AU2010243276B2 - Gene vector - Google Patents

Gene vector Download PDF

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Publication number
AU2010243276B2
AU2010243276B2 AU2010243276A AU2010243276A AU2010243276B2 AU 2010243276 B2 AU2010243276 B2 AU 2010243276B2 AU 2010243276 A AU2010243276 A AU 2010243276A AU 2010243276 A AU2010243276 A AU 2010243276A AU 2010243276 B2 AU2010243276 B2 AU 2010243276B2
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AU
Australia
Prior art keywords
cell
cells
mir
expression
galc
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AU2010243276A
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AU2010243276A1 (en
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
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Fondazione Telethon
Ospedale San Raffaele SRL
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Fondazione Telethon
Ospedale San Raffaele SRL
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Assigned to FONDAZIONE TELETHON, OSPEDALE SAN RAFFAELE S.R.L. reassignment FONDAZIONE TELETHON Request for Assignment Assignors: FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR, FONDAZIONE TELETHON
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Publication of AU2010243276B2 publication Critical patent/AU2010243276B2/en
Assigned to OSPEDALE SAN RAFFAELE S.R.L., FONDAZIONE TELETHON ETS reassignment OSPEDALE SAN RAFFAELE S.R.L. Request to Amend Deed and Register Assignors: OSPEDALE SAN RAFFAELE S.R.L., FONDAZIONE TELETHON
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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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/555—Interferons [IFN]
    • C07K14/56—IFN-alpha
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (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)
AU2010243276A 2009-04-30 2010-04-30 Gene vector Active AU2010243276B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
US61/174,124 2009-04-30
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (2)

Publication Number Publication Date
AU2010243276A1 AU2010243276A1 (en) 2011-11-17
AU2010243276B2 true AU2010243276B2 (en) 2016-09-15

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2010243276A Active AU2010243276B2 (en) 2009-04-30 2010-04-30 Gene vector

Country Status (23)

Country Link
US (4) US10287579B2 (enExample)
EP (2) EP2424571B1 (enExample)
JP (2) JP6144911B2 (enExample)
KR (1) KR101793615B1 (enExample)
CN (1) CN102596255B (enExample)
AU (1) AU2010243276B2 (enExample)
BR (1) BRPI1010873B1 (enExample)
CA (1) CA2759438C (enExample)
CY (1) CY1123218T1 (enExample)
DK (1) DK2424571T3 (enExample)
EA (1) EA023938B1 (enExample)
ES (1) ES2786039T3 (enExample)
HR (1) HRP20200647T1 (enExample)
HU (1) HUE049845T2 (enExample)
IL (1) IL215804A (enExample)
LT (1) LT2424571T (enExample)
MX (1) MX2011011508A (enExample)
PL (1) PL2424571T3 (enExample)
PT (1) PT2424571T (enExample)
SG (2) SG175839A1 (enExample)
SI (1) SI2424571T1 (enExample)
SM (1) SMT202000224T1 (enExample)
WO (1) WO2010125471A2 (enExample)

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PT3363902T (pt) 2012-12-06 2019-12-19 Sigma Aldrich Co Llc Modificação e regulação de genoma baseadas em crispr
LT2956477T (lt) 2013-02-15 2021-02-10 Bioverativ Therapeutics Inc. Optimizuotas faktoriaus viii genas
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US10188750B1 (en) * 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
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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
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IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
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WO2019152692A1 (en) 2018-02-01 2019-08-08 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing factor viii
CN119955796A (zh) 2018-08-09 2025-05-09 比奥维拉迪维治疗股份有限公司 核酸分子及其用于非病毒基因疗法的用途
BR112021010047A2 (pt) 2018-12-06 2021-08-24 Bioverativ Therapeutics Inc. Uso de vetores lentivirais expressando o fator ix
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
KR20220097487A (ko) * 2019-11-12 2022-07-07 더 리전트 오브 더 유니버시티 오브 캘리포니아 X-연관 만성 육아종 질환을 치료하기 위한 조혈 줄기 세포에서의 렌티바이러스 벡터
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
EP4392443A1 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Optimized factor viii genes
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
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
CA3232988A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
EP4561638A2 (en) * 2022-07-25 2025-06-04 ModernaTX, Inc. Engineered polynucleotides for cell selective expression
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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Also Published As

Publication number Publication date
HUE049845T2 (hu) 2020-10-28
BRPI1010873A2 (pt) 2016-03-15
EP3733214A1 (en) 2020-11-04
SG175839A1 (en) 2011-12-29
JP6370278B2 (ja) 2018-08-08
KR20120038403A (ko) 2012-04-23
EA201171335A1 (ru) 2012-05-30
LT2424571T (lt) 2020-08-10
US10287579B2 (en) 2019-05-14
DK2424571T3 (da) 2020-05-04
SMT202000224T1 (it) 2020-05-08
JP2012525141A (ja) 2012-10-22
EP2424571B1 (en) 2020-04-08
JP6144911B2 (ja) 2017-06-07
US9951328B2 (en) 2018-04-24
CN102596255B (zh) 2017-10-13
US20220333103A1 (en) 2022-10-20
PL2424571T3 (pl) 2020-10-19
BRPI1010873B1 (pt) 2020-04-14
CA2759438A1 (en) 2010-11-04
IL215804A0 (en) 2012-01-31
PT2424571T (pt) 2020-05-06
US11407996B2 (en) 2022-08-09
MX2011011508A (es) 2012-02-13
EP2424571A2 (en) 2012-03-07
CA2759438C (en) 2021-03-30
CY1123218T1 (el) 2021-10-29
US20190367911A1 (en) 2019-12-05
US20120128643A1 (en) 2012-05-24
JP2016028582A (ja) 2016-03-03
IL215804A (en) 2016-10-31
SI2424571T1 (sl) 2020-10-30
EA023938B1 (ru) 2016-07-29
ES2786039T3 (es) 2020-10-08
KR101793615B1 (ko) 2017-11-03
WO2010125471A3 (en) 2010-12-29
US20180044670A1 (en) 2018-02-15
AU2010243276A1 (en) 2011-11-17
SG10201610827RA (en) 2017-02-27
CN102596255A (zh) 2012-07-18
WO2010125471A2 (en) 2010-11-04
HRP20200647T1 (hr) 2020-10-02

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