[go: up one dir, main page]

MX2018012872A - Composiciones y metodos para mejorar la expresion genetica de pklr. - Google Patents

Composiciones y metodos para mejorar la expresion genetica de pklr.

Info

Publication number
MX2018012872A
MX2018012872A MX2018012872A MX2018012872A MX2018012872A MX 2018012872 A MX2018012872 A MX 2018012872A MX 2018012872 A MX2018012872 A MX 2018012872A MX 2018012872 A MX2018012872 A MX 2018012872A MX 2018012872 A MX2018012872 A MX 2018012872A
Authority
MX
Mexico
Prior art keywords
methods
pklr
compositions
improve
genetic expression
Prior art date
Application number
MX2018012872A
Other languages
English (en)
Other versions
MX382997B (es
Inventor
C Segovia Jose
G Gomez Maria
Navarro Susana
Meza Nestor
Bueren Juan
G Bravo Maria
Original Assignee
Centro De Investig Energeticas Medioambientales Y Tecnologicas O A 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 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P filed Critical Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
Publication of MX2018012872A publication Critical patent/MX2018012872A/es
Publication of MX382997B publication Critical patent/MX382997B/es

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
    • 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
    • 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
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/0104—Pyruvate kinase (2.7.1.40)
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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

La presente divulgación proporciona casetes de polinucleótidos, vectores de expresión y métodos para la expresión de un gen en células de mamífero para proporcionar terapia génica para deficiencia de piruvato cinasa.
MX2018012872A 2016-04-20 2017-04-20 Composiciones y metodos para mejorar la expresion genetica de pklr. MX382997B (es)

Applications Claiming Priority (2)

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

Publications (2)

Publication Number Publication Date
MX2018012872A true MX2018012872A (es) 2019-08-22
MX382997B MX382997B (es) 2025-03-13

Family

ID=60116402

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018012872A MX382997B (es) 2016-04-20 2017-04-20 Composiciones y metodos para mejorar la expresion genetica de pklr.

Country Status (17)

Country Link
US (3) US11286501B2 (es)
EP (1) EP3445406B1 (es)
JP (3) JP7045362B2 (es)
KR (3) KR102673203B1 (es)
CN (2) CN116179580A (es)
AU (3) AU2017254665C1 (es)
BR (1) BR112018071695A2 (es)
CA (1) CA3021572A1 (es)
DK (1) DK3445406T3 (es)
ES (1) ES2885833T3 (es)
HU (1) HUE055862T2 (es)
IL (1) IL262459B2 (es)
MX (1) MX382997B (es)
PL (1) PL3445406T3 (es)
PT (1) PT3445406T (es)
SG (1) SG11201809096VA (es)
WO (1) WO2017184903A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017254665C1 (en) 2016-04-20 2021-03-11 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Compositions and methods for enhanced gene expression of PKLR
SG11202002263TA (en) * 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
MX2020010718A (es) * 2018-04-11 2021-01-20 Spacecraft Seven Llc Composiciones y metodos para el trasplante de celulas madre.
US12378576B2 (en) 2018-04-27 2025-08-05 Spacecraft Seven, Llc Gene therapy for CNS degeneration
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector

