Gene therapy success raises hopes for approach in Rett syndrome
Success in a small clinical trial of spinal muscular atrophy suggests gene therapy might treat single-gene conditions related to autism.
Success in a small clinical trial of spinal muscular atrophy suggests gene therapy might treat single-gene conditions related to autism.
Watch the complete replay of this journal club, which featured two papers that explore therapies for Rett syndrome.
A candidate drug in clinical trials for Huntington’s disease reverses gait problems in a mouse model of Rett syndrome.
A virus that delivers a working copy of the Rett syndrome gene to the brain improves features of the condition in mice, and appears to be safe in monkeys.
Mutations in the Rett syndrome gene MECP2 have different effects on subtypes of neurons.
Exposure to certain types of light at night may exacerbate sleep issues among people with autism.
Delivering a fragment of the Rett syndrome gene MECP2 into neurons eases features of the syndrome in a mouse model.
Drugs that block certain brain enzymes could help treat two conditions associated with autism.
Mutations in MECP2, the gene mutated in Rett syndrome, may alter the proportions and activity of sensory neurons in rats.
People with autism aren’t easily surprised, the social camouflage some girls and women with autism use may preclude diagnosis, and autism-related genes are rooted deep in human ancestry.