RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1

@article{Keiser2013RNAiOO,
  title={RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1},
  author={Megan S. Keiser and James C. Geoghegan and Ryan L. Boudreau and Kim A. Lennox and Beverly L. Davidson},
  journal={Neurobiology of Disease},
  year={2013},
  volume={56},
  pages={6-13}
}
Spinocerebellar Ataxia Type 1 (SCA1) is an autosomal dominant late onset neurodegenerative disease caused by an expanded polyglutamine tract in ataxin-1. Here, we compared the protective effects of overexpressing ataxin-1-like using recombinant AAVs, or reducing expression of mutant ataxin-1 using virally delivered RNA interference (RNAi), in a transgenic mouse model of SCA1. For the latter, we used an artificial microRNA (miR) design that optimizes potency, efficacy and safety to suppress… CONTINUE READING

Citations

Publications citing this paper.
Showing 1-10 of 17 extracted citations

Autosomal-dominant cerebellar ataxias.

Handbook of clinical neurology • 2018
View 1 Excerpt

MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy

Molecular therapy : the journal of the American Society of Gene Therapy • 2016

References

Publications referenced by this paper.
Showing 1-10 of 38 references

Spinocerebellar ataxia type 1.

Clinical neuroscience • 1995
View 9 Excerpts
Highly Influenced

Rational design of therapeutic siRNAs: minimizing offtargeting potential to improve the safety of RNAi therapy for Huntington's disease

Boudreau, R.L
Mol. Ther • 2011

Similar Papers

Loading similar papers…