Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2′OMethyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice

Duchenne muscular dystrophy (DMD) is a fatal disorder characterised by progressive muscle wasting. It is caused by mutations in the dystrophin gene, which disrupt the open reading frame leading to the loss of functional dystrophin protein in muscle fibres. Antisense oligonucleotide (AON)-mediated sk...

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Detalles Bibliográficos
Autores principales: Georgiadou, Michaella, Christou, Melina, Sokratous, Kleitos, Wengel, Jesper, Michailidou, Kyriaki, Kyriacou, Kyriacos, Koutsoulidou, Andrie, Mastroyiannopoulos, Nikolaos P., Phylactou, Leonidas A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8622172/
https://www.ncbi.nlm.nih.gov/pubmed/34832896
http://dx.doi.org/10.3390/ph14111113

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8622172/
https://www.ncbi.nlm.nih.gov/pubmed/34832896
http://dx.doi.org/10.3390/ph14111113

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