Perturbation of cellular proteostasis networks identifies pathways that modulate precursor and intermediate but not mature levels of frataxin

Por Nabhan, Joseph F., Gooch, Renea L., Piatnitski Chekler, Eugene L., Pierce, Betsy, Bulawa, Christine E.
Publicado no Sci Rep (2015)

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

Por 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.
Publicado no Sci Rep (2016)