Family Cites Families (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3853886T2 (de) 1987-09-17 1995-10-05 Massachusetts Inst Technology Menschliche, erythroid-spezifische transkriptionsenhancer.
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
EP0689601B1 (en) 1993-02-22 2006-10-04 The Rockefeller University Production of high titer helper-free retroviruses by transient transfection
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
HU223733B1 (hu) 1993-10-25 2004-12-28 Canji, Inc. Rekombináns adenovírus vektor és eljárás alkalmazására
US5484720A (en) 1994-09-08 1996-01-16 Genentech, Inc. Methods for calcium phosphate transfection
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
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
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6607879B1 (en) 1998-02-09 2003-08-19 Incyte Corporation Compositions for the detection of blood cell and immunological response gene expression
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
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)
CA2382265A1 (en) 1999-08-13 2001-02-22 Taisho Pharmaceutical Co., Ltd. Prostaglandin derivatives
ATE351916T1 (de) 1999-10-12 2007-02-15 Pasteur Institut Lentivirale triplex-dns, und vektoren und rekombinante zellen, die lentivirale triplex-dns enthalten
DE19960113A1 (de) * 1999-12-08 2001-06-13 Wita Proteomics Ag Mittel und Verfahren zur Diagnose von Lyme Borreliose sowie Borreliose-Impfstoff
US20020013294A1 (en) 2000-03-31 2002-01-31 Delong Mitchell Anthony Cosmetic and pharmaceutical compositions and methods using 2-decarboxy-2-phosphinico derivatives
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
DK1412493T3 (da) 2001-08-02 2012-01-09 Inst Clayton De La Rech Fremgangsmåder og sammensætninger der angår forbedrede lentiviral-vektor.-produktionsystemer
NZ532060A (en) 2001-10-02 2005-11-25 Inst Clayton De La Rech Methods and compositions relating to restricted expression lentiviral vectors and their applications
WO2003066086A2 (en) 2002-02-04 2003-08-14 DeveloGen Aktiengesellschaft für entwicklungsbiologische Forschung Proteins involved in the regulation of energy homeostatis
DE60329747D1 (de) 2002-05-02 2009-12-03 Univ Rochester Vektoren mit beiden isoformen von beta-hexosaminidase
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
BE1016793A4 (fr) 2005-10-04 2007-06-05 Artelis Procede de culture de cellules et dispositif permettant sa mise en oeuvre.
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
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
AU2008262478C1 (en) 2007-05-29 2014-06-19 Aldevron, L.L.C. Vectors and methods for genetic immunization
DK2520643T3 (da) 2007-12-07 2020-01-20 Miltenyi Biotec Bv & Co Kg Prøvebehandlingssystemer og -fremgangsmåder
DE102008063606A1 (de) 2008-12-18 2010-06-24 Eufets Ag Ein-Vektor Tet-System und dessen Verwendung
AU2010241864B2 (en) 2009-04-30 2014-02-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
WO2011043719A1 (en) 2009-10-05 2011-04-14 Ya-Fang Mei Replicating viral vectors for gene therapy
DE102010003419B4 (de) 2010-03-30 2019-09-12 Evonik Degussa Gmbh Verfahren zur fermentativen Herstellung von L-Ornithin
US20140377288A1 (en) * 2011-09-16 2014-12-25 The Board Of Regents Of The University Of Texas System Compositions and methods related to dna damage repair
DK3763820T3 (da) 2012-02-29 2022-02-07 Helmholtz Zentrum Muenchen Deutsches Forschungszentrum Gesundheit & Umwelt Gmbh Retroviral transduktion ved hjælp af poloxamerer
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
EP3808844A1 (en) 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2014093444A1 (en) 2012-12-11 2014-06-19 Atmi Packaging, Inc. Recipient for cell cultivation
KR20150105635A (ko) * 2012-12-12 2015-09-17 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 crispr-cas 성분 시스템, 방법 및 조성물
US20160194660A1 (en) 2012-12-21 2016-07-07 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
US9932588B2 (en) 2013-11-15 2018-04-03 Northwestern University Inhibition of oxidative stress in atrial fibrillation
US20170037431A1 (en) 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
