A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
Spectrum: Autism Research News
Tag: Rett syndrome
Mutations in the MECP2 gene, which are associated with autism and Rett syndrome, interfere with a cell’s ability to form droplets of DNA that silence gene expression.
Researchers have used the gene-editing tool CRISPR to disable the MECP2 gene in marmoset embryos. The six such marmosets created seem to recapitulate aspects of Rett syndrome.
A single dose of cannabidiol, a component of marijuana, eases seizures and improves learning and sociability in mice with mutations in an autism gene called CDKL5.
Heart-rate patterns and changes in pupil size may herald Rett syndrome, autism or related conditions.
An experimental leukemia drug and a chemical in black pepper ease breathing and movement problems in a mouse model of Rett syndrome.
Problems with protein-filled parcels called exosomes may contribute to Rett syndrome, a condition related to autism.