MX2020003954A - Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa. - Google Patents

Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa.

Info

Publication number
MX2020003954A
MX2020003954A MX2020003954A MX2020003954A MX2020003954A MX 2020003954 A MX2020003954 A MX 2020003954A MX 2020003954 A MX2020003954 A MX 2020003954A MX 2020003954 A MX2020003954 A MX 2020003954A MX 2020003954 A MX2020003954 A MX 2020003954A
Authority
MX
Mexico
Prior art keywords
pyruvate kinase
pklr
treat
delivery
kinase deficiency
Prior art date
Application number
MX2020003954A
Other languages
English (en)
Inventor
Jose C Segovia
Maria G Gomez
Susana Navarro
Nestor Meza
Juan Bueren
Maria G Bravo
Original Assignee
Consorcio Centro De Investig Biomedica En Red M P
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 Consorcio Centro De Investig Biomedica En Red M P filed Critical Consorcio Centro De Investig Biomedica En Red M P
Publication of MX2020003954A publication Critical patent/MX2020003954A/es

Links

Classifications

    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • 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
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
    • C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Mycology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente descripción proporciona casetes de polinucleótidos, vectores de expresión y métodos para expresión de un gen en células de mamífero, a los efectos de brindar una terapia génica contra la deficiencia de piruvato quinasa.
MX2020003954A 2017-10-16 2018-10-16 Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa. MX2020003954A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762573037P 2017-10-16 2017-10-16
PCT/US2018/056136 WO2019079338A1 (en) 2017-10-16 2018-10-16 LENTIVIRAL VECTORS FOR PKLR ADMINISTRATION TO TREAT PYRUVATE KINASE DEFICIENCY

Publications (1)

Publication Number Publication Date
MX2020003954A true MX2020003954A (es) 2020-10-05

Family

ID=66174611

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020003954A MX2020003954A (es) 2017-10-16 2018-10-16 Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa.

Country Status (12)

Country Link
US (2) US11642422B2 (es)
EP (1) EP3697452A4 (es)
JP (2) JP7403461B2 (es)
KR (1) KR20200116076A (es)
CN (1) CN111163810A (es)
AU (1) AU2018352583A1 (es)
BR (1) BR112020007444A2 (es)
CA (1) CA3076253A1 (es)
IL (1) IL273737A (es)
MX (1) MX2020003954A (es)
SG (1) SG11202002263TA (es)
WO (1) WO2019079338A1 (es)

Families Citing this family (7)

* 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
CN110904102A (zh) * 2018-09-18 2020-03-24 中国科学院上海生命科学研究院 一种用于重组蛋白质表达的启动子
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN111733187B (zh) * 2020-08-26 2020-11-06 和元生物技术（上海）股份有限公司 一种wpre突变体病毒载体及其应用
KR102540747B1 (ko) 2020-11-30 2023-06-08 제이케이생명과학 주식회사 음료 제조방법
BR112023023798A2 (pt) * 2021-05-13 2024-01-30 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Ciemat Vetores lentivirais e usos dos mesmos
CN114898802B (zh) * 2022-07-14 2022-09-30 臻和(北京)生物科技有限公司 基于血浆游离dna甲基化测序数据的末端序列频率分布特征确定方法、评价方法及装置

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Also Published As

Publication number Publication date
JP2021500920A (ja) 2021-01-14
SG11202002263TA (en) 2020-04-29
US20210187126A1 (en) 2021-06-24
EP3697452A1 (en) 2020-08-26
US20230355807A1 (en) 2023-11-09
WO2019079338A1 (en) 2019-04-25
KR20200116076A (ko) 2020-10-08
RU2020114948A (ru) 2021-10-28
AU2018352583A1 (en) 2020-04-09
BR112020007444A2 (pt) 2020-10-20
US11642422B2 (en) 2023-05-09
EP3697452A4 (en) 2021-11-24
IL273737A (en) 2020-05-31
JP7403461B2 (ja) 2023-12-22
JP2024037845A (ja) 2024-03-19
CN111163810A (zh) 2020-05-15
CA3076253A1 (en) 2019-04-25
RU2020114948A3 (es) 2021-12-24

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