The people-focused researcher shares her secrets to carving out time for grant writing, creating work-life balance and letting go of unread emails.

A meeting in Texas reckons with the future of treatment, following two setbacks in 2020.

A new analysis package enables researchers to distill multiple measures of Angelman syndrome mice into a single ‘severity score.’

Roche’s gene therapy drug Rugonersen boosts expression of the protein missing in the syndrome in mice and monkeys, but whether it works in people remains to be seen.

Interim results from the previously paused trial suggest that doses of the experimental gene therapy drug GTX-102 are well tolerated in children with the autism-linked condition.

A deactivated form of the gene editor restores UBE3A expression in mice and human neurons without cutting the genome. It may hold promise for future Angelman gene therapies.

The machine-learning tool, which automatically counts how many marbles a mouse buries, appears to distinguish between hyperactivity and repetitive behaviors.

Model animals don’t develop the usual behavioral and motor problems when reared in an enclosure containing exercise wheels, toys and treats.

How chemical tags called methyl groups position themselves on genetic sequences may hint at some of the causes of autism.

A potential new gene therapy delivered via a single injection mimics ratios of proteins normally found in cells.