Single-dose base editing corrects PKD1 mutation and extends survival in ADPKD preclinical models

Mayo Clinic researchers have developed a promising gene-editing therapy that directly corrects a genetic mutation responsible ...

Mutation-specific defects in neurological disorders mapped, pointing toward personalized therapies

Patients with CaV2.1 channelopathies face severe and often debilitating symptoms, such as seizures, migraines, tremors, and ...