US11030531B2 (en) 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
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
GB201417042D0 (en) 2014-09-29 2014-11-12 Fkd Therapies Oy Method
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
WO2016118780A1 (en) 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
MA41506A (fr) 2015-02-13 2017-12-19 Takeda Vaccines Inc Procédés de production de virus pour produire des vaccins
UA128249C2 (uk) 2015-03-10 2024-05-22 Зе Трастіс Оф Коламбія Юніверсіті Ін Зе Сіті Оф Нью-Йорк Рекомбінантна конструкція glut1 аденоасоційованого вірусного вектора і спосіб відновлення експресії glut1 на її основі
WO2017077135A1 (en) 2015-11-05 2017-05-11 Centro De Investigación Biomédica En Red 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.
RU2758489C2 (ru) 2015-11-11 2021-10-28 Пресиджен, Инк. Композиции и способы для экспрессии нескольких биологически активных полипептидов с одного вектора для лечения заболеваний сердца и других патологий
US11065347B2 (en) 2016-01-19 2021-07-20 The Regents Of The University Of California Methods for the treatment of Danon disease and other disorders of autophagy
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
JP6970106B2 (ja) 2016-02-12 2021-11-24 ブルーバード バイオ， インコーポレイテッド Ｖｃｎエンハンサー組成物およびその使用方法
US11261416B2 (en) 2016-04-14 2022-03-01 Trizell Ltd. Fixed-bed bioreactor with constant-flow pump / tubing system
AU2017254665C1 (en) 2016-04-20 2021-03-11 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Compositions and methods for enhanced gene expression of PKLR
US20220323612A1 (en) 2016-06-13 2022-10-13 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
EP3471743B1 (en) 2016-06-17 2025-08-06 Fred Hutchinson Cancer Center Strategies to assess and/or produce cell populations with predictive engraftment potential
CA3035605A1 (en) 2016-09-08 2018-03-15 Fundacion Instituto De Investigacion Sanitaria Fundacion Jimenez Diaz Gene therapy for patients with fanconi anemia
MA47847A (fr) 2016-12-06 2020-01-29 Bluebird Bio Inc Thérapie génique pour traiter la mucopolysaccharidose de type i
MX2019006551A (es) 2016-12-06 2019-10-15 Bluebird Bio Inc Terapia génica para mucopolisacaridosis de tipo ii.
US20180326022A1 (en) 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
US20200199534A1 (en) 2017-04-27 2020-06-25 Fred Hutchinson Cancer Research Center Therapeutic formulations containing cd34+ stem cells derived from negative selection
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
MX2020010718A (es) 2018-04-11 2021-01-20 Spacecraft Seven Llc Composiciones y metodos para el trasplante de celulas madre.
US12378576B2 (en) 2018-04-27 2025-08-05 Spacecraft Seven, Llc Gene therapy for CNS degeneration
WO2020014523A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
CA3106241A1 (en) 2018-07-30 2020-02-06 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Methods for gene modification of hematopoietic cells
WO2020037249A1 (en) 2018-08-16 2020-02-20 Rocket Pharmaceuticals, Ltd. Production methods for viral vectors
KR20210125999A (ko) 2019-02-12 2021-10-19 스페이스크래프트 세븐, 엘엘씨 다논병 치료를 위한 유전자 요법 벡터
AU2020278046A1 (en) 2019-05-23 2022-01-27 Spacecraft Seven, Llc Gene therapy vectors for infantile malignant osteopetrosis
WO2021236981A2 (en) 2020-05-20 2021-11-25 Spacecraft Seven, Llc Engineered parkin and uses thereof
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
CN116113700A (zh) 2020-08-05 2023-05-12 太空飞船七有限责任公司 用于glut1表达的腺相关病毒载体及其用途
JP2023536618A (ja) 2020-08-05 2023-08-28 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
JP2023552443A (ja) 2020-12-07 2023-12-15 スペースクラフト セブン リミテッド ライアビリティ カンパニー ダノン病の治療
US20240335565A1 (en) 2021-12-08 2024-10-10 Spacecraft Seven, Llc Junctophilin-2 (jph2) gene therapy using aav vector
JP2024545507A (ja) 2021-12-10 2024-12-09 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを用いたトロポニンｃ（ｔｎｎｃ１）遺伝子治療
MX2024009685A (es) 2022-02-08 2024-08-19 Spacecraft Seven Llc Vector viral adenoasociado para la expresion de glut1 y usos del mismo.
AU2023258039A1 (en) 2022-04-22 2024-11-21 Spacecraft Seven, Llc B-cell lymphoma 2–associated anthanogene 3 (bag3) gene therapy using aav vector

