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
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
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)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
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

ID=42790671

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) SG10201610827RA (enExample)
SI (1) SI2424571T1 (enExample)
SM (1) SMT202000224T1 (enExample)
WO (1) WO2010125471A2 (enExample)

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WO2012006577A2 (en) * 2010-07-08 2012-01-12 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
US10266850B2 (en) 2012-05-25 2019-04-23 The Regents Of The University Of California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
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
CA3034794A1 (en) 2012-12-06 2014-06-12 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
RS64664B1 (sr) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
DK3620534T3 (da) 2013-03-14 2021-12-06 Caribou Biosciences Inc Crispr-cas sammensætninger af nucleinsyre-targeting nucleinsyrer
CA2927712A1 (en) 2013-10-24 2015-04-30 Ospedale San Raffaele S.R.L. Method for the genetic modification of haematopoietic stem and progenitor cells (hspcs) and the use of genetically modified hspcs in gene therapy
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
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WO2016201507A1 (en) * 2015-06-15 2016-12-22 Murdoch Childrens Research Institute Method of measuring chimerism
JP6990176B2 (ja) 2015-10-05 2022-02-03 モデルナティエックス インコーポレイテッド メッセンジャーリボ核酸薬物の治療投与のための方法
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
PH12018501628B1 (en) 2016-02-01 2023-11-10 Bioverativ Therapeutics Inc Optimized factor viii genes
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
CR20190205A (es) * 2016-10-18 2020-01-07 Univ Oregon Health & Science Vectores de citomegalovirus que provocan células t restringidas por moléculas del complejo mayor de histocompatibilidad e
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
EP4491233A3 (en) * 2017-04-21 2025-04-16 Ospedale San Raffaele S.r.l. Gene therapy
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
JP2021512126A (ja) 2018-02-01 2021-05-13 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 第ｖｉｉｉ因子を発現するレンチウイルスベクターの使用
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
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
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
IL299333A (en) 2020-06-24 2023-02-01 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
EP4392566A2 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022332276A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
WO2023056331A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
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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
WO2010125471A2 (en) 2010-11-04
JP2016028582A (ja) 2016-03-03
US11407996B2 (en) 2022-08-09
CA2759438A1 (en) 2010-11-04
SG175839A1 (en) 2011-12-29
AU2010243276A1 (en) 2011-11-17
US20190367911A1 (en) 2019-12-05
CA2759438C (en) 2021-03-30
US20180044670A1 (en) 2018-02-15
HUE049845T2 (hu) 2020-10-28
EA201171335A1 (ru) 2012-05-30
IL215804A (en) 2016-10-31
IL215804A0 (en) 2012-01-31
BRPI1010873B1 (pt) 2020-04-14
US10287579B2 (en) 2019-05-14
HRP20200647T1 (hr) 2020-10-02
BRPI1010873A2 (pt) 2016-03-15
WO2010125471A3 (en) 2010-12-29
JP2012525141A (ja) 2012-10-22
CY1123218T1 (el) 2021-10-29
EP2424571A2 (en) 2012-03-07
SG10201610827RA (en) 2017-02-27
EA023938B1 (ru) 2016-07-29
KR20120038403A (ko) 2012-04-23
PT2424571T (pt) 2020-05-06
KR101793615B1 (ko) 2017-11-03
SMT202000224T1 (it) 2020-05-08
CN102596255B (zh) 2017-10-13
SI2424571T1 (sl) 2020-10-30
US20220333103A1 (en) 2022-10-20
EP3733214A1 (en) 2020-11-04
US9951328B2 (en) 2018-04-24
LT2424571T (lt) 2020-08-10
MX2011011508A (es) 2012-02-13
ES2786039T3 (es) 2020-10-08
US20120128643A1 (en) 2012-05-24
CN102596255A (zh) 2012-07-18
PL2424571T3 (pl) 2020-10-19
JP6144911B2 (ja) 2017-06-07
DK2424571T3 (da) 2020-05-04
EP2424571B1 (en) 2020-04-08
JP6370278B2 (ja) 2018-08-08

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Owner name: FONDAZIONE TELETHON; OSPEDALE SAN RAFFAELE S.R.L.

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Owner name: FONDAZIONE TELETHON ETS

Free format text: FORMER NAME(S): FONDAZIONE TELETHON; OSPEDALE SAN RAFFAELE S.R.L.

Owner name: OSPEDALE SAN RAFFAELE S.R.L.

Free format text: FORMER NAME(S): FONDAZIONE TELETHON; OSPEDALE SAN RAFFAELE S.R.L.