Article
Effect of Readthrough Treatment in Fibroblasts of Patients Affected by Lysosomal Diseases Caused by Premature Termination Codons.
Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics - 1 Oct 2015
Matalonga Leslie, Arias Ángela, Tort Frederic, Ferrer-Cortés Xènia, Garcia-Villoria Judit, Coll Maria Josep, Gort Laura, Ribes Antonia
Abstract excerpt
Aminoglycoside antibiotics, such as gentamicin, may induce premature termination codon (PTC) readthrough and elude the nonsense-mediated mRNA decay mechanism. Because PTCs are frequently involved in lysosomal diseases, readthrough compounds may be useful as potential therapeutic agents. The aim of our study was to identify patients responsive to gentamicin treatment in order to be used as positive controls to...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
