Neurotherapeutics

, Volume 10, Issue 3, pp 473–485

Recent Advances in RNA Interference Therapeutics for CNS Diseases

  • Pavitra S. Ramachandran
  • Megan S. Keiser
  • Beverly L. Davidson
Article

DOI: 10.1007/s13311-013-0183-8

Cite this article as:
Ramachandran, P.S., Keiser, M.S. & Davidson, B.L. Neurotherapeutics (2013) 10: 473. doi:10.1007/s13311-013-0183-8

Abstract

Over the last decade, RNA interference technology has shown therapeutic promise in rodent models of dominantly inherited brain diseases, including those caused by polyglutamine repeat expansions in the coding region of the affected gene. For some of these diseases, proof-of concept studies in model organisms have transitioned to safety testing in larger animal models, such as the nonhuman primate. Here, we review recent progress on RNA interference-based therapies in various model systems. We also highlight outstanding questions or concerns that have emerged as a result of an improved (and ever advancing) understanding of the technologies employed.

Keywords

RNAiCNSmiRNAshRNAsiRNApolyQ

Supplementary material

13311_2013_183_MOESM1_ESM.pdf (499 kb)
ESM 1(PDF 499 kb)

Copyright information

© The American Society for Experimental NeuroTherapeutics, Inc. 2013

Authors and Affiliations

  • Pavitra S. Ramachandran
    • 1
  • Megan S. Keiser
    • 2
  • Beverly L. Davidson
    • 1
    • 2
    • 3
  1. 1.Interdisciplinary program in GeneticsUniversity of IowaIowa CityUSA
  2. 2.Interdisciplinary program in NeuroscienceUniversity of IowaIowa CityUSA
  3. 3.Department of Internal MedicineUniversity of IowaIowa CityUSA