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Hexose Potentiates Peptide-Conjugated Morpholino Oligomer Efficacy in Cardiac Muscles of Dystrophic Mice in an Age-Dependent Manner

Insufficient delivery of oligonucleotides to muscle and heart remains a barrier for clinical implementation of antisense oligonucleotide (AO)-mediated exon-skipping therapeutics in Duchenne muscular dystrophy (DMD), a lethal monogenic disorder caused by frame-disrupting mutations in the DMD gene. We...

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Detalles Bibliográficos
Autores principales: Han, Gang, Gu, Ben, Lin, Caorui, Ning, Hanhan, Song, Jun, Gao, Xianjun, Moulton, Hong M., Yin, HaiFang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6807288/
https://www.ncbi.nlm.nih.gov/pubmed/31629961
http://dx.doi.org/10.1016/j.omtn.2019.09.012