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 …

Glycogen storage diseases (GSDs) are a group of rare, monogenic disorders that share a
defect in the synthesis or breakdown of glycogen. This Primer describes the multi-organ …

With the improvement of gene vectors, the rise of chimeric antigen receptor T cell
immunotherapy and breakthroughs in the genome editing technology, gene therapy had …

The human genetic code encrypted in thousands of genes holds the secret for synthesis of
proteins that drive all biological processes necessary for normal life and death. Though the …

Ischaemic heart disease is a leading cause of death worldwide. Injury to the heart is
followed by loss of the damaged cardiomyocytes, which are replaced with fibrotic scar …

Antisense oligonucleotides (ASOs) represent a new and highly promising class of drugs for
personalized medicine. In the last decade, major chemical developments and improvements …

Type 1 diabetes affects millions of people globally and requires careful management to
avoid serious long-term complications, including heart and kidney disease, stroke, and loss …

The achievements of vaccine research and development bring a hope to our societies that
we may cope with the COVID‐19 pandemic. There are two aspects that should be …

Recent progress in developing programmable nucleases, such as zinc-finger nucleases,
transcription activator–like effector nucleases (TALENs) and clustered regularly interspaced …

Messenger RNAs (mRNAs) present a great potential as therapeutics for the treatment and
prevention of a wide range of human pathologies, allowing for protein replacement …