Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia
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.
Published in Scientific reports (17.02.2016)
Published in Scientific reports (17.02.2016)
Get full text
Journal Article
Perturbation of cellular proteostasis networks identifies pathways that modulate precursor and intermediate but not mature levels of frataxin
Nabhan, Joseph F., Gooch, Renea L., Piatnitski Chekler, Eugene L., Pierce, Betsy, Bulawa, Christine E.
Published in Scientific reports (16.12.2015)
Published in Scientific reports (16.12.2015)
Get full text
Journal Article