BR112018071695A2 - composições e métodos para a expressão gênica acentuada de pklr - Google Patents

composições e métodos para a expressão gênica acentuada de pklr

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Publication number
BR112018071695A2
BR112018071695A2 BR112018071695-2A BR112018071695A BR112018071695A2 BR 112018071695 A2 BR112018071695 A2 BR 112018071695A2 BR 112018071695 A BR112018071695 A BR 112018071695A BR 112018071695 A2 BR112018071695 A2 BR 112018071695A2
Authority
BR
Brazil
Prior art keywords
methods
pklr
compositions
gene expression
enhanced gene
Prior art date
Application number
BR112018071695-2A
Other languages
English (en)
Inventor
C. Segovia Jose
G. Gomez Maria
Navarro Susana
Meza Nestor
Bueren Juan
G. Bravo Maria
Original Assignee
Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas
Fundacion Instituto De Investigacion Sanitaria Fundacion Jimenez Diaz
Centro De Investigacion Biomedica En Red
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 Investigaciones Energeticas, Medioambientales Y Tecnologicas, Fundacion Instituto De Investigacion Sanitaria Fundacion Jimenez Diaz, Centro De Investigacion Biomedica En Red filed Critical Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas
Publication of BR112018071695A2 publication Critical patent/BR112018071695A2/pt

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    • C—CHEMISTRY; METALLURGY
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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
    • 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
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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
    • 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
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    • C12N2510/00—Genetically modified cells
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    • 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
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    • 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
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    • 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)

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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

a presente invenção propõe cassetes de polinucleotídeo, vetores de ex-pressão e métodos para a expressão de um gene em células mamíferas a fim de obter uma terapia gênica contra a deficiência de piruvato quinase.
BR112018071695-2A 2016-04-20 2017-04-20 composições e métodos para a expressão gênica acentuada de pklr BR112018071695A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662325397P 2016-04-20 2016-04-20
US62/325,397 2016-04-20
PCT/US2017/028695 WO2017184903A1 (en) 2016-04-20 2017-04-20 Compositions and methods for enhanced gene expression of pklr

Publications (1)

Publication Number Publication Date
BR112018071695A2 true BR112018071695A2 (pt) 2019-02-19

Family

ID=60116402

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018071695-2A BR112018071695A2 (pt) 2016-04-20 2017-04-20 composições e métodos para a expressão gênica acentuada de pklr

Country Status (17)

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3445406T3 (pl) 2016-04-20 2022-02-07 Centro De Investigaciones Energéticas, Medioambientales Y Tecnológicas, O.A., M.P. Kompozycje i sposoby zwiększonej ekspresji genów PKLR
KR20200116076A (ko) * 2017-10-16 2020-10-08 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터
MX2023001615A (es) 2020-08-07 2023-03-08 Spacecraft Seven Llc Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.

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FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
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KR20200116076A (ko) 2017-10-16 2020-10-08 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터

Also Published As

Publication number Publication date
AU2017254665B2 (en) 2020-11-12
AU2021200872B2 (en) 2024-04-04
CN109641063B (zh) 2022-11-15
JP7045362B2 (ja) 2022-03-31
KR102440880B1 (ko) 2022-09-06
MX2018012872A (es) 2019-08-22
PT3445406T (pt) 2021-08-30
JP2019514422A (ja) 2019-06-06
KR20190053136A (ko) 2019-05-17
AU2017254665C1 (en) 2021-03-11
AU2021200872A1 (en) 2021-03-04
JP2022101543A (ja) 2022-07-06
WO2017184903A1 (en) 2017-10-26
RU2018140783A3 (pt) 2020-08-17
EP3445406A1 (en) 2019-02-27
EP3445406A4 (en) 2019-12-25
RU2018140783A (ru) 2020-05-20
IL262459A (en) 2018-12-31
EP3445406B1 (en) 2021-05-26
PL3445406T3 (pl) 2022-02-07
ES2885833T3 (es) 2021-12-15
US20220177920A1 (en) 2022-06-09
KR20220147607A (ko) 2022-11-03
CA3021572A1 (en) 2017-10-26
US11286501B2 (en) 2022-03-29
CN109641063A (zh) 2019-04-16
CN116179580A (zh) 2023-05-30
AU2017254665A1 (en) 2018-11-22
HUE055862T2 (hu) 2021-12-28
US20190284573A1 (en) 2019-09-19
SG11201809096VA (en) 2018-11-29
DK3445406T3 (da) 2021-08-30

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Owner name: CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS (ES) ; FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ (ES) ; CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P. (ES)

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B25L Entry of change of name and/or headquarter and transfer of application, patent and certificate of addition of invention: publication cancelled

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B25D Requested change of name of applicant approved

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B25G Requested change of headquarter approved

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B25F Entry of change of name and/or headquarter and transfer of application, patent and certif. of addition of invention: change of name on requirement

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B25L Entry of change of name and/or headquarter and transfer of application, patent and certificate of addition of invention: publication cancelled

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B350 Update of information on the portal [chapter 15.35 patent gazette]
B25D Requested change of name of applicant approved

Owner name: FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ (ES) ; CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS, O.A., M.P. (ES) ; CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P. (ES)

B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]
B154 Notification of filing of divisional application [chapter 15.50 patent gazette]

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