AR126660A1 - Mejorando la eficacia de la terapia génica dirigida a músculos - Google Patents

Mejorando la eficacia de la terapia génica dirigida a músculos

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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
English (en)
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/es

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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)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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.
ARP220102000A 2021-07-28 2022-07-27 Mejorando la eficacia de la terapia génica dirigida a músculos AR126660A1 (es)

Applications Claiming Priority (1)

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

Publications (1)

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

Family

ID=77398387

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220102000A AR126660A1 (es) 2021-07-28 2022-07-27 Mejorando la eficacia de la terapia génica dirigida a músculos

Country Status (3)

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

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3240616A1 (en) * 2021-12-10 2023-06-15 Christopher Dean HERZOG Troponin c (tnnc1) gene therapy using aav vector
CN116926125B (zh) * 2023-09-07 2024-06-11 昆明理工大学 一种抑制炎症与基因编辑同时进行的基因载体

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
DK2282764T3 (da) 2008-04-22 2019-10-14 Vib Vzw Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf
WO2011051450A1 (en) 2009-10-29 2011-05-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
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
EP3800261A1 (en) 2014-01-21 2021-04-07 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
JP6879486B2 (ja) 2015-03-17 2021-06-02 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ Ｆｖｉｉｉおよびｆｉｘ用の最適化された肝臓特異的発現系
JP2020515572A (ja) 2017-03-27 2020-05-28 ブレイエ・ユニバージテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｔｅｉｔ Ｂｒｕｓｓｅｌ 横隔膜特異的核酸調節エレメントならびにその方法および使用
CN111936621A (zh) * 2018-02-05 2020-11-13 奥登茨治疗公司 转录调控元件和它的用途
KR102624831B1 (ko) * 2018-10-31 2024-01-16 서울대학교산학협력단 근육 특이적 pck1 과발현 형질전환 개 생산

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

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