HUE055862T2 - Készítmények és eljárások PKLR fokozott génexpressziójára - Google Patents

Készítmények és eljárások PKLR fokozott génexpressziójára

Info

Publication number
HUE055862T2
HUE055862T2 HUE17786662A HUE17786662A HUE055862T2 HU E055862 T2 HUE055862 T2 HU E055862T2 HU E17786662 A HUE17786662 A HU E17786662A HU E17786662 A HUE17786662 A HU E17786662A HU E055862 T2 HUE055862 T2 HU E055862T2
Authority
HU
Hungary
Prior art keywords
pklr
compositions
methods
gene expression
enhanced gene
Prior art date
Application number
HUE17786662A
Other languages
English (en)
Inventor
Jose C Segovia
Maria G Gomez
Susana Navarro
Nestor Meza
Juan Bueren
Maria G Bravo
Original Assignee
Centro De Investig Energeticas
Fundacion Instituto De Investig
Consorcio Centro De Investig
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 Centro De Investig Energeticas, Fundacion Instituto De Investig, Consorcio Centro De Investig filed Critical Centro De Investig Energeticas
Publication of HUE055862T2 publication Critical patent/HUE055862T2/hu

Links

Classifications

    • 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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • A61P7/06—Antianaemics
    • 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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0641—Erythrocytes
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1205—Phosphotransferases with an alcohol group as acceptor (2.7.1), e.g. protein kinases
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/0104—Pyruvate kinase (2.7.1.40)
HUE17786662A 2016-04-20 2017-04-20 Készítmények és eljárások PKLR fokozott génexpressziójára HUE055862T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662325397P 2016-04-20 2016-04-20

Publications (1)

Publication Number Publication Date
HUE055862T2 true HUE055862T2 (hu) 2021-12-28

Family

ID=60116402

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE17786662A HUE055862T2 (hu) 2016-04-20 2017-04-20 Készítmények és eljárások PKLR fokozott génexpressziójára

Country Status (17)

Country Link
US (2) US11286501B2 (hu)
EP (1) EP3445406B1 (hu)
JP (2) JP7045362B2 (hu)
KR (2) KR102440880B1 (hu)
CN (2) CN116179580A (hu)
AU (2) AU2017254665C1 (hu)
BR (1) BR112018071695A2 (hu)
CA (1) CA3021572A1 (hu)
DK (1) DK3445406T3 (hu)
ES (1) ES2885833T3 (hu)
HU (1) HUE055862T2 (hu)
IL (1) IL262459A (hu)
MX (1) MX2018012872A (hu)
PL (1) PL3445406T3 (hu)
PT (1) PT3445406T (hu)
SG (1) SG11201809096VA (hu)
WO (1) WO2017184903A1 (hu)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3445406B1 (en) 2016-04-20 2021-05-26 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. Compositions and methods for enhanced gene expression of pklr
BR112020007444A2 (pt) 2017-10-16 2020-10-20 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. vetores lentivirais para a liberação de pklr para tratar deficiência de piruvato quinase
AU2021320902A1 (en) 2020-08-07 2023-04-06 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector

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FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US6607879B1 (en) 1998-02-09 2003-08-19 Incyte Corporation Compositions for the detection of blood cell and immunological response gene expression
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
ES2377721T3 (es) 1999-10-12 2012-03-30 Institut Pasteur ADN de triple hélice lentiviral, y vectores y células recombinantes que contienen ADN de triple hélice lentiviral
DE19960113A1 (de) 1999-12-08 2001-06-13 Wita Proteomics Ag Mittel und Verfahren zur Diagnose von Lyme Borreliose sowie Borreliose-Impfstoff
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
GB0024550D0 (hu) * 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
IL161229A0 (en) 2001-10-02 2004-09-27 Inst Clayton De La Rech Methods and compositions relating to restricted expression lentiviral vectors and their applications.
AU2003226973A1 (en) 2002-02-04 2003-09-02 Develogen Aktiengesellschaft Fur Entwicklungsbiologische Forschung Proteins involved in the regulation of energy homeostatis
JP2006524051A (ja) 2003-04-24 2006-10-26 フォンダッチォーネ・セントロ・サン・ラファエル・デル・モンテ・タボール 両方向性合成プロモーターを備えたレンチウイルスベクターおよびその使用
US20070048285A1 (en) * 2005-08-24 2007-03-01 Christopher Baum Self-inactivating retroviral vector
ES2325711B1 (es) 2007-04-17 2010-06-17 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas (Ciemat) Vectores de expresion que comprenden el promotor del gen pklr humano y su uso para la elaboracion de composiciones farmaceuticas destinadas a terapia genica somatica con expresion especifica en celulas eritroides.
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EP3445406B1 (en) 2016-04-20 2021-05-26 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. Compositions and methods for enhanced gene expression of pklr
BR112020007444A2 (pt) 2017-10-16 2020-10-20 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. vetores lentivirais para a liberação de pklr para tratar deficiência de piruvato quinase

Also Published As

Publication number Publication date
AU2017254665C1 (en) 2021-03-11
PL3445406T3 (pl) 2022-02-07
WO2017184903A1 (en) 2017-10-26
US20220177920A1 (en) 2022-06-09
CN109641063A (zh) 2019-04-16
EP3445406B1 (en) 2021-05-26
CN109641063B (zh) 2022-11-15
RU2018140783A3 (hu) 2020-08-17
AU2021200872B2 (en) 2024-04-04
AU2021200872A1 (en) 2021-03-04
US11286501B2 (en) 2022-03-29
SG11201809096VA (en) 2018-11-29
IL262459A (en) 2018-12-31
CN116179580A (zh) 2023-05-30
EP3445406A4 (en) 2019-12-25
PT3445406T (pt) 2021-08-30
EP3445406A1 (en) 2019-02-27
KR20220147607A (ko) 2022-11-03
DK3445406T3 (da) 2021-08-30
MX2018012872A (es) 2019-08-22
KR102440880B1 (ko) 2022-09-06
JP7045362B2 (ja) 2022-03-31
US20190284573A1 (en) 2019-09-19
ES2885833T3 (es) 2021-12-15
AU2017254665B2 (en) 2020-11-12
JP2019514422A (ja) 2019-06-06
BR112018071695A2 (pt) 2019-02-19
KR20190053136A (ko) 2019-05-17
JP2022101543A (ja) 2022-07-06
RU2018140783A (ru) 2020-05-20
CA3021572A1 (en) 2017-10-26
AU2017254665A1 (en) 2018-11-22

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