LT2424571T - Genų vektorius - Google Patents

Genų vektorius

Info

Publication number
LT2424571T
LT2424571T LTEP10723305.8T LT10723305T LT2424571T LT 2424571 T LT2424571 T LT 2424571T LT 10723305 T LT10723305 T LT 10723305T LT 2424571 T LT2424571 T LT 2424571T
Authority
LT
Lithuania
Prior art keywords
gene vector
gene
vector
Prior art date
Application number
LTEP10723305.8T
Other languages
English (en)
Inventor
Alessandra Biffi
Bernhard 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 LT2424571T publication Critical patent/LT2424571T/lt

Links

Classifications

    • 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
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • 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
    • 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
    • C12N15/63Introduction 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
    • 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
    • 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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (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)
LTEP10723305.8T 2009-04-30 2010-04-30 Genų vektorius LT2424571T (lt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
LT2424571T true LT2424571T (lt) 2020-08-10

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP10723305.8T LT2424571T (lt) 2009-04-30 2010-04-30 Genų vektorius

Country Status (22)

Country Link
US (4) US10287579B2 (lt)
EP (2) EP3733214A1 (lt)
JP (2) JP6144911B2 (lt)
KR (1) KR101793615B1 (lt)
CN (1) CN102596255B (lt)
AU (1) AU2010243276B2 (lt)
BR (1) BRPI1010873B1 (lt)
CA (1) CA2759438C (lt)
CY (1) CY1123218T1 (lt)
DK (1) DK2424571T3 (lt)
EA (1) EA023938B1 (lt)
ES (1) ES2786039T3 (lt)
HR (1) HRP20200647T1 (lt)
HU (1) HUE049845T2 (lt)
IL (1) IL215804A (lt)
LT (1) LT2424571T (lt)
MX (1) MX2011011508A (lt)
PL (1) PL2424571T3 (lt)
PT (1) PT2424571T (lt)
SG (2) SG10201610827RA (lt)
SI (1) SI2424571T1 (lt)
WO (1) WO2010125471A2 (lt)

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BRPI1010873B1 (pt) * 2009-04-30 2020-04-14 Fond Centro San Raffaele Del Monte Tabor usos de um vetor de genes, de uma composição farmacêutica e de uma célula
ES2788198T3 (es) 2010-05-14 2020-10-20 Univ Oregon Health & Science Vectores de CMVH y CMVRh recombinantes que codifican un antígeno heterólogo aislado del virus de la hepatitis B y usos de los mismos
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
DK3401400T3 (da) 2012-05-25 2019-06-03 Univ California Fremgangsmåder og sammensætninger til rna-styret mål-dna-modifikation og til rna-styret transskriptionsmodulering
JP6329537B2 (ja) 2012-07-11 2018-05-23 サンガモ セラピューティクス, インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
JP6620018B2 (ja) 2012-12-06 2019-12-11 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーSigma−Aldrich Co., LLC Crisprに基づくゲノム修飾および制御
ES2959747T3 (es) 2013-02-15 2024-02-28 Bioverativ Therapeutics Inc Gen del factor VIII optimizado
EP2971167B1 (en) 2013-03-14 2019-07-31 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US10617721B2 (en) 2013-10-24 2020-04-14 Ospedale San Raffaele S.R.L. Methods for genetic modification of stem cells
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
JP2018517421A (ja) * 2015-06-15 2018-07-05 マードック チルドレンズ リサーチ インスティチュート キメリズムを測定する方法
EP3359670B2 (en) 2015-10-05 2024-02-14 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
LT3411478T (lt) 2016-02-01 2022-09-26 Bioverativ Therapeutics Inc. Optimizuoti viii faktoriaus genai
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
BR112019007982A2 (pt) * 2016-10-18 2019-07-02 Univ Oregon Health & Science vetores de citomegalovírus elicitando células t restringidas por moléculas do complexo de histocompatibilidade e principal
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
JP7252135B2 (ja) * 2017-04-21 2023-04-04 オスペダーレ サン ラファエレ エス.アール.エル 遺伝子療法
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
WO2019152692A1 (en) 2018-02-01 2019-08-08 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
WO2021067389A1 (en) 2019-09-30 2021-04-08 Bioverativ Therapeutics Inc. Lentiviral vector formulations
US20220378937A1 (en) * 2019-11-12 2022-12-01 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease
KR20230074703A (ko) 2020-06-24 2023-05-31 바이오버라티브 테라퓨틱스 인크. 유리 인자 viii을 상기 단백질이 발현되도록 변형된 렌티바이러스 벡터의 제제로부터 제거하는 방법
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
TW202323274A (zh) 2021-08-23 2023-06-16 美商百歐維拉提夫治療公司 優化因子viii基因
EP4392566A2 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Baculovirus expression system
CN118019758A (zh) 2021-09-30 2024-05-10 比奥维拉迪维治疗股份有限公司 编码免疫原性降低的因子viii多肽的核酸
WO2024026257A2 (en) * 2022-07-25 2024-02-01 Modernatx, Inc. Engineered polynucleotides for cell selective expression

