Angelman syndrome
Rare brain disorder—Angelman syndrome—could be treated with gene therapy in the womb
Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains ...
Gene therapy for autism? Rare Angelman syndrome could answer key questions
[T]he success of genetic therapies [for autism] is not guaranteed. Even if disrupted genes can be replaced, repaired or otherwise ...