Finding a drug target for Friedreich''s Ataxia

Here, an overview is given on previous research and trial endeavours to treat Friedreich''s ataxia (FRDA), the most common autosomal recessive neurodegenerative disease for which there are currently no effective treatments available. This book describes the search for possible drug targets at the DNA level with an emphasis on epigenetic mechanisms. During detailed investigation of the first part of the Frataxin (FXN) gene, the presence of an RNA pol II transcriptional pausing site was evident. This may represent a crucial site for new drug development approaches and might explain the relatively poor outcomes of current drug approaches to up-regulate this gene. This book should be a valuable source of information for anyone who is interested in FRDA and its molecular mechanisms.