AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action.

Myotonic Dystrophy / genetics Humans Trinucleotide Repeat Expansion MicroRNAs / genetics RNA-Binding Proteins / genetics Myotonin-Protein Kinase / genetics Myoblasts / metabolism Gene Expression Regulation Antagomirs / pharmacology Cells, Cultured

Journal

Science advances
ISSN: 2375-2548
Titre abrégé: Sci Adv
Pays: United States
ID NLM: 101653440

Informations de publication

Date de publication:
11 Oct 2024
Historique:
medline: 9 10 2024
pubmed: 9 10 2024
entrez: 9 10 2024
Statut: ppublish

Résumé

This study evaluated therapeutic antimiRs in primary myoblasts from patients with myotonic dystrophy type 1 (DM1). DM1 results from unstable CTG repeat expansions in the

Identifiants

DOI: 10.1126/sciadv.adn6525 PMID: 39383229
pubmed: 39383229
doi: 10.1126/sciadv.adn6525
doi:

Substances chimiques

MicroRNAs 0
RNA-Binding Proteins 0
MBNL1 protein, human 0
Myotonin-Protein Kinase EC 2.7.11.1
DMPK protein, human 0
MIRN23a microRNA, human 0
Antagomirs 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

eadn6525

Auteurs

Estefanía Cerro-Herreros (E)

Human Translational Genomics Group. University Research Institute for Biotechnology and Biomedicine (BIOTECMED), Universidad de Valencia, 46100 Burjassot, Valencia, Spain.
INCLIVA Biomedical Research Institute, Avenue Menéndez Pelayo 4 acc, 46010 Valencia, Spain.
ARTHEx Biotech, Parque Científico de la Universidad de Valencia. Calle del Catedrático Agustín Escardino Benlloch, 9, 46980 Paterna, Valencia, Spain.
ORCID: 0000-0003-3247-5900

Judit Núñez-Manchón (J)

Group of REsearch Neuromuscular of BAdalona (GRENBA), Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol (IGTP), Campus Can Ruti, Universitat Autònoma de Barcelona, 08916 Badalona, Spain.

Neia Naldaiz-Gastesi (N)

Neuromuscular Diseases Group, Neurosciences Area, Biodonostia Health Research Institute, 20014 Donostia/San Sebastián, Spain.
CIBERNED, Carlos III Institute, Spanish Ministry of Science and Innovation, Madrid, Spain.
ORCID: 0000-0003-0901-8429

Marc Carrascosa-Sàez (M)

ARTHEx Biotech, Parque Científico de la Universidad de Valencia. Calle del Catedrático Agustín Escardino Benlloch, 9, 46980 Paterna, Valencia, Spain.
ORCID: 0000-0002-5477-8323

Andrea García-Rey (A)

Human Translational Genomics Group. University Research Institute for Biotechnology and Biomedicine (BIOTECMED), Universidad de Valencia, 46100 Burjassot, Valencia, Spain.
INCLIVA Biomedical Research Institute, Avenue Menéndez Pelayo 4 acc, 46010 Valencia, Spain.

Diego Piqueras Losilla (DP)

ARTHEx Biotech, Parque Científico de la Universidad de Valencia. Calle del Catedrático Agustín Escardino Benlloch, 9, 46980 Paterna, Valencia, Spain.
ORCID: 0009-0003-2667-234X

Irene González-Martínez (I)

Human Translational Genomics Group. University Research Institute for Biotechnology and Biomedicine (BIOTECMED), Universidad de Valencia, 46100 Burjassot, Valencia, Spain.
INCLIVA Biomedical Research Institute, Avenue Menéndez Pelayo 4 acc, 46010 Valencia, Spain.
Centre for Biomedical Network Research on Rare Diseases (CIBERER), CB23/07/00005, Carlos III Health Institute, 28029 Madrid, Spain.
ORCID: 0000-0002-3507-5227

Jorge Espinosa-Espinosa (J)

Human Translational Genomics Group. University Research Institute for Biotechnology and Biomedicine (BIOTECMED), Universidad de Valencia, 46100 Burjassot, Valencia, Spain.
INCLIVA Biomedical Research Institute, Avenue Menéndez Pelayo 4 acc, 46010 Valencia, Spain.
Centre for Biomedical Network Research on Rare Diseases (CIBERER), CB23/07/00005, Carlos III Health Institute, 28029 Madrid, Spain.
Experimental and Applied Biomedicine Research Group, Health Sciences Faculty, Universidad Particular Internacional SEK (UISEK), Quito 170302, Ecuador.
ORCID: 0000-0003-0098-5201

Kevin Moreno (K)

ARTHEx Biotech, Parque Científico de la Universidad de Valencia. Calle del Catedrático Agustín Escardino Benlloch, 9, 46980 Paterna, Valencia, Spain.

