Yinghua Jing

Friedreich's ataxia (FRDA) is the most common autosomal recessive ataxia, typically characterized by progressive motor incoordination, dysarthria, and cardiomyopathy.1-4 FRDA is mainly caused by the GAA trinucleotide repeat expansions in the first intron of the frataxin gene,5, 6 with the number of GAA repeats being closely associated with disease onset and progression.2 The deterioration of ataxia symptoms could be reliably monitored using the Scale for the Assessment and Rating of Ataxia...

1 Introduction Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder caused by a cytosine-adenine-guanine (CAG) repeat expansion in the Huntingtin gene (HTT) [1]. Accumulated toxicity of the mutant HTT (mHTT) protein leads to cellular degeneration of neurons in the brain (especially in the striatum), thereby progressively impairing the motor, cognitive, and psychiatric abilities of patients leading to death [2].