Ex vivo treatment with a novel synthetic aminoglycoside NB54 in primary fibroblasts from Rett syndrome patients suppresses MECP2 nonsense mutations.

by Manuela Vecsler, Bruria Ben Zeev, Igor Nudelman, Yair Anikster, Amos J Simon, Ninette Amariglio, Gideon Rechavi, Timor Baasov, Eva Gak
Published 2011-01-01

Attenuation of nonsense-mediated mRNA decay enhances in vivo nonsense suppression.

by Kim M Keeling, Dan Wang, Yanying Dai, Srinivasan Murugesan, Balachandra Chenna, Jeremy Clark, Valery Belakhov, Jeyakumar Kandasamy, Sadanandan E Velu, Timor Baasov, David M Bedwell
Published 2013-01-01