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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Taylor & Francis
2017
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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 |