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PL2424571T3 - Wektor genowy - Google Patents

Wektor genowy

Info

Publication number
PL2424571T3
PL2424571T3 PL10723305T PL10723305T PL2424571T3 PL 2424571 T3 PL2424571 T3 PL 2424571T3 PL 10723305 T PL10723305 T PL 10723305T PL 10723305 T PL10723305 T PL 10723305T PL 2424571 T3 PL2424571 T3 PL 2424571T3
Authority
PL
Poland
Prior art keywords
gene vector
gene
vector
Prior art date
Application number
PL10723305T
Other languages
English (en)
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
Original Assignee
Ospedale San Raffaele S.R.L
Fondazione Telethon
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 Ospedale San Raffaele S.R.L, Fondazione Telethon filed Critical Ospedale San Raffaele S.R.L
Publication of PL2424571T3 publication Critical patent/PL2424571T3/pl

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/555Interferons [IFN]
    • C07K14/56IFN-alpha
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
PL10723305T 2009-04-30 2010-04-30 Wektor genowy PL2424571T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
EP10723305.8A EP2424571B1 (en) 2009-04-30 2010-04-30 Gene vector
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (1)

Publication Number Publication Date
PL2424571T3 true PL2424571T3 (pl) 2020-10-19

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
PL10723305T PL2424571T3 (pl) 2009-04-30 2010-04-30 Wektor genowy

Country Status (22)

Country Link
US (4) US10287579B2 (pl)
EP (2) EP2424571B1 (pl)
JP (2) JP6144911B2 (pl)
KR (1) KR101793615B1 (pl)
CN (1) CN102596255B (pl)
AU (1) AU2010243276B2 (pl)
BR (1) BRPI1010873B1 (pl)
CA (1) CA2759438C (pl)
CY (1) CY1123218T1 (pl)
DK (1) DK2424571T3 (pl)
EA (1) EA023938B1 (pl)
ES (1) ES2786039T3 (pl)
HR (1) HRP20200647T1 (pl)
HU (1) HUE049845T2 (pl)
IL (1) IL215804A (pl)
LT (1) LT2424571T (pl)
MX (1) MX2011011508A (pl)
PL (1) PL2424571T3 (pl)
PT (1) PT2424571T (pl)
SG (2) SG175839A1 (pl)
SI (1) SI2424571T1 (pl)
WO (1) WO2010125471A2 (pl)

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EP2424571B1 (en) * 2009-04-30 2020-04-08 Ospedale San Raffaele S.r.l. Gene vector
LT2772265T (lt) 2010-05-14 2018-05-25 Oregon Health & Science University Rekombinantiniai žcmv ir rhcmv vektoriai ir jų panaudojimas
EP2591095B1 (en) * 2010-07-08 2016-04-27 Duke University Direct reprogramming of cells to cardiac myocyte fate
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
US20150190532A1 (en) * 2012-04-04 2015-07-09 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
KR20170134766A (ko) 2012-05-25 2017-12-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 Rna-유도된 표적 dna 변형 및 전사의 rna-유도된 조절을 위한 방법 및 조성물
CA2878037C (en) 2012-07-11 2021-08-31 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
US20160017366A1 (en) 2012-12-06 2016-01-21 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
EP3889173B1 (en) 2013-02-15 2023-07-05 Bioverativ Therapeutics Inc. Optimized factor viii gene
DK3620534T3 (da) 2013-03-14 2021-12-06 Caribou Biosciences Inc Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
KR20160075676A (ko) 2013-10-24 2016-06-29 오스페달레 산 라파엘 에스.알.엘. 방법
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
US10501811B2 (en) 2015-04-22 2019-12-10 Kyoto University Method for sorting tissue cells
WO2016201507A1 (en) * 2015-06-15 2016-12-22 Murdoch Childrens Research Institute Method of measuring chimerism
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
MX2018009375A (es) 2016-02-01 2018-09-05 Bioverativ Therapeutics Inc Genes del factor viii optimizados.
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017346788A1 (en) * 2016-10-18 2019-05-02 Oregon Health & Science University Cytomegalovirus vectors eliciting T cells restricted by major histocompatibility complex E molecules
CN106566838B (zh) * 2016-11-14 2019-11-01 上海伯豪生物技术有限公司 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用
KR20200020677A (ko) * 2017-04-21 2020-02-26 오스페달레 산 라파엘 에스.알.엘. 유전자 치료법
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
TW201920255A (zh) 2017-08-09 2019-06-01 美商生物化學醫療公司 核酸分子及其用途
AU2019215063A1 (en) 2018-02-01 2020-09-03 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
CN113396223A (zh) 2018-12-06 2021-09-14 比奥维拉迪维治疗股份有限公司 表达因子ix的慢病毒载体的用途
JP2022549932A (ja) 2019-09-30 2022-11-29 バイオベラティブ セラピューティクス インコーポレイテッド レンチウイルスベクター製剤
WO2021097109A1 (en) * 2019-11-12 2021-05-20 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease
EP4171657A1 (en) 2020-06-24 2023-05-03 Bioverativ Therapeutics Inc. Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
IL310879A (en) 2021-08-23 2024-04-01 Bioverativ Therapeutics Inc Expression system in colovirus
AU2022332276A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
CA3232988A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
TW202417019A (zh) * 2022-07-25 2024-05-01 美商現代公司 用於細胞選擇性表現之經工程化多核苷酸

