Girls and young women with Rett syndrome have less severe anxiety and breathing problems after treatment with trofinetide, an analogue of insulin-like growth factor 1, according to an unpublished placebo-controlled trial.

Excitatory and inhibitory neurons can derive from the same cellular source in the developing human brain, a new study suggests, overturning a 20-year-old hypothesis. Tomasz Nowakowski discusses the finding’s implications for autism research.

Infection during pregnancy can tweak a mouse’s gut microbiome in ways that have lasting effects on her pups’ immune system and increase their chances of gut inflammation, a new study suggests.

A new viral variant can deliver genes exclusively to the brain, overcoming a key hurdle in treating neurological conditions using gene therapy.

In mice with a mutated copy of SHANK3, stress induces social deficits and alters gene expression in certain excitatory neurons. But eliminating a stress-related protein that regulates SHANK3 restores typical social functioning in the animals.

New software can automatically identify and quantify mouse social behavior from videos, saving researchers time.

Deleting a copy of the gene TBX1 depletes the fatty myelin insulation that surrounds neurons and reduces cognitive speed in mice.

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

People with autism have more mutations than others do in both mitochondrial DNA and nuclear DNA that affects mitochondrial function.

Clues that problems with mitochondria contribute to autism have been accumulating for decades. In the past five years, a mutant mouse and a flurry of findings have energized the field.