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
US11286501B2 (en) 2016-04-20 2022-03-29 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas O.A, M.P. Methods of treating or preventing pyruvate kinase deficiency
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
EP4339291A1 (en) * 2021-05-13 2024-03-20 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas O.A., M.P. (CIEMAT) Lentiviral vectors and uses thereof
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

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE123059T1 (de) 1987-09-17 1995-06-15 Massachusetts Inst Technology Menschliche, erythroid-spezifische transkriptionsenhancer.
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)
AU784910B2 (en) 1999-10-12 2006-07-27 Centre National De La Recherche Scientifique Lentiviral triplex DNA, and vectors and recombinant cells containing lentiviral triplex DNA
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 (es) 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
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
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
AU2004233315A1 (en) 2003-04-24 2004-11-04 Fondazione Centro San Raffaele Del Monte Tabor Lentiviral vectors carrying synthetic bi-directional promoters and uses thereof
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
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.
WO2008136670A2 (en) 2007-05-02 2008-11-13 Erasmus University Medical Center Rotterdam Improved methods and means for lentiviral gene delivery
US8747290B2 (en) 2007-12-07 2014-06-10 Miltenyi Biotec Gmbh Centrifuge for separating a sample into at least two components
DE102010003419B4 (de) 2010-03-30 2019-09-12 Evonik Degussa Gmbh Verfahren zur fermentativen Herstellung von L-Ornithin
AU2013243946A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
US20140189896A1 (en) 2012-12-12 2014-07-03 Feng Zhang Crispr-cas component systems, methods and compositions for sequence manipulation
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
CN104020298A (zh) 2014-06-12 2014-09-03 中国科学院化学研究所 自供能量的微管-驱动蛋白运输体系及其制备方法
US10350245B2 (en) 2015-01-21 2019-07-16 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
AU2016348782A1 (en) 2015-11-05 2018-05-31 Cellectis Process of gene-editing of cells isolated from a subject suffering from a metabolic disease affecting the erythroid lineage, cells obtained by said process and uses thereof.
US11286501B2 (en) 2016-04-20 2022-03-29 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas O.A, M.P. Methods of treating or preventing pyruvate kinase deficiency

Also Published As

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

Similar Documents

Publication Publication Date Title
MX2020003954A (es) Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa.
MX2019011040A (es) Composiciones y metodos para potenciar la expresion genica.
CL2020002914A1 (es) Métodos y composiciones para tratar el cáncer
NZ765695A (en) Immune cell organoid co-cultures
NI201900019A (es) Inhibidores de procesos metabólicos celulares
CO2018006133A2 (es) Materiales y métodos para el tratamiento de miopatías basadas en titina y otras titinopatías
AU2017241807A1 (en) Medium, methods, cells and secreted factors for stem cell culture and therapy
MX2020004541A (es) Edicion de genes de celulas primarias.
MX2023006304A (es) Administracion de celulas t dise?adas para tratamiento de canceres en el sistema nervioso central.
MX2019003616A (es) Celulas beta derivadas de celulas madre (sc-beta) y composiciones y metodos para generarlas.
MX2019010735A (es) Sistemas y metodos para la produccion dirigida de una proteina terapeutica dentro de una celula objetivo.
AU2015311681A8 (en) Globin gene therapy for treating hemoglobinopathies
PE20150223A1 (es) Anticuerpos antagonistas mejorados contra factor-8 de crecimiento y diferenciacion y usos de los mismos
CL2015002807A1 (es) Terapia de combinación
MX2015013177A (es) Combinacion de tratamientod de cancer usando microarns e inhibidores de egfr-tki.
NI201400111A (es) Tratamiento del cáncer con inhibidores tor cinasa
BR112019024256A2 (pt) composição de ácido nucleico, método para tratar hemofilia b e para produzir uma partícula de vírus adenoassociado, e, uso de uma composição de ácido nucleico.
BR112014011925A2 (pt) mutações no receptor notch humano e seu uso
MX2023000549A (es) Combinaciones para el tratamiento de neoplasias usando localizacion de celulas quiescentes e inhibidores de egfr.
EP3998341A3 (en) Adenoviral vectors
MX2019012938A (es) Uso del inhibidor mejorador de homologo zeste 2 (ezh2) combinado con el inhibidor tirosina cinasa de bruton (btk) en la preparacion de farmacos para tratar tumores.
BR112021008179A2 (pt) Construtos plasmídicos para tratamento contra câncer e métodos de uso
EA202190474A1 (ru) Терапия на основе микро-рнк, направленная против lcp-1-позитивных раковых опухолей
MX2021013913A (es) Composiciones y metodos para el tratamiento de enfermedades mediadas por atpasa.
UY35439A (es) Formas salinas del (2-(2-(4-fluorofenil)tiazol-4-il)propan-2-il)carbamato de (S)-quinuclidin-3-ilo.