Mitochondrial DNA (mtDNA) editing has emerged as a revolutionary approach in the fight against neurodegenerative diseases (NDDs).

Mitochondrial DNA (mtDNA) editing has emerged as a revolutionary approach in the fight against neurodegenerative diseases (NDDs). As these diseases ...

PTC518 is designed to reduce levels of the mutated huntingtin protein that leads to injury and death of neurons, which results in Huntington's disease progression. It has emerged from the same ...

Mitochondrial DNA (mtDNA) editing has emerged as a revolutionary approach in the fight against neurodegenerative diseases (NDDs).

At least 370 genetic disorders are known to derive ... by modulating alternative splicing of the SMN2 exon which is mutated in the disease. This is however "laborious, slow and costly", according ...

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...

An Orange County woman is opening up about what it’s like to live with a fatal, rare disease that’s been compared to having ALS, Parkinson’s and Alzheimer’s all at once. Carrie Bryson, a ...

Huntington’s disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It only affects about one in every 10,000 people and, so far, does not have a ...