CY1123218T1 - Διαβιβαστης γονιδιου - Google Patents

Διαβιβαστης γονιδιου

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Publication number
CY1123218T1
CY1123218T1 CY20201100405T CY201100405T CY1123218T1 CY 1123218 T1 CY1123218 T1 CY 1123218T1 CY 20201100405 T CY20201100405 T CY 20201100405T CY 201100405 T CY201100405 T CY 201100405T CY 1123218 T1 CY1123218 T1 CY 1123218T1
Authority
CY
Cyprus
Prior art keywords
gene transfer
hspc
hsc
nucleotide sequence
cell
Prior art date
Application number
CY20201100405T
Other languages
English (en)
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
Original Assignee
Ospedale San Raffaele S.R.L.
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. filed Critical Ospedale San Raffaele S.R.L.
Publication of CY1123218T1 publication Critical patent/CY1123218T1/el

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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
    • C12N15/11—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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (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)
  • 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)

Abstract

Διαβιβαστής γονιδίου για χρήση σε γονιδιακή θεραπεία που εμπεριέχει τουλάχιστον μία αλληλουχία miRNA στόχου λειτουργικά συνδεδεμένη με μία αλληλουχία νουκλεοτιδίων που έχει ένα αντίστοιχο miRNA σε ένα αιμοποιητικό προγονικό κύτταρο (HSPC) ή αιμοποιητικό βλαστικό κύτταρο (HSC) που αποτρέπει ή μειώνει την έκφραση της αλληλουχίας νουκλεοτιδίων σε ένα HSPC ή HSC αλλά όχι σε ένα διαφοροποιημένο κύτταρο.
CY20201100405T 2009-04-30 2020-05-04 Διαβιβαστης γονιδιου CY1123218T1 (el)

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
CY1123218T1 true CY1123218T1 (el) 2021-10-29

Family

ID=42790671

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20201100405T CY1123218T1 (el) 2009-04-30 2020-05-04 Διαβιβαστης γονιδιου

Country Status (22)

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

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CA2804791C (en) * 2010-07-08 2019-07-30 Duke University Direct reprogramming of cells to cardiac myocyte fate
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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
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EP2872154B1 (en) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Methods and compositions for delivery of biologics
EP3617309A3 (en) 2012-12-06 2020-05-06 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
HRP20231183T1 (hr) 2013-02-15 2024-01-05 Bioverativ Therapeutics Inc. Optimizirani gen faktora viii
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SI3411478T1 (sl) 2016-02-01 2022-10-28 Bioverativ Therapeutics Inc. Optimirani geni dejavnika VIII
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
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CA3060145A1 (en) * 2017-04-21 2018-10-25 Ospedale San Raffaele S.R.L. Cytokine gene therapy for treating or preventing cancer
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WO2021097109A1 (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
TW202208619A (zh) 2020-06-24 2022-03-01 美商百歐維拉提夫治療公司 用於純化病毒載體之方法
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IL310997A (en) 2021-08-23 2024-04-01 Bioverativ Therapeutics Inc Factor VIII gene optimization
KR20240049821A (ko) 2021-08-23 2024-04-17 바이오버라티브 테라퓨틱스 인크. 바큘로바이러스 발현 시스템
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Also Published As

Publication number Publication date
US20120128643A1 (en) 2012-05-24
IL215804A0 (en) 2012-01-31
US20220333103A1 (en) 2022-10-20
KR20120038403A (ko) 2012-04-23
JP2016028582A (ja) 2016-03-03
US11407996B2 (en) 2022-08-09
LT2424571T (lt) 2020-08-10
BRPI1010873B1 (pt) 2020-04-14
JP2012525141A (ja) 2012-10-22
SG175839A1 (en) 2011-12-29
CA2759438C (en) 2021-03-30
EA201171335A1 (ru) 2012-05-30
AU2010243276A1 (en) 2011-11-17
CA2759438A1 (en) 2010-11-04
US9951328B2 (en) 2018-04-24
JP6144911B2 (ja) 2017-06-07
HUE049845T2 (hu) 2020-10-28
EA023938B1 (ru) 2016-07-29
IL215804A (en) 2016-10-31
AU2010243276B2 (en) 2016-09-15
SI2424571T1 (sl) 2020-10-30
US20190367911A1 (en) 2019-12-05
KR101793615B1 (ko) 2017-11-03
JP6370278B2 (ja) 2018-08-08
HRP20200647T1 (hr) 2020-10-02
ES2786039T3 (es) 2020-10-08
EP2424571B1 (en) 2020-04-08
DK2424571T3 (da) 2020-05-04
CN102596255A (zh) 2012-07-18
US10287579B2 (en) 2019-05-14
WO2010125471A3 (en) 2010-12-29
WO2010125471A2 (en) 2010-11-04
SG10201610827RA (en) 2017-02-27
PL2424571T3 (pl) 2020-10-19
EP3733214A1 (en) 2020-11-04
PT2424571T (pt) 2020-05-06
BRPI1010873A2 (pt) 2016-03-15
CN102596255B (zh) 2017-10-13
US20180044670A1 (en) 2018-02-15
EP2424571A2 (en) 2012-03-07
MX2011011508A (es) 2012-02-13

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