Dominic Paul Lee

This study develops a gene therapy strategy using snRNAs to correct mis-splicing of STMN2 and UNC13A in TDP-43 proteinopathies, showing restoration of protein levels and axonal regeneration in motor neurons.

The paper shows that CTCF loss in mouse heart cells reorganizes enhancer activity-by-contact scores, reduces CRH numbers, increases CRH size, and increases CRH crossing TAD boundaries.

This study shows that loss of CTCF in mouse cardiomyocytes disrupts gene neighbourhoods, leading to ectopic enhancer-promoter interactions and heart failure.