837 WO2018049273A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Publications (1)

Publication Number Publication Date
MX2019002699A true MX2019002699A (es) 2019-12-16

Family

ID=61561668

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019002699A MX2019002699A (es) 2016-09-08 2017-09-08 Terapia genica para pacientes con anemia de fanconi.

Country Status (12)

Country Link
US (1) US20190203225A1 (es)
EP (1) EP3510162A4 (es)
JP (3) JP7197466B2 (es)
KR (2) KR102672636B1 (es)
CN (1) CN110536966A (es)
AU (3) AU2017322511B2 (es)
BR (1) BR112019004594A2 (es)
CA (1) CA3035605A1 (es)
IL (2) IL265196B2 (es)
MX (1) MX2019002699A (es)
SG (1) SG11201901718VA (es)
WO (1) WO2018049273A1 (es)

Families Citing this family (14)

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KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
CN119345398A (zh) 2017-10-16 2025-01-24 能源环境和技术研究中心O.A.,M.P. 用于递送pklr以治疗丙酮酸激酶缺乏症的慢病毒载体
KR20210003738A (ko) * 2018-04-11 2021-01-12 로켓 파마슈티컬스, 리미티드 줄기 세포 이식을 위한 조성물 및 방법
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
KR20210030965A (ko) * 2018-07-12 2021-03-18 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
CN112888777A (zh) * 2018-07-30 2021-06-01 能源环境和技术研究中心O.A., M.P. 用于造血细胞的基因修饰的方法
CN110904102A (zh) * 2018-09-18 2020-03-24 中国科学院上海生命科学研究院 一种用于重组蛋白质表达的启动子
WO2020169666A1 (en) * 2019-02-21 2020-08-27 F. Hoffmann-La Roche Ag Improved nucleic acid target enrichment and related methods
KR20220012266A (ko) * 2019-05-23 2022-02-03 스페이스크래프트 세븐, 엘엘씨 영아 악성 골화석증을 위한 유전자 요법 벡터
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
AU2021320902A1 (en) 2020-08-07 2023-04-06 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector
JP2024518770A (ja) * 2021-04-26 2024-05-02 ストーム バイオ インコーポレイテッド ファンコニ貧血のための巨核球由来細胞外小胞に関する組成物及び方法
EP4499153A2 (en) * 2022-03-25 2025-02-05 ModernaTX, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2025137373A1 (en) * 2023-12-19 2025-06-26 Cellarity, Inc. Disease detection systems and methods

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US5952190A (en) * 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
GB0024550D0 (es) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US20070048285A1 (en) 2005-08-24 2007-03-01 Christopher Baum Self-inactivating retroviral vector
EP2424887B1 (en) 2009-04-30 2015-09-30 The U.S.A. as represented by the Secretary, Department of Health and Human Services Inducible interleukin-12
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KR20200016407A (ko) 2011-05-16 2020-02-14 젠자임 코포레이션 Ｃｘｃｒ４ 길항제의 용도
EP3494997B1 (en) * 2012-07-25 2019-09-18 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
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Also Published As

Publication number Publication date
KR20240151726A (ko) 2024-10-18
JP2019533434A (ja) 2019-11-21
JP7197466B2 (ja) 2022-12-27
CN110536966A (zh) 2019-12-03
AU2021273525A1 (en) 2021-12-16
IL265196A (en) 2019-05-30
RU2019108981A3 (es) 2020-12-24
AU2017322511A1 (en) 2019-03-28
IL265196B2 (en) 2025-10-01
AU2017322511B2 (en) 2021-08-26
BR112019004594A2 (pt) 2019-07-02
JP2025043346A (ja) 2025-03-28
IL320371A (en) 2025-06-01
AU2024287211A1 (en) 2025-01-30
KR20190062426A (ko) 2019-06-05
AU2021273525B2 (en) 2024-09-26
US20190203225A1 (en) 2019-07-04
IL265196B1 (en) 2025-06-01
JP2022160505A (ja) 2022-10-19
RU2019108981A (ru) 2020-10-08
EP3510162A4 (en) 2020-02-19
CA3035605A1 (en) 2018-03-15
WO2018049273A1 (en) 2018-03-15
KR102672636B1 (ko) 2024-06-10
EP3510162A1 (en) 2019-07-17
SG11201901718VA (en) 2019-03-28

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