Charles A Thornton
Affiliation: University of Rochester
- Carrell S, Carrell E, Auerbach D, Pandey S, Bennett C, Dirksen R, et al. Dmpk gene deletion or antisense knockdown does not compromise cardiac or skeletal muscle function in mice. Hum Mol Genet. 2016;25:4328-4338 pubmed publisher..Our results support the feasibility and safety of using ASOs for post-transcriptional silencing of DMPK in muscle and heart. ..
- Thornton C, Wang E, Carrell E. Myotonic dystrophy: approach to therapy. Curr Opin Genet Dev. 2017;44:135-140 pubmed publisher..Preclinical data have suggested that RNA dominance is responsive to therapeutic intervention and that DM therapy can be approached at several different levels. Here we review recent efforts to alleviate RNA toxicity in DM. ..