A humanized knock-in Col6a1 mouse recapitulates a deep-intronic splice-activating variant
Véronique Bolduc, Fady Guirguis, Berit Lubben, Lindsey Trank, Sarah Silverstein, Astrid Brull, Matthew Nalls, Jun Cheng, Lisa Garrett, Carsten G. Bönnemann
bioRxiv 2024.03.21.581572; doi: https://doi.org/10.1101/2024.03.21.581572 New Results