Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

This publication has 48 references indexed in Scilit:

• Rational Design of Therapeutic siRNAs: Minimizing Off-targeting Potential to Improve the Safety of RNAi Therapy for Huntington's Disease
  Molecular Therapy, 2011
• Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates
  Molecular Therapy, 2011
• Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
  Molecular Therapy, 2010
• Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates
  Molecular Therapy, 2010
• A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells
  Molecular Therapy, 2009
• Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice
  Molecular Therapy, 2009
• Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients
  Current Biology, 2009
• Sustained effects of nonallele‐specific Huntingtin silencing
  Annals of Neurology, 2009
• Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
  Proceedings of the National Academy of Sciences, 2008
• MicroRNAs: small RNAs with a big role in gene regulation
  Nature Reviews Genetics, 2004