Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense oligonucleotides (ASOs) has recently gained recognition as therapeutic approach in DMD. Conjugation of a peptide to the phosphor...

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Detalles Bibliográficos
Autores principales: Tsoumpra, Maria K., Fukumoto, Seiji, Matsumoto, Toshio, Takeda, Shin'ichi, Wood, Matthew J.A., Aoki, Yoshitsugu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6642283/
https://www.ncbi.nlm.nih.gov/pubmed/31257147
http://dx.doi.org/10.1016/j.ebiom.2019.06.036

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6642283/
https://www.ncbi.nlm.nih.gov/pubmed/31257147
http://dx.doi.org/10.1016/j.ebiom.2019.06.036

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