Selective reduction of toxic RNAs rescue splicing dysregulation across repeat expansion diseases

« The primary goal of the Berglund lab at the RNA Institute is to understand the molecular mechanisms of pre-mRNA splicing and toxic RNA disorders, with a focus on the most common form of adult-onset muscular dystrophy – myotonic dystrophy. We use biochemical, genomic, and cellular approaches to study the fundamental rules that govern RNA processing and how these events are disrupted in disease. Knowledge gained from basic biological studies is applied to developing therapeutic strategies, including small molecules, antisense oligonucleotides, and gene editing, for myotonic dystrophy and related devastating repeat expansion diseases. As a researcher and the Director of the RNA Institute, I work collaboratively with RNA Institute faculty to advance our mission to develop and deliver tools, analytics, and early-stage discoveries necessary for the progression of RNA-based therapeutics and diagnostics. » (J. Andrew Berglund).

https://www.albany.edu/rna/research-rna-institute/berglund-lab