Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia

In Friedreich’s ataxia (FRDA) patients, diminished frataxin (FXN) in sensory neurons is thought to yield the predominant pathology associated with disease. In this study, we demonstrate successful usage of RNA transcript therapy (RTT) as an exogenous human FXN supplementation strategy in vitro and i...

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Vydáno v:Sci Rep
Hlavní autoři: Nabhan, Joseph F., Wood, Kristy M., Rao, Varada P., Morin, Jeffrey, Bhamidipaty, Surya, LaBranche, Timothy P., Gooch, Renea L., Bozal, Fazli, Bulawa, Christine E., Guild, Braydon C.
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2016
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4756688/
https://ncbi.nlm.nih.gov/pubmed/26883577
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep20019
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