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)
Polish (pl)
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 nucleic acids [NA]
    • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (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
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector
EP10723305.8A EP2424571B1 (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 (23)

Country Link
US (4) US10287579B2 (cg-RX-API-DMAC7.html)
EP (2) EP3733214A1 (cg-RX-API-DMAC7.html)
JP (2) JP6144911B2 (cg-RX-API-DMAC7.html)
KR (1) KR101793615B1 (cg-RX-API-DMAC7.html)
CN (1) CN102596255B (cg-RX-API-DMAC7.html)
AU (1) AU2010243276B2 (cg-RX-API-DMAC7.html)
BR (1) BRPI1010873B1 (cg-RX-API-DMAC7.html)
CA (1) CA2759438C (cg-RX-API-DMAC7.html)
CY (1) CY1123218T1 (cg-RX-API-DMAC7.html)
DK (1) DK2424571T3 (cg-RX-API-DMAC7.html)
EA (1) EA023938B1 (cg-RX-API-DMAC7.html)
ES (1) ES2786039T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20200647T1 (cg-RX-API-DMAC7.html)
HU (1) HUE049845T2 (cg-RX-API-DMAC7.html)
IL (1) IL215804A (cg-RX-API-DMAC7.html)
LT (1) LT2424571T (cg-RX-API-DMAC7.html)
MX (1) MX2011011508A (cg-RX-API-DMAC7.html)
PL (1) PL2424571T3 (cg-RX-API-DMAC7.html)
PT (1) PT2424571T (cg-RX-API-DMAC7.html)
SG (2) SG175839A1 (cg-RX-API-DMAC7.html)
SI (1) SI2424571T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000224T1 (cg-RX-API-DMAC7.html)
WO (1) WO2010125471A2 (cg-RX-API-DMAC7.html)

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KR101793615B1 (ko) 2009-04-30 2017-11-03 오스페달레 산 라파엘 에스.알.엘. 유전자 벡터
WO2011143653A2 (en) 2010-05-14 2011-11-17 Oregon Health & Science University Recombinant hcmv and rhcmv vectors and uses thereof
CA2804791C (en) * 2010-07-08 2019-07-30 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
WO2013152230A1 (en) * 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP2872154B1 (en) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Methods and compositions for delivery of biologics
LT3138910T (lt) 2012-12-06 2017-11-10 Sigma-Aldrich Co. Llc Crispr pagrįstas genomo modifikavimas ir reguliavimas
SI2956477T2 (sl) 2013-02-15 2024-05-31 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
SG11201603166UA (en) 2013-10-24 2016-05-30 Ospedale San Raffaele Srl Method
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
EP3318630B1 (en) * 2015-04-22 2020-11-04 iPS Academia Japan, Inc. Method for sorting tissue cells
AU2016281193B2 (en) * 2015-06-15 2022-06-16 Murdoch Childrens Research Institute Method of measuring chimerism
ES2810701T5 (es) 2015-10-05 2024-07-11 Modernatx Inc Procedimientos para la administración terapéutica de medicamentos de ácido ribonucleico mensajero
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
PT3411478T (pt) 2016-02-01 2022-09-13 Bioverativ Therapeutics Inc Genes do fator viii otimizados
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
EP3529363B1 (en) 2016-10-18 2025-07-30 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
PL3612624T3 (pl) * 2017-04-21 2025-02-24 Ospedale San Raffaele S.R.L. Terapia genowa
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
KR102780287B1 (ko) 2017-08-09 2025-03-14 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
AU2019215063B2 (en) 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
BR112021002017A2 (pt) 2018-08-09 2021-05-11 Bioverativ Therapeutics Inc. moléculas de ácido nucleico e usos das mesmas para terapia genética não viral
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
CN114829614A (zh) * 2019-11-12 2022-07-29 加利福尼亚大学董事会 治疗x连锁慢性肉芽肿病的造血干细胞中的慢病毒载体
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
CN116194152A (zh) 2020-08-23 2023-05-30 比奥维拉迪维治疗股份有限公司 用于改进封闭端DNA(ceDNA)的产生的经修饰的杆状病毒系统
AU2022332276A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
JP2024534123A (ja) 2021-08-23 2024-09-18 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 バキュロウイルス発現システム
CN115725656A (zh) * 2021-09-01 2023-03-03 联邦生物科技(珠海横琴)有限公司 一种以细胞质rna病毒为载体递送干扰rna的方法
KR20240067122A (ko) 2021-09-30 2024-05-16 바이오버라티브 테라퓨틱스 인크. 감소된 면역원성을 갖는 인자 viii 폴리펩타이드를 암호화하는 핵산
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
WO2025122739A1 (en) * 2023-12-05 2025-06-12 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Compositions and methods for treating a heart disease

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KR101793615B1 (ko) * 2009-04-30 2017-11-03 오스페달레 산 라파엘 에스.알.엘. 유전자 벡터

Also Published As

Publication number Publication date
PT2424571T (pt) 2020-05-06
MX2011011508A (es) 2012-02-13
US9951328B2 (en) 2018-04-24
BRPI1010873B1 (pt) 2020-04-14
HRP20200647T1 (hr) 2020-10-02
US20220333103A1 (en) 2022-10-20
AU2010243276B2 (en) 2016-09-15
US20180044670A1 (en) 2018-02-15
US10287579B2 (en) 2019-05-14
US20190367911A1 (en) 2019-12-05
WO2010125471A2 (en) 2010-11-04
JP2012525141A (ja) 2012-10-22
US11407996B2 (en) 2022-08-09
IL215804A (en) 2016-10-31
US20120128643A1 (en) 2012-05-24
CN102596255A (zh) 2012-07-18
KR101793615B1 (ko) 2017-11-03
JP6144911B2 (ja) 2017-06-07
WO2010125471A3 (en) 2010-12-29
CY1123218T1 (el) 2021-10-29
DK2424571T3 (da) 2020-05-04
SG10201610827RA (en) 2017-02-27
SG175839A1 (en) 2011-12-29
HUE049845T2 (hu) 2020-10-28
EA023938B1 (ru) 2016-07-29
IL215804A0 (en) 2012-01-31
EP2424571B1 (en) 2020-04-08
CA2759438C (en) 2021-03-30
LT2424571T (lt) 2020-08-10
EP3733214A1 (en) 2020-11-04
EA201171335A1 (ru) 2012-05-30
EP2424571A2 (en) 2012-03-07
SI2424571T1 (sl) 2020-10-30
ES2786039T3 (es) 2020-10-08
JP6370278B2 (ja) 2018-08-08
CN102596255B (zh) 2017-10-13
KR20120038403A (ko) 2012-04-23
AU2010243276A1 (en) 2011-11-17
CA2759438A1 (en) 2010-11-04
JP2016028582A (ja) 2016-03-03
SMT202000224T1 (it) 2020-05-08
BRPI1010873A2 (pt) 2016-03-15

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