Precision medicine through antisense oligonucleotide-mediated exon skipping
Clinical implementation of two recently approved antisense RNA therapeutics–Exondys51®
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
Optimization of 2′, 4′-BNA/LNA-Based Oligonucleotides for Splicing Modulation In Vitro
T Shimo, S Obika - Exon Skipping and Inclusion Therapies: Methods and …, 2018 - Springer
Antisense oligonucleotide-mediated splicing modulation is an attractive strategy for treating
genetic disorders. In 2016, two splice-switching oligonucleotides (SSOs) were approved by …
genetic disorders. In 2016, two splice-switching oligonucleotides (SSOs) were approved by …