Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Spinal muscular atrophy (SMA) is a neurodegenerative disorder caused by mutations in
SMN1 (encoding survival motor neuron protein (SMN)). Reduced expression of SMN leads …

The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Metastasis is a major contributor to cancer-associated deaths. It is characterized by a
multistep process that occurs through the acquisition of molecular and phenotypic changes …

The removal of introns from mRNA precursors and its regulation by alternative splicing are
key for eukaryotic gene expression and cellular function, as evidenced by the numerous …

Antisense technology is now beginning to deliver on its promise to treat diseases by
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …

RNA-binding proteins (RBPs) are critical effectors of gene expression, and as such their
malfunction underlies the origin of many diseases. RBPs can recognize hundreds of …

More than a hundred genes have been identified that, when disrupted, impart large risk for
autism spectrum disorder (ASD). Current knowledge about the encoded proteins—although …

Recent advances in sequencing technologies and collaborative efforts have led to
substantial progress in identifying the genetic causes of amyotrophic lateral sclerosis (ALS) …