RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs),
antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR–Cas9, have …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

Most of the current methods for programmable RNA drug therapies are unsuitable for the
clinic due to low uptake efficiency and high cytotoxicity. Extracellular vesicles (EVs) could …

Syndromic autism spectrum disorders represent a group of childhood neurological
conditions, typically associated with chromosomal abnormalities or mutations in a single …

Angelman syndrome is a devastating neurogenetic disorder for which there is currently no
effective treatment. It is caused by mutations or epimutations affecting the expression or …

Recently, increasing numbers of rare pathogenic genetic variants have been identified that
are associated with variably elevated risks of a range of neurodevelopmental outcomes …

Dysfunction of microRNA (miRNA) metabolism is thought to underlie diseases affecting
motoneurons. One miRNA, miR-218, is abundantly and selectively expressed by developing …

Despite the remarkable number of scientific breakthroughs of the last 100 years, the
treatment of neurodevelopmental disorders (eg, autism spectrum disorder, intellectual …

RNA interference (RNAi) is a fundamental cellular process for the posttranscriptional
regulation of gene expression. RNAi can exogenously be modulated by small RNA …

The common chemical and biological properties of antisense oligonucleotides provide the
opportunity to identify and characterize chemical class effects across species. The chemical …