Allele-specific silencing of dominant disease genes.

  title={Allele-specific silencing of dominant disease genes.},
  author={Victor M. Miller and Haibin Xia and Ginger L Marrs and Cynthia M. Gouvion and Gloria Lee and Beverly L. Davidson and Henry L. Paulson},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  volume={100 12},
Small interfering RNA (siRNA) holds therapeutic promise for silencing dominantly acting disease genes, particularly if mutant alleles can be targeted selectively. In mammalian cell models we demonstrate that allele-specific silencing of disease genes with siRNA can be achieved by targeting either a linked single-nucleotide polymorphism (SNP) or the disease mutation directly. For a polyglutamine neurodegenerative disorder in which we first determined that selective targeting of the disease… CONTINUE READING
Highly Influential
This paper has highly influenced 15 other papers. REVIEW HIGHLY INFLUENTIAL CITATIONS
171 Citations
2 References
Similar Papers


Publications citing this paper.
Showing 1-10 of 171 extracted citations


Publications referenced by this paper.
Showing 1-2 of 2 references cgi doi

  • K. Tanemura, M. Murayama, +5 authors A. Takashima
  • J. Neurosci. 22,
  • 2002

Allele-specific silencing of Tau in cells simulating the heterozygous state

  • Miller
  • PNAS June
  • 2000

Similar Papers

Loading similar papers…