Two decades of research on RNA interference (RNAi) have transformed a breakthrough
discovery in biology into a robust platform for a new class of medicines that modulate mRNA …

More than 25 years after its discovery, the post-transcriptional gene regulation mechanism
termed RNAi is now transforming pharmaceutical development, proved by the recent FDA …

Over the past decade, non-coding RNA-based therapeutics have proven as a great potential
for the development of targeted therapies for cancer and other diseases. The discovery of …

RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

The first wave of genetically targeted therapies for cancer focused on drugging gene
products that are recurrently mutated in specific cancer types. However, mutational analysis …

Class 2 CRISPR-Cas systems endow microbes with diverse mechanisms for adaptive
immunity. Here, we analyzed prokaryotic genome and metagenome sequences to identify …

Most human epithelial tumors harbor numerous alterations, making it difficult to predict
which genes are required for tumor survival. To systematically identify cancer dependencies …

Ninety-seven percent of drug-indication pairs that are tested in clinical trials in oncology
never advance to receive US Food and Drug Administration approval. While lack of efficacy …

Conversion of glial cells into functional neurons represents a potential therapeutic approach
for replenishing neuronal loss associated with neurodegenerative diseases and brain injury …

A synthetic lethal interaction occurs between two genes when the perturbation of either gene
alone is viable but the perturbation of both genes simultaneously results in the loss of …