GeneticsbioRxivPreprint
A humanized knock-in Col6a1 mouse recapitulates a deep-intronic splice-activating variant
Véronique Bolduc, Fady Guirguis, Berit Lubben…·Carsten G. Bönnemann
The paper creates a humanized knock-in mouse model with a deep-intronic splice-activating variant to test antisense therapeutics for Collagen VI-related muscular dystrophies.
1 citation