Javier Poyatos-García (J)

Neuromuscular and Ataxias Research Group, Health Research Institute Hospital La Fe (IIS La Fe), Valencia, Spain.
Centre for Biomedical Network Research on Rare Diseases (CIBERER), U763, CB06/05/0091, Madrid, Spain.
ORCID: 0000-0003-3117-4743

Juan J Vilchez (JJ)

Neuromuscular and Ataxias Research Group, Health Research Institute Hospital La Fe (IIS La Fe), Valencia, Spain.
Centre for Biomedical Network Research on Rare Diseases (CIBERER), U763, CB06/05/0091, Madrid, Spain.
Neuromuscular Referral Center, European Reference Network on Rare Neuromuscular Diseases (ERN EURO-NMD), Universitary and Polytechnic La Fe Hospital, Valencia, Spain.
ORCID: 0000-0002-0532-2872

Adolfo López de Munain (AL)

Neuromuscular Diseases Group, Neurosciences Area, Biodonostia Health Research Institute, 20014 Donostia/San Sebastián, Spain.
CIBERNED, Carlos III Institute, Spanish Ministry of Science and Innovation, Madrid, Spain.
Department of Neurology, Donostialdea Integrated Health Organization, Osakidetza Basque Health Service, 20014 Donostia/San Sebastián, Spain.
Department of Neurosciences, Faculty of Medicine and Nursery, University of the Basque Country UPV-EHU, 20014 Donostia/San Sebastián, Spain.

Mònica Suelves (M)

Group of REsearch Neuromuscular of BAdalona (GRENBA), Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol (IGTP), Campus Can Ruti, Universitat Autònoma de Barcelona, 08916 Badalona, Spain.
ORCID: 0000-0001-7095-5804

Gisela Nogales-Gadea (G)

Group of REsearch Neuromuscular of BAdalona (GRENBA), Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol (IGTP), Campus Can Ruti, Universitat Autònoma de Barcelona, 08916 Badalona, Spain.
ORCID: 0000-0002-7414-212X

Beatriz Llamusí (B)

ARTHEx Biotech, Parque Científico de la Universidad de Valencia. Calle del Catedrático Agustín Escardino Benlloch, 9, 46980 Paterna, Valencia, Spain.

Rubén Artero (R)

Human Translational Genomics Group. University Research Institute for Biotechnology and Biomedicine (BIOTECMED), Universidad de Valencia, 46100 Burjassot, Valencia, Spain.
INCLIVA Biomedical Research Institute, Avenue Menéndez Pelayo 4 acc, 46010 Valencia, Spain.
Centre for Biomedical Network Research on Rare Diseases (CIBERER), CB23/07/00005, Carlos III Health Institute, 28029 Madrid, Spain.
ORCID: 0000-0003-1596-047X

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female

Evaluation of Low-Value Services Across Major Medicare Advantage Insurers and Traditional Medicare.

Ciara Duggan, Adam L Beckman, Ishani Ganguli et al.
1.00
Humans United States Aged Cross-Sectional Studies Medicare Part C

Effectiveness of Virtual Yoga for Chronic Low Back Pain: A Randomized Clinical Trial.

Hallie Tankha, Devyn Gaskins, Amanda Shallcross et al.
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH

questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Dystrophies musculaires › Dystrophie myotonique › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Phénomènes génétiques › Mutagenèse › Expansion de séquence répétée de l'ADN › Expansion de trinucléotide répété › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Acides nucléiques › ARN › ARN non traduit › Petit ARN non traduit › microARN › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Nucléoprotéines › Protéines de liaison à l'ARN › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines et peptides de signalisation intracellulaire › Protein-Serine-Threonine Kinases › Myotonin-protein kinase › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Cellules › Cellules souches › Myoblastes › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Nucléotides › Polynucléotides › Oligonucléotides › Antagomirs › AntimiR treatment corrects myotonic dystrophy...
questionsmedicales.fr › Cellules › Cellules cultivées › AntimiR treatment corrects myotonic dystrophy...