Article
Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinA.
Annals of neurology - 1 Jun 2003
Gonzalez-Alegre Pedro, Miller Victor M, Davidson Beverly L, Paulson Henry L
Abstract excerpt
A three-nucleotide (GAG) deletion in the TOR1A gene is the most common cause of inherited dystonia, DYT1. Because the mutant protein, TorsinA (TA), is thought to act in a dominant manner to cause disease, inhibiting expression from the mutant gene represents a potentially powerful therapeutic strategy. In an effort to develop therapy for this disease, we tested whether small interfering RNA (siRNA) could...
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