CA2759438C - Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell - Google Patents

Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell Download PDF

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Publication number
CA2759438C
CA2759438C CA2759438A CA2759438A CA2759438C CA 2759438 C CA2759438 C CA 2759438C CA 2759438 A CA2759438 A CA 2759438A CA 2759438 A CA2759438 A CA 2759438A CA 2759438 C CA2759438 C CA 2759438C
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cell
cells
mir
expression
galc
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English (en)
French (fr)
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CA2759438A1 (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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    • 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
    • C12N15/63—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
    • 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
    • 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
    • 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

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  • 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)
CA2759438A 2009-04-30 2010-04-30 Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell Active CA2759438C (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
CA2759438A1 CA2759438A1 (en) 2010-11-04
CA2759438C true CA2759438C (en) 2021-03-30

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2759438A Active CA2759438C (en) 2009-04-30 2010-04-30 Gene vectors and uses thereof for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem or progenitor cell but not in a differentiated cell

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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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
GB2537000C (en) 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
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CA2891347C (en) 2012-12-06 2018-02-27 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
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US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
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US10849920B2 (en) 2015-10-05 2020-12-01 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
RS63548B1 (sr) 2016-02-01 2022-09-30 Bioverativ Therapeutics Inc Optimizovani geni faktora viii
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
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CA3040303A1 (en) * 2016-10-18 2018-04-26 Oregon Health & Science University Cytomegalovirus vectors eliciting t cells restricted by major histocompatibility complex e molecules
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WO2018193119A1 (en) * 2017-04-21 2018-10-25 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
KR20250040746A (ko) 2017-08-09 2025-03-24 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
CN111918674A (zh) 2018-02-01 2020-11-10 比奥维拉迪维治疗股份有限公司 表达因子viii的慢病毒载体的用途
JP7602454B2 (ja) 2018-08-09 2024-12-18 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 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
SG11202105880TA (en) 2018-12-06 2021-07-29 Bioverativ Therapeutics Inc Use of lentiviral vectors expressing factor ix
US12403164B2 (en) 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CA3161175A1 (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
BR112022026127A2 (pt) 2020-06-24 2023-01-17 Bioverativ Therapeutics Inc Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
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
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
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
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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
US20120128643A1 (en) 2012-05-24
JP2016028582A (ja) 2016-03-03
BRPI1010873B1 (pt) 2020-04-14
SG10201610827RA (en) 2017-02-27
CY1123218T1 (el) 2021-10-29
IL215804A0 (en) 2012-01-31
LT2424571T (lt) 2020-08-10
IL215804A (en) 2016-10-31
DK2424571T3 (da) 2020-05-04
EA023938B1 (ru) 2016-07-29
AU2010243276B2 (en) 2016-09-15
ES2786039T3 (es) 2020-10-08
KR101793615B1 (ko) 2017-11-03
MX2011011508A (es) 2012-02-13
JP6370278B2 (ja) 2018-08-08
WO2010125471A3 (en) 2010-12-29
CN102596255A (zh) 2012-07-18
WO2010125471A2 (en) 2010-11-04
US20190367911A1 (en) 2019-12-05
EP2424571A2 (en) 2012-03-07
JP6144911B2 (ja) 2017-06-07
US9951328B2 (en) 2018-04-24
PL2424571T3 (pl) 2020-10-19
US10287579B2 (en) 2019-05-14
EP3733214A1 (en) 2020-11-04
CA2759438A1 (en) 2010-11-04
US20180044670A1 (en) 2018-02-15
HUE049845T2 (hu) 2020-10-28
AU2010243276A1 (en) 2011-11-17
JP2012525141A (ja) 2012-10-22
US20220333103A1 (en) 2022-10-20
HRP20200647T1 (hr) 2020-10-02
EA201171335A1 (ru) 2012-05-30
SI2424571T1 (sl) 2020-10-30
CN102596255B (zh) 2017-10-13
US11407996B2 (en) 2022-08-09
SG175839A1 (en) 2011-12-29
SMT202000224T1 (it) 2020-05-08
EP2424571B1 (en) 2020-04-08
PT2424571T (pt) 2020-05-06
BRPI1010873A2 (pt) 2016-03-15
KR20120038403A (ko) 2012-04-23

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