AR126660A1 - IMPROVING THE EFFICACY OF MUSCLE-TARGETED GENE THERAPY - Google Patents

IMPROVING THE EFFICACY OF MUSCLE-TARGETED GENE THERAPY

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Publication number
AR126660A1
AR126660A1 ARP220102000A ARP220102000A AR126660A1 AR 126660 A1 AR126660 A1 AR 126660A1 AR P220102000 A ARP220102000 A AR P220102000A AR P220102000 A ARP220102000 A AR P220102000A AR 126660 A1 AR126660 A1 AR 126660A1
Authority
AR
Argentina
Prior art keywords
sequences
nucleic acid
dph
cre
muscle
Prior art date
Application number
ARP220102000A
Other languages
Spanish (es)
Inventor
Thierry Vandendriessche
Lay Khim Chuah
Original Assignee
Univ Brussel Vrije
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 Univ Brussel Vrije filed Critical Univ Brussel Vrije
Publication of AR126660A1 publication Critical patent/AR126660A1/en

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    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente invención se relaciona con elementos regulatorios de ácidos nucleicos que son capaces de aumentar la expresión de genes específica para el diafragma, en particular la expresión en el diafragma propiamente dicha, o en combinación con la expresión en músculo cardíaco y/o en músculo esquelético, métodos que emplean estos elementos regulatorios y usos de estos elementos. Se divulgan también casetes de expresión y vectores que contienen estos elementos regulatorios de ácidos nucleicos. La presente invención es particularmente útil para aplicaciones que usan terapia génica, más particularmente terapia génica dirigida a músculo y/o tejido cardíaco, y para propósitos de vacunación. Reivindicación 1: Un casete de expresión de ácido nucleico que comprende una combinación de los siguientes 4 elementos cis-regulatorios (CREs): Dph-CRE-05, Dph-CRE-02, Dph-CRE-04 y Dph-CRE-06, definidos respectivamente por las secuencias de ácido nucleico de SEQ ID Nº 9 a 12, o por secuencias que tienen al menos un 95%, 96%, 97%, 98% o 99%, de identidad con dichas secuencias, el complemento de dichas secuencias, o secuencias que hibridan en condiciones estrictas con los ácidos nucleicos definidos por dichas secuencias, unidas operativamente a un promotor específico del diafragma, del corazón y/o del músculo esquelético. Reivindicación 12: Un vector que comprende el casete de expresión de ácido nucleico de acuerdo con una cualquiera de las reivindicaciones 1 a 11. Reivindicación 15: Una composición farmacéutica que comprende el casete de expresión de ácido nucleico de acuerdo con una cualquiera de las reivindicaciones 1 a 11, o el vector de acuerdo con una cualquiera de las reivindicaciones 12 a 14, y un portador farmacéuticamente aceptable.The present invention relates to nucleic acid regulatory elements that are capable of increasing diaphragm-specific gene expression, in particular expression in the diaphragm itself, or in combination with expression in cardiac muscle and/or skeletal muscle. , methods that employ these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly gene therapy targeting muscle and/or cardiac tissue, and for vaccination purposes. Claim 1: A nucleic acid expression cassette comprising a combination of the following 4 cis-regulatory elements (CREs): Dph-CRE-05, Dph-CRE-02, Dph-CRE-04 and Dph-CRE-06, defined respectively by the nucleic acid sequences of SEQ ID NO: 9 to 12, or by sequences having at least 95%, 96%, 97%, 98% or 99% identity with said sequences, the complement of said sequences , or sequences that hybridize under strict conditions with the nucleic acids defined by said sequences, operatively linked to a specific promoter of the diaphragm, heart and/or skeletal muscle. Claim 12: A vector comprising the nucleic acid expression cassette according to any one of claims 1 to 11. Claim 15: A pharmaceutical composition comprising the nucleic acid expression cassette according to any one of claims 1 to 11, or the vector according to any one of claims 12 to 14, and a pharmaceutically acceptable carrier.

ARP220102000A 2021-07-28 2022-07-27 IMPROVING THE EFFICACY OF MUSCLE-TARGETED GENE THERAPY AR126660A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP21188306 2021-07-28

Publications (1)

Publication Number Publication Date
AR126660A1 true AR126660A1 (en) 2023-11-01

Family

ID=77398387

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220102000A AR126660A1 (en) 2021-07-28 2022-07-27 IMPROVING THE EFFICACY OF MUSCLE-TARGETED GENE THERAPY

Country Status (3)

Country Link
AR (1) AR126660A1 (en)
TW (1) TW202310880A (en)
WO (1) WO2023006890A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023108129A1 (en) * 2021-12-10 2023-06-15 Spacecraft Seven, Llc Troponin c (tnnc1) gene therapy using aav vector
CN116926125B (en) * 2023-09-07 2024-06-11 昆明理工大学 Gene vector for inhibiting inflammation and gene editing simultaneously

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009071679A1 (en) 2007-12-05 2009-06-11 Vib Vzw Novel aav vector and uses thereof
WO2009130208A1 (en) 2008-04-22 2009-10-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US9353164B2 (en) 2009-10-29 2016-05-31 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
CA2888931C (en) 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
WO2014063753A1 (en) 2012-10-26 2014-05-01 Vrije Universiteit Brussel Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof
SG11201605906UA (en) 2014-01-21 2016-08-30 Univ Bruxelles Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2016146757A1 (en) 2015-03-17 2016-09-22 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
EP3600445A1 (en) 2017-03-27 2020-02-05 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
MA51753A (en) * 2018-02-05 2020-12-16 Audentes Therapeutics Inc ELEMENTS OF TRANSCRIPTION REGULATION AND ASSOCIATED USES
WO2020091471A1 (en) * 2018-10-31 2020-05-07 서울대학교산학협력단 Production of transgenic dog overexpressing muscle-specific pck1

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Publication number Publication date
TW202310880A (en) 2023-03-16
WO2023006890A1 (en) 2023-02-02

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