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KR20130114758A (ko) * 2005-05-27 2013-10-17 오스페달레 산 라파엘 에스.알.엘. Mi-rna를 포함하는 유전자 벡터
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EP2468890B1 (en) 2006-01-05 2014-03-19 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers of the breast or lung
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
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LT2158322T (lt) 2007-06-06 2017-08-25 Genzyme Corporation Lizosomų kaupimo ligų genų terapija
WO2010055413A1 (en) 2008-11-12 2010-05-20 Fondazione Centro San Raffaele Del Monte Tabor Gene vector for inducing transgene-specific immune tolerance
BRPI1010873B1 (pt) 2009-04-30 2020-04-14 Fond Centro San Raffaele Del Monte Tabor usos de um vetor de genes, de uma composição farmacêutica e de uma célula

Also Published As

Publication number Publication date
JP6370278B2 (ja) 2018-08-08
IL215804A0 (en) 2012-01-31
EA201171335A1 (ru) 2012-05-30
US10287579B2 (en) 2019-05-14
WO2010125471A3 (en) 2010-12-29
US20220333103A1 (en) 2022-10-20
EP2424571B1 (en) 2020-04-08
IL215804A (en) 2016-10-31
SI2424571T1 (sl) 2020-10-30
US20180044670A1 (en) 2018-02-15
CY1123218T1 (el) 2021-10-29
CN102596255B (zh) 2017-10-13
DK2424571T3 (da) 2020-05-04
SG10201610827RA (en) 2017-02-27
ES2786039T3 (es) 2020-10-08
US20120128643A1 (en) 2012-05-24
SG175839A1 (en) 2011-12-29
AU2010243276B2 (en) 2016-09-15
EA023938B1 (ru) 2016-07-29
JP2012525141A (ja) 2012-10-22
AU2010243276A1 (en) 2011-11-17
PL2424571T3 (pl) 2020-10-19
HUE049845T2 (hu) 2020-10-28
KR20120038403A (ko) 2012-04-23
WO2010125471A2 (en) 2010-11-04
CN102596255A (zh) 2012-07-18
EP2424571A2 (en) 2012-03-07
BRPI1010873B1 (pt) 2020-04-14
KR101793615B1 (ko) 2017-11-03
PT2424571T (pt) 2020-05-06
MX2011011508A (es) 2012-02-13
JP6144911B2 (ja) 2017-06-07
CA2759438A1 (en) 2010-11-04
BRPI1010873A2 (pt) 2016-03-15
US9951328B2 (en) 2018-04-24
EP3733214A1 (en) 2020-11-04
US11407996B2 (en) 2022-08-09
US20190367911A1 (en) 2019-12-05
JP2016028582A (ja) 2016-03-03
HRP20200647T1 (hr) 2020-10-02
CA2759438C (en) 2021-03-30

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