Evaluation of oligonucleotides for therapy of Friedreich ataxia

Brunel University London. 22/12/2016. Principal Investigator: Dr Mark Pook, Funding body: RaNA Therapeutics.

The aim of this project is firstly to characterise a novel transgenic mouse model of the multi-system autosomal inherited genetic disorder, Friedreich ataxia (FRDA). This mouse model, designated YG8LR, contains the human frataxin transgene together with an inserted 410 GAA repeat expansion mutation. Our studies aim to characterise the FRDA mouse model at molecular, biochemical, histopathological and behavioural levels. Once characterised, the FRDA mouse model will then be used in preclinical studies to investigate the potential of specific frataxin oligonucleotides generated by RaNA Therapeutics to stabilize frataxin mRNA and hence increase frataxin expression.

 Evaluation of oligonucleotides for therapy of Friedreich ataxia