H. L. True

Allele-selective knockdown of mutant DNAJB6 improves molecular features in a primary mouse myotube model of LGMDD1

This study uses allele-selective knockdown to reduce mutant DNAJB6 in a mouse model of LGMDD1, improving molecular features associated with the disease.

Disruption of the nascent polypeptide-associated complex leads to reduced polyglutamine aggregation and toxicity

This study shows that disrupting the NAC reduces polyglutamine aggregation and toxicity in cells.