An encouraging report on a study using RNA-targeted therapies in both ALS and SCA2
Scientists report a significant step toward combatting two degenerative brain diseases. A targeted therapy developed by scientists at University of Utah Health slows the progression of Spinocerebellar Ataxia type 2 (SCA2) in a mouse model of this condition. In a parallel collaborative study, led by researchers at Stanford University, a nearly identical treatment improves the health of mice that model Amyotrophic Lateral Sclerosis (ALS). You can read the press release here.
Scientists use gene editing technique to restore production of frataxin protein in mice
Researchers have reported in a recent publication encouraging results about a study where they have successfully corrected a mutated frataxin gene in a mouse model of FA, a model which Dr Mark Pook created with Ataxia UK’s funding. You can find more information and the publication here.
A recent publication is reporting a potential new therapeutic target for Friedreich’s ataxia
RNF126 depletion results in frataxin accumulation in cells derived from FRDA patients, highlighting the relevance of RNF126 as a new therapeutic target for Friedreich’s ataxia. A combined therapy aimed to increasing frataxin levels, either by promoting its gene transcription or through gene therapy or protein replacement, and simultaneously interfering with its RNF126-mediated degradation could be envisioned. You can read the full article here.