Delicate dosage issues are just one complication of developing gene therapy for neurodevelopmental disorders.

The protein missing in people with fragile X syndrome may activate the expression of a signaling protein dubbed NOS1 during prenatal development of brain regions involved in language and social skills, according to a study published 11 May in Cell.

FMRP, the protein missing in people with fragile X syndrome, localizes in clusters of proteins at neuronal junctions that relay sensory and motor information, according to a study published 23 April in The Journal of Comparative Neurology.

Two new strains of mice carrying different mutations in the SHANK2 gene show similar autism-like behaviors but opposing effects on brain signaling, according to two independent studies published 14 June in Nature.

Amyloid-beta, the small protein that forms plaques in the brains of people with Alzheimer’s disease, is more prevalent in postmortem brains from individuals with autism than in those from controls, according to a study published 2 May in PLoS One.

Seaside Therapeutics, a small biotech, and Swiss pharmaceutical giant Roche have announced a partnership to develop drugs for fragile X syndrome and autism.

Luca Santarelli, head of neuroscience at Roche, explains why he is optimistic that pharmaceutical companies can overcome the obstacles in autism drug development.

Individuals with Cornelia de Lange syndrome, a rare genetic disorder often accompanied by autism, have subtle differences in the nature of their social deficits compared with those who have autism alone, according to a report published 10 April in the Journal of Child Psychology and Psychiatry.

Researchers typically use only one ‘cohort,’ a group of about three dozen mice, for a given set of experiments. When others repeat the experiments with a different set of animals, sometimes the results hold up, and sometimes they don’t.

Preliminary results from a placebo-controlled trial of the antibiotic minocycline in children with fragile X syndrome suggest the drug alleviates some aspects of the disorder, according to research presented Friday at the International Meeting for Autism Research in Toronto.