Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic Dystrophy

por González-Barriga, Anchel, Mulders, Susan AM, van de Giessen, Jeroen, Hooijer, Jeroen D, Bijl, Suzanne, van Kessel, Ingeborg DG, van Beers, Josee, van Deutekom, Judith CT, Fransen, Jack AM, Wieringa, Bé, Wansink, Derick G
Publicado 2013

CRISPR/Cas9-Induced (CTG⋅CAG)(n) Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

por van Agtmaal, Ellen L., André, Laurène M., Willemse, Marieke, Cumming, Sarah A., van Kessel, Ingeborg D.G., van den Broek, Walther J.A.A., Gourdon, Geneviève, Furling, Denis, Mouly, Vincent, Monckton, Darren G., Wansink, Derick G., Wieringa, Bé
Publicado 2017

Intracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro

por González-Barriga, Anchel, Nillessen, Bram, Kranzen, Julia, van Kessel, Ingeborg D.G., Croes, Huib J.E., Aguilera, Begoña, de Visser, Peter C., Datson, Nicole A., Mulders, Susan A.M., van Deutekom, Judith C.T., Wieringa, Bé, Wansink, Derick G.
Publicado 2017