CY1121630T1 - Ρυθμιση της εκφρασης γονιδιου σφαιρινης διαμεσολαβουμενη απο νουκλεασες - Google Patents

Ρυθμιση της εκφρασης γονιδιου σφαιρινης διαμεσολαβουμενη απο νουκλεασες

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Publication number
CY1121630T1
CY1121630T1 CY20191100521T CY191100521T CY1121630T1 CY 1121630 T1 CY1121630 T1 CY 1121630T1 CY 20191100521 T CY20191100521 T CY 20191100521T CY 191100521 T CY191100521 T CY 191100521T CY 1121630 T1 CY1121630 T1 CY 1121630T1
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Cyprus
Prior art keywords
nuclease
gene expression
global gene
mediated regulation
mediated
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CY20191100521T
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English (en)
Inventor
Stuart H. Orkin
Andreas Reik
Fyodor Urnov
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Children's Medical Center Corporation
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Publication date
Application filed by Children's Medical Center Corporation filed Critical Children's Medical Center Corporation
Publication of CY1121630T1 publication Critical patent/CY1121630T1/el

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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
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    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

Η παρούσα αποκάλυψη εμπίπτει στο πεδίο της γονιδιωματικής μηχανικής, συγκεκριμένα στοχευμένης τροποποίησης του γονιδιώματος ενός αιμοποιητικού κυττάρου.
CY20191100521T 2013-11-13 2019-05-15 Ρυθμιση της εκφρασης γονιδιου σφαιρινης διαμεσολαβουμενη απο νουκλεασες CY1121630T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361903823P 2013-11-13 2013-11-13
US201462042075P 2014-08-26 2014-08-26
PCT/US2014/065473 WO2015073683A2 (en) 2013-11-13 2014-11-13 Nuclease-mediated regulation of gene expression

Publications (1)

Publication Number Publication Date
CY1121630T1 true CY1121630T1 (el) 2020-07-31

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Country Status (13)

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US (2) US11021696B2 (el)
EP (3) EP3492593B1 (el)
CY (1) CY1121630T1 (el)
DK (2) DK3492593T3 (el)
ES (2) ES2895069T3 (el)
HR (2) HRP20211706T1 (el)
HU (2) HUE056436T2 (el)
LT (2) LT3492593T (el)
PL (2) PL3068881T3 (el)
PT (2) PT3492593T (el)
RS (2) RS58671B1 (el)
SI (2) SI3492593T1 (el)
WO (1) WO2015073683A2 (el)

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