The modulation of RNA splicing by small molecules has emerged as a promising strategy for treating pathogenic infections, human genetic diseases, and cancer; however, the principles by which splicing ...

Rapid advances in sequencing technologies have transformed our ability to diagnose human genetic disorders, yet many patients still lack a molecular ...

As a PhD student at the University of Münster in the early 2000s, Sven Diederichs would bike three kilometers each morning to a compact lab in a brick building on the hospital campus. There, he’d ...

Merck KGaA has swooped into a neurological-focused pact with RNA biotech Skyhawk Therapeutics that could top out at $2 billion. Under the agreement, Skyhawk will use its RNA splicing platform, dubbed ...