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Behavioral improvement in dystrophic mdx23 mouse following repeated antisense oligonucleotides injections

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by mutations in the DMD gene that disrupt the production of functional dystrophin proteins. Intellectual disability and neurobehavioral complications including autism spectrum disorder, attention-deficit disorders, and anxie...

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Principais autores: Artadokht Aghaeipour, Manuela Mitsogiannis, Claire Fergus, Konstantina Tetorou, Talia Gileadi, Erwina Stojek, Silvia Talavera, Simran Singh, Matthew Siddle, Jack Prendeville, Ewa Sokolowska, Vincent Kelly, Jennifer Morgan, Federica Montanaro, Francesco Muntoni
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2025-12-01
coleção:Molecular Therapy: Nucleic Acids
Assuntos:
MT: Oligonucleotides: therapies and applications
antisense oligonucleotide
Duchenne muscular dystrophy
neuromuscular disease
brain dystrophin
brain comorbidities
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S216225312500304X
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