Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in ...

How can cells be programmed to respond to complex signals and make targeted decisions, similar to a logical circuit in a computer? Researchers have developed a new approach: an RNA-based genetic ...

Chief Executive Officer Joe Payne told investors at the Leerink Partners Global Healthcare Conference that the company has advanced its inhaled messenger RNA cystic fibrosis (CF) program through three ...

The COVID-19 pandemic brought messenger RNA (mRNA) vaccines to the forefront of global health care. After their clinical ...

Researchers engineered the first RNA-based NAND gate in living cells using deep learning and Bayesian optimization, testing ...

Christine Dunham is a leading expert on the ribosome—an elaborate macro-molecular machine that operates like a factory within ...

A team from University of Toronto Engineering is the first to synthesize long noncoding RNA (lncRNA) outside the cell—a new approach to drug discovery that has already yielded some promising ...

Messenger RNA therapy restores sperm production, enabling healthy offspring in a mouse model of genetic male infertilityResearchers in Japan have demonstrated that targeted delivery of messenger ...

Some genetic mutations that are expected to completely stop a gene from working surprisingly cause only mild or even no symptoms. Researchers in ...

Nobel laureate Tom Cech will address political leaders, CEOs and tech pioneers from around the globe at the World Economic Forum (WEF) annual meeting in Davos, Switzerland this week. His message: RNA ...