Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle

Exon skipping is a promising therapeutic strategy for Duchenne muscular dystrophy (DMD), employing morpholino antisense oligonucleotides (PMO-AO) to exclude disruptive exons from the mutant DMD transcript and elicit production of truncated dystrophin protein. Clinical trials for PMO show variable an...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
में प्रकाशित:Nat Commun
मुख्य लेखकों: Novak, James S., Hogarth, Marshall W., Boehler, Jessica F., Nearing, Marie, Vila, Maria C., Heredia, Raul, Fiorillo, Alyson A., Zhang, Aiping, Hathout, Yetrib, Hoffman, Eric P., Jaiswal, Jyoti K., Nagaraju, Kanneboyina, Cirak, Sebahattin, Partridge, Terence A.
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: Nature Publishing Group UK 2017
विषय:
Article
ऑनलाइन पहुंच:https://ncbi.nlm.nih.gov/pmc/articles/PMC5643396/
https://ncbi.nlm.nih.gov/pubmed/29038471
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-017-00924-7
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