Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …

Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent
and treat various diseases. To function in vivo, mRNA requires safe, effective and stable …

Circular RNAs (circRNAs) are stable and prevalent RNAs in eukaryotic cells that arise from
back-splicing. Synthetic circRNAs and some endogenous circRNAs can encode proteins …

RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

Phenylketonuria (PKU; also known as phenylalanine hydroxylase (PAH) deficiency) is an
autosomal recessive disorder of phenylalanine metabolism, in which especially high …

Due to the high prevalence and long incubation periods often without symptoms, the severe
acute respiratory syndrome coronavirus-2 (SARS-CoV-2) has infected millions of individuals …

Delivery of genetic information to the interior of target cells in vivo has been a major
challenge facing gene therapies. This barrier is now being overcome, owing in part to …

In the last decade, the development of messenger RNA (mRNA) therapeutics by lipid
nanoparticles (LNP) leads to facilitate clinical trial recruitment, which improves the efficacy of …

Nucleic Acid (NA) based therapeutics are poised to disrupt modern medicine and augment
traditional pharmaceutics in a meaningful way. However, a key challenge to advancing NA …

Endosomal escape remains a fundamental barrier hindering the advancement of nucleic
acid therapeutics. Taking inspiration from natural phospholipids that comprise biological …