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JP2005504539A (ja) * 2001-10-02 2005-02-17 インスティテュット クレイトン ド ラ リシェルシュ 制限発現レンチウイルス性ベクターに関連する方法及び組成物並びにその応用
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WO2007000668A2 (en) * 2005-05-27 2007-01-04 Fondazione Centro San Raffaele Del Monte Tabor Gene vector comprising mi-rna
WO2006128245A1 (en) * 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression
US20090180989A1 (en) * 2005-10-05 2009-07-16 Synageva Biopharma Corp. Compositions and methods for delivering nucleotide sequences to vertebrates
EP2468897A3 (en) 2006-01-05 2013-02-27 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers
US20080095750A1 (en) 2006-05-10 2008-04-24 Pennington Biomedical Research Center, Louisiana State University System Use of adipose-derived stem cells for treatment of leukodystrophies
WO2008073921A2 (en) 2006-12-08 2008-06-19 Asuragen, Inc. Mir-126 regulated genes and pathways as targets for therapeutic intervention
PT2158322T (pt) 2007-06-06 2017-08-09 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
US9938540B2 (en) 2008-11-12 2018-04-10 Ospedale San Raffaele S.R.L. Gene vector for inducing transgene-specific immune tolerance
EP2424571B1 (en) * 2009-04-30 2020-04-08 Ospedale San Raffaele S.r.l. Gene vector

Also Published As

Publication number Publication date
BRPI1010873B1 (pt) 2020-04-14
SG175839A1 (en) 2011-12-29
WO2010125471A2 (en) 2010-11-04
MX2011011508A (es) 2012-02-13
EP2424571B1 (en) 2020-04-08
US9951328B2 (en) 2018-04-24
BRPI1010873A2 (pt) 2016-03-15
JP6144911B2 (ja) 2017-06-07
US20220333103A1 (en) 2022-10-20
CY1123218T1 (el) 2021-10-29
CA2759438C (en) 2021-03-30
LT2424571T (lt) 2020-08-10
KR20120038403A (ko) 2012-04-23
SI2424571T1 (sl) 2020-10-30
CN102596255A (zh) 2012-07-18
US10287579B2 (en) 2019-05-14
EP2424571A2 (en) 2012-03-07
JP6370278B2 (ja) 2018-08-08
KR101793615B1 (ko) 2017-11-03
JP2016028582A (ja) 2016-03-03
DK2424571T3 (da) 2020-05-04
US11407996B2 (en) 2022-08-09
US20120128643A1 (en) 2012-05-24
CN102596255B (zh) 2017-10-13
AU2010243276A1 (en) 2011-11-17
SG10201610827RA (en) 2017-02-27
IL215804A (en) 2016-10-31
EA201171335A1 (ru) 2012-05-30
US20190367911A1 (en) 2019-12-05
HUE049845T2 (hu) 2020-10-28
PT2424571T (pt) 2020-05-06
ES2786039T3 (es) 2020-10-08
US20180044670A1 (en) 2018-02-15
HRP20200647T1 (hr) 2020-10-02
AU2010243276B2 (en) 2016-09-15
JP2012525141A (ja) 2012-10-22
IL215804A0 (en) 2012-01-31
CA2759438A1 (en) 2010-11-04
EA023938B1 (ru) 2016-07-29
WO2010125471A3 (en) 2010-12-29
EP3733214A1 (en) 2020-11-04

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