Using a molecule designed to overcome a roadblock formed by a common type of genetic flaw, researchers have made progress towards novel molecular treatments for Friedreich’s ataxia — a rare but fatal disorder — in the laboratory dish and in animals.
Using a molecule designed to overcome a roadblock formed by a common type of genetic flaw, researchers have made progress towards novel molecular treatments for Friedreich’s ataxia — a rare but fatal disorder — in the laboratory dish and in animals.