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Yiran Wang
BSc PhD
MNDA Junior Research Fellow
Exploring RNA regulation and therapeutics in amyotrophic lateral sclerosis
Research Summary
My research focuses on aberrant RNA and protein dysregulation driving neurodegeneration in amyotrophic lateral sclerosis (ALS), a devastating condition marked by progressive loss of motor neurons. Current therapies offer only modest benefits, but recent evidence implicates disruptions in RNA metabolism and protein homeostasis as key contributors to ALS pathology. Nucleic acid–based therapies, including antisense oligonucleotides (ASOs), have been developed to address these mechanisms and show promising potential for treatment.
In particular, I explore an RNA alternative splicing event, called intron retention, which occurs in ALS-associated genes and leads to downstream RNA-binding protein dysfunction. I use induced pluripotent stem cells (iPSCs), disease-relevant cellular assays, and splicing-modulating ASOs and CRISPRs to uncover the mechanisms of neurodegeneration and explore potential therapeutic strategies for ALS.
My research is supported by the Motor Neurone Disease Association.