Article
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.
Experimental neurology - 1 Jun 2009
Lombardi Maria Stella, Jaspers Leonie, Spronkmans Christine, Gellera Cinzia, Taroni Franco, Di Maria Emilio, Donato Stefano Di, Kaemmerer William F
Abstract excerpt
Use of RNA interference to reduce huntingtin protein (htt) expression in affected brain regions may provide an effective treatment for Huntington disease (HD), but it remains uncertain whether suppression of both wild-type and mutant alleles in a heterozygous patient will provide more benefit than harm. Previous research has shown suppression of just the mutant allele is achievable using siRNA targeted to regions...
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