All five participants in a clinical trial of a gene therapy for Angelman syndrome experienced leg weakness, leading sponsors to pause the study.

A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.

Doctors often conflate autism and intellectual disability, and no wonder: The biological distinction between them is murky. Scientific progress depends on knowing where the conditions intersect — and part ways.

The mutation that causes Angelman syndrome makes neurons hyperexcitable, which may explain the frequent seizures that most people with the syndrome have.

Rats missing UBE3A, the gene mutated in people with Angelman syndrome, squeak frequently but tend not to be responsive to the play and squeaks of other rats.

Advances in research and help from families have brought scientists to the brink of an effective therapy for Angelman syndrome.

One form of the protein implicated in Angelman syndrome and autism clusters in the nucleus, and it’s this form that may be critical to brain development.

The timing of treatment is crucial for conditions related to autism — and more so for some conditions than for others.

Researchers are edging closer to a therapy for Angelman syndrome — a condition related to autism — that involves injecting the gene-editing enzyme CRISPR into the fetal brain.

Finding a mutation linked to autism traits can have life-changing consequences for autistic individuals and their families.