Hong Zhen Yin

The paper identifies a nuclear RNA degradation code (NRDC) that recognizes transcripts with both a 5' splice site and a poly(A) junction, leading to their degradation and contributing to disease.

This study shows that ASO-mediated Gys1 knockdown in a mouse model of Pompe disease reduces glycogen accumulation and improves motor function, both alone and in combination with ERT.