Also Published As

Publication number Publication date
WO2017184903A1 (en) 2017-10-26
AU2017254665B2 (en) 2020-11-12
KR20220147607A (ko) 2022-11-03
KR20240096679A (ko) 2024-06-26
RU2018140783A3 (es) 2020-08-17
KR102440880B1 (ko) 2022-09-06
IL262459A (en) 2018-12-31
PL3445406T3 (pl) 2022-02-07
IL262459B2 (en) 2025-10-01
US20190284573A1 (en) 2019-09-19
RU2018140783A (ru) 2020-05-20
US12163151B2 (en) 2024-12-10
KR102821903B1 (ko) 2025-06-18
US11286501B2 (en) 2022-03-29
MX382997B (es) 2025-03-13
AU2021200872A1 (en) 2021-03-04
ES2885833T3 (es) 2021-12-15
HUE055862T2 (hu) 2021-12-28
JP2022101543A (ja) 2022-07-06
DK3445406T3 (da) 2021-08-30
AU2021200872B2 (en) 2024-04-04
AU2017254665C1 (en) 2021-03-11
EP3445406A1 (en) 2019-02-27
JP2019514422A (ja) 2019-06-06
IL262459B1 (en) 2025-06-01
AU2024204597A1 (en) 2024-07-18
KR20190053136A (ko) 2019-05-17
JP7522782B2 (ja) 2024-07-25
US20250215454A1 (en) 2025-07-03
EP3445406B1 (en) 2021-05-26
EP3445406A4 (en) 2019-12-25
CA3021572A1 (en) 2017-10-26
CN116179580A (zh) 2023-05-30
CN109641063B (zh) 2022-11-15
JP2024150536A (ja) 2024-10-23
JP7045362B2 (ja) 2022-03-31
PT3445406T (pt) 2021-08-30
BR112018071695A2 (pt) 2019-02-19
CN109641063A (zh) 2019-04-16
US20220177920A1 (en) 2022-06-09
KR102673203B1 (ko) 2024-06-10
AU2017254665A1 (en) 2018-11-22
SG11201809096VA (en) 2018-11-29

Similar Documents

Publication Publication Date Title
MX2019011040A (es) Composiciones y metodos para potenciar la expresion genica.
MX2018012872A (es) Composiciones y metodos para mejorar la expresion genetica de pklr.
MX382223B (es) Regulacion de expresion genica mediada por nucleasa.
MX391155B (es) Metodos y composiciones para modificaciones geneticas dirigidas y metodos de uso.
PH12019501262A1 (en) Modified guide rnas
MX2020004541A (es) Edicion de genes de celulas primarias.
BR112016025519A2 (pt) engenharia genética in vivo com vetores de adenovírus
CY1120291T1 (el) Μεθοδοι και συνθεσεις για κατευθυνομενη απο rna τροποποιηση dna στοχου και για κατευθυνομενη απο rna ρυθμιση της μεταγραφης
MY208943A (en) Taurine supplemented cell culture medium and methods of use
SG10201810150UA (en) Compositions and methods for enhanced gene expression in cone cells
MX2021008143A (es) Metodos para cosechar cultivos de celulas de mamifero.
MX2018001617A (es) Composiciones de crispr-cas9 diseñados y metodos de uso.
NZ726989A (en) Improved t cell compositions
MX2017004890A (es) Metodos y composiciones para la generación o el mantenimiento de células pluripotentes.
MX2020009670A (es) Metodos de cultivo por perfusion y usos de los mismos.
MX2019003616A (es) Celulas beta derivadas de celulas madre (sc-beta) y composiciones y metodos para generarlas.
GB2548740A (en) Methods for generation of podocytes from pluripotent stem cells and cells produced by the same
MX2020003954A (es) Vectores lentivirales para suministro de pklr para el tratamiento de deficiencia de piruvato quinasa.
BR112017018885A2 (pt) método de cultura celular para células-tronco mesenquimais
CA2863600A1 (en) Modulators of acyl-coa lysocardiolipin acyltransferase 1 ( alcat1) and uses thereof
BR112018009732A8 (pt) ?polinucleotídeo, vetor de vírus adeno-associado, partícula de vírus adeno-associado, célula hospedeira, métodos para produzir uma partícula de vírus adeno-associado, para tratar hemofilia a, para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado?
BR112018070293A2 (pt) diferenciação de células-tronco pluripotentes em células do endoderma intestinal do intestino médio
MX2020011470A (es) Metodos de terapia genica.
MX2021001981A (es) Metodos para preparar un poloxamero para usar en medio de cultivo celular.
MX2014003094A (es) Metodos para promover diferenciacion.