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...

Descripción completa

Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4756688/
https://www.ncbi.nlm.nih.gov/pubmed/26883577
http://dx.doi.org/10.1038/srep20019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4756688/
https://www.ncbi.nlm.nih.gov/pubmed/26883577
http://dx.doi.org/10.1038/srep20019

Ejemplares similares