Ontology highlight
ABSTRACT:
SUBMITTER: Pires VB
PROVIDER: S-EPMC5524367 | biostudies-literature | 2017
REPOSITORIES: biostudies-literature
Pires Vanessa Borges VB Simões Ricardo R Mamchaoui Kamel K Carvalho Célia C Carmo-Fonseca Maria M
PloS one 20170724 7
Splice-switching antisense oligonucleotides (SSOs) offer great potential for RNA-targeting therapies, and two SSO drugs have been recently approved for treating Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). Despite promising results, new developments are still needed for more efficient chemistries and delivery systems. Locked nucleic acid (LNA) is a chemically modified nucleic acid that presents several attractive properties, such as high melting temperature when bound to ...[more]