IL215804A - Gene vector, a pharmaceutical composition and a cell for use in gene therapy for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem cell or a hematopoietic progenitor cell but not in a differentiated cell and a method of determining in vitro the differentiation stage of a hematopoietic stem cell or a hematopoietic progenitor cell - Google Patents

Gene vector, a pharmaceutical composition and a cell for use in gene therapy for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem cell or a hematopoietic progenitor cell but not in a differentiated cell and a method of determining in vitro the differentiation stage of a hematopoietic stem cell or a hematopoietic progenitor cell

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Publication number
IL215804A
IL215804A IL215804A IL21580411A IL215804A IL 215804 A IL215804 A IL 215804A IL 215804 A IL215804 A IL 215804A IL 21580411 A IL21580411 A IL 21580411A IL 215804 A IL215804 A IL 215804A
Authority
IL
Israel
Prior art keywords
sorted
hematopoietic
vitro
expression
nucleotide sequence
Prior art date
Application number
IL215804A
Other languages
English (en)
Hebrew (he)
Other versions
IL215804A0 (en
Original Assignee
Fond Telethon
Ospedale San Raffaele S R L In Shortened Form San Raffaele S R L
Fond Centro San Raffaele Del Monte Tabor
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 Fond Telethon, Ospedale San Raffaele S R L In Shortened Form San Raffaele S R L, Fond Centro San Raffaele Del Monte Tabor filed Critical Fond Telethon
Publication of IL215804A0 publication Critical patent/IL215804A0/en
Publication of IL215804A publication Critical patent/IL215804A/en

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    • 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
    • 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
    • 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
    • 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

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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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
IL215804A 2009-04-30 2011-10-23 Gene vector, a pharmaceutical composition and a cell for use in gene therapy for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem cell or a hematopoietic progenitor cell but not in a differentiated cell and a method of determining in vitro the differentiation stage of a hematopoietic stem cell or a hematopoietic progenitor cell IL215804A (en)

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 (2)

Publication Number Publication Date
IL215804A0 IL215804A0 (en) 2012-01-31
IL215804A true IL215804A (en) 2016-10-31

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
IL215804A IL215804A (en) 2009-04-30 2011-10-23 Gene vector, a pharmaceutical composition and a cell for use in gene therapy for preventing or reducing expression of a nucleotide sequence in a hematopoietic stem cell or a hematopoietic progenitor cell but not in a differentiated cell and a method of determining in vitro the differentiation stage of a hematopoietic stem cell or a hematopoietic progenitor cell

Country Status (23)

Country Link
US (4) US10287579B2 (https=)
EP (2) EP2424571B1 (https=)
JP (2) JP6144911B2 (https=)
KR (1) KR101793615B1 (https=)
CN (1) CN102596255B (https=)
AU (1) AU2010243276B2 (https=)
BR (1) BRPI1010873B1 (https=)
CA (1) CA2759438C (https=)
CY (1) CY1123218T1 (https=)
DK (1) DK2424571T3 (https=)
EA (1) EA023938B1 (https=)
ES (1) ES2786039T3 (https=)
HR (1) HRP20200647T1 (https=)
HU (1) HUE049845T2 (https=)
IL (1) IL215804A (https=)
LT (1) LT2424571T (https=)
MX (1) MX2011011508A (https=)
PL (1) PL2424571T3 (https=)
PT (1) PT2424571T (https=)
SG (2) SG10201610827RA (https=)
SI (1) SI2424571T1 (https=)
SM (1) SMT202000224T1 (https=)
WO (1) WO2010125471A2 (https=)

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PT3351636T (pt) 2010-05-14 2020-09-24 Univ Oregon Health & Science Vectores de hcmv e rhcmv recombinantes codificadores de um antigénio heterólogo isolado a partir de um vírus paramyxoviridae e seus usos
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
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014011901A2 (en) * 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
MX374090B (es) 2013-03-14 2025-03-05 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos al ácido nucleico.
ES2747951T3 (es) 2013-10-24 2020-03-12 Ospedale San Raffaele Srl Método
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
JP6960159B6 (ja) * 2015-04-22 2022-06-24 国立大学法人京都大学 組織細胞の選別方法
US20180327820A1 (en) * 2015-06-15 2018-11-15 Murdoch Childrens Research Institute Method of measuring chimerism
AU2016336344A1 (en) 2015-10-05 2018-04-19 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
WO2017136358A1 (en) 2016-02-01 2017-08-10 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
CN110036112B (zh) 2016-10-18 2024-05-10 俄勒冈健康与科学大学 引发受主要组织相容性复合体e分子限制的t细胞的巨细胞病毒载体
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
PT3612624T (pt) * 2017-04-21 2024-11-29 Ospedale San Raffaele Srl Terapia génica
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
AU2019215063B2 (en) 2018-02-01 2025-10-16 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
AU2019393880A1 (en) 2018-12-06 2021-07-15 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor IX
JP7803014B2 (ja) 2019-09-30 2026-01-21 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
AU2020384286A1 (en) * 2019-11-12 2022-06-09 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat X-linked chronic granulomatous disease
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CA3229323A1 (en) 2021-08-23 2023-03-02 Ajay MAGHODIA Optimized factor viii genes
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
CN115725656B (zh) * 2021-09-01 2026-01-16 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
MA71632A (fr) * 2022-07-25 2025-05-30 Modernatx, Inc. Polynucléotides modifiés pour l'expression sélective de cellules
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
JP2016028582A (ja) 2016-03-03
HRP20200647T1 (hr) 2020-10-02
SMT202000224T1 (it) 2020-05-08
EP2424571A2 (en) 2012-03-07
EP2424571B1 (en) 2020-04-08
HUE049845T2 (hu) 2020-10-28
EA201171335A1 (ru) 2012-05-30
AU2010243276A1 (en) 2011-11-17
SI2424571T1 (sl) 2020-10-30
KR20120038403A (ko) 2012-04-23
EP3733214A1 (en) 2020-11-04
CN102596255B (zh) 2017-10-13
IL215804A0 (en) 2012-01-31
BRPI1010873A2 (pt) 2016-03-15
WO2010125471A2 (en) 2010-11-04
US20190367911A1 (en) 2019-12-05
WO2010125471A3 (en) 2010-12-29
JP6370278B2 (ja) 2018-08-08
CA2759438A1 (en) 2010-11-04
EA023938B1 (ru) 2016-07-29
DK2424571T3 (da) 2020-05-04
CY1123218T1 (el) 2021-10-29
KR101793615B1 (ko) 2017-11-03
SG10201610827RA (en) 2017-02-27
JP2012525141A (ja) 2012-10-22
LT2424571T (lt) 2020-08-10
CA2759438C (en) 2021-03-30
US10287579B2 (en) 2019-05-14
US20220333103A1 (en) 2022-10-20
BRPI1010873B1 (pt) 2020-04-14
PL2424571T3 (pl) 2020-10-19
SG175839A1 (en) 2011-12-29
ES2786039T3 (es) 2020-10-08
AU2010243276B2 (en) 2016-09-15
MX2011011508A (es) 2012-02-13
US20120128643A1 (en) 2012-05-24
US9951328B2 (en) 2018-04-24
US20180044670A1 (en) 2018-02-15
US11407996B2 (en) 2022-08-09
US12577560B2 (en) 2026-03-17
PT2424571T (pt) 2020-05-06
JP6144911B2 (ja) 2017-06-07
CN102596255A (zh) 2012-07-18

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