Encontrado 1 documento, a visualizar página 1 de 1

Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a ...

Recent studies have demonstrated that RNAi is a promising approach for treating autosomal dominant disorders. However, discrimination between wild-type and mutant transcripts is essential, to preserve wild-type expression and function. A single nucleotide polymorphism (SNP) is present in more than 70% of patients with Machado-Joseph disease (MJD). We investigated whether this SNP could be used to inactivate mut...