Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinA.

@article{GonzalezAlegre2003TowardTF,
  title={Toward therapy for DYT1 dystonia: allele-specific silencing of mutant TorsinA.},
  author={Pedro Gonzalez-Alegre and Victor M. Miller and Beverly L. Davidson and Henry L. Paulson},
  journal={Annals of neurology},
  year={2003},
  volume={53 6},
  pages={781-7}
}
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 selectively silence expression of mutant TA. Exploiting the three-base pair difference… CONTINUE READING
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