For disorders driven by a known genetic component, research understandably focuses on developing a drug targeting that genetic signature. When that works, the outcome benefits patients but the end ...

Between 10 to 15% of all genetic diseases have the same underlying cause: a nonsense mutation. 1 This type of mutation results from a stop codon in the middle of a gene sequence, which terminates ...

Genetic disorders — like cystic fibrosis and Huntington’s disease — are considered incurable, with gene mutations occurring in essentially every cell of the body. Gene mutations occur when one ...