Gene Therapy Halts Progression of Cerebral Adrenoleukodystrophy in Clinical Trial

This originally appeared on Vector, Boston Children’s Hospital’s blog. Adrenoleukodystrophy — depicted in the 1992 movie “Lorenzo’s Oil” — is a genetic disease that most severely affects boys. Caused by a defective gene on the X chromosome, it triggers a build-up of fatty acids that damage the protective myelin sheaths of the brain’s neurons, leading to cognitive … Read more