A preclinical safety study of adeno-associated viruses (AAVs) for RNA interference (RNAi) for spinocerebellar ataxia type 1 therapy showed toxicity in nonhuman primates but not rodents, due to unexpected AAV inverted terminal repeat transcriptional activity that was mitigated on altering the RNAi expression environment.
- Megan S. Keiser
- Paul T. Ranum
- Beverly L. Davidson
