Deletion of exons 45 to 55 in the DMD gene: from the therapeutic perspective to the in vitro model
J. Poyatos-Garcia, P. Soblechero-Martin, A. Liquori, A. Lopez-Martinez, E. Gonzalez-Romero, R. P. Vazquez-Manrique, N. Muelas, G. Garcia-Garcia, J. Ohana, V. Arechavala-Gomeza, J. J. Vilchez
The paper restores dystrophin expression in DMD cells by replicating a specific exon deletion and creates a new cell model to study the mutation's effects.
bioRxiv · Sep 13, 2023 · PreprintCell Biology