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 …

The growing understanding of RNA functions and their crucial roles in diseases promotes
the application of various RNAs to selectively function on hitherto “undruggable” proteins …

Therapeutic targeting of noncoding RNAs (ncRNAs), such as microRNAs (miRNAs) and
long noncoding RNAs (lncRNAs), represents an attractive approach for the treatment of …

Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Huntington disease (HD) is a neurodegenerative disease caused by CAG repeat expansion
in the huntingtin gene (HTT) and involves a complex web of pathogenic mechanisms …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in
1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was …

Short interfering RNAs (siRNA) are a powerful class of genetic medicines whose clinical
translation can be hindered by their suboptimal delivery properties in vivo. Here, we provide …

RNA plays a central role in the expression of all genes. Because any sequence within RNA
can be recognized by complementary base pairing, synthetic oligonucleotides and …

RNAi therapeutics have been growing. Patisiran and givosiran, two siRNA-based drugs,
were approved by the Food and Drug Administration in 2018 and 2019, respectively …