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Long-Term Morpholino Oligomers in Hexose Elicits Long-Lasting Therapeutic Improvements in mdx Mice

Approval of antisense oligonucleotide eteplirsen highlights the promise of exon-skipping therapeutics for Duchenne muscular dystrophy patients. However, the limited efficacy of eteplirsen underscores the importance to improve systemic delivery and efficacy. Recently, we demonstrated that a glucose a...

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Detalles Bibliográficos
Autores principales: Han, Gang, Lin, Caorui, Ning, Hanhan, Gao, Xianjun, Yin, HaiFang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6070676/
https://www.ncbi.nlm.nih.gov/pubmed/30195785
http://dx.doi.org/10.1016/j.omtn.2018.06.005