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Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice

Antisense oligonucleotide therapy for Duchenne muscular dystrophy has shown great potential in preclinical and clinical trials, but its therapeutic applications are still limited due to inefficient delivery. In this study, we investigated a few polyquaterniums (PQs) with different size and compositi...

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Detalles Bibliográficos
Autores principales: Wang, Mingxing, Wu, Bo, Shah, Sapana N, Lu, Peijuan, Lu, Qilong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Taylor & Francis 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8241187/
https://www.ncbi.nlm.nih.gov/pubmed/28633548
http://dx.doi.org/10.1080/10717544.2017.1337827