A new approach for delivering gene therapy to the brain reduces nerve damage in primates and could help make gene therapies for conditions related to autism safer.
Spectrum: Autism Research News
Tag: Rett syndrome
Mutations in the autism-linked gene DNMT3A lead to the behaviors and gene-expression changes seen in different neurodevelopmental conditions.
Female mice missing a copy of the autism-linked gene MECP2 in a specific set of inhibitory neurons have a hard time heeding pups’ calls and herding litters.
Treatments for Rett syndrome that aim to boost levels of the protein MECP2 may not help everyone with the condition.
Our staff picks the stories, podcasts and special reports that stood out from the rest this past year.
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
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.
This year’s hot topics in autism research center around brain organoids, heart rate, the gut microbiome, treatment timing and early detection.
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.