Recently, advances in wearable technologies, data science and machine learning have
begun to transform evidence-based medicine, offering a tantalizing glimpse into a future of …

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 …

Rare diseases are a leading cause of infant mortality and lifelong disability. To improve
outcomes, timely diagnosis and effective treatments are needed. Genomic sequencing has …

Recent advances in the generation, purification and cellular delivery of RNA have enabled
development of RNA-based therapeutics for a broad array of applications. RNA therapeutics …

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

The substantial investments in human genetics and genomics made over the past three
decades were anticipated to result in many innovative therapies. Here we investigate the …

Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder;
however, no disease-modifying interventions are available for patients with this disease. The …

Nucleic acid‐based therapeutics that regulate gene expression have been developed
towards clinical use at a steady pace for several decades, but in recent years the field has …

Tandem repeats represent one of the most abundant class of variations in human genomes,
which are polymorphic by nature and become highly unstable in a length-dependent …

Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the
path for a broader second wave of therapies and laid the foundation for next-generation …