Die Blockierung von Stickstoffmonoxid, einem häufigen Gehirngas, kehrt autismusähnliche Merkmale bei Mäusen um. Die Behandlung menschlicher Nervenzellen mit Stickoxidblockern führte zu einem ähnlichen Ergebnis. Darüber hinaus enthielten Proben von autistischen Kindern viel geringere Mengen des TSC2-Bremsproteins, das Stickoxid blockiert.

Blocking a common brain gas reverses autism-like traits in mice

A newly discovered biological chain reaction explains how high levels of a common brain chemical can lead to cellular overdrive in autism spectrum disorder. By tracing how nitric oxide disables a protective protein to accelerate cell growth pathways, researchers have identified a specific target that might one day yield new therapies. The findings were recently published in the journal Molecular Psychiatry.

The researchers also wanted to prove that the specific nitric oxide attachment point on the TSC2 protein was the root of the issue. They used a genetic technique to alter the brake protein in a way that prevented nitric oxide from attaching to it. They then injected this modified protein into the prefrontal cortex of the mutant mice.

This tiny genetic edit successfully protected the brake protein from being destroyed by nitric oxide. Consequently, the cell growth pathway returned to normal. The mice also became more social and spent more time exploring the open arms of the elevated maze.

To expand their research beyond animal models, the scientists grew human nerve cells in the laboratory. They engineered these human cells to carry the Shank3 genetic mutation. Just like the mouse models, these human cells showed a loss of the TSC2 brake protein and an overactive growth pathway.

Treating these human nerve cells with the nitric oxide blocker produced a familiar result. The drug protected the brake protein and calmed the cellular overdrive. This confirmed that the nitric oxide mechanism operates similarly in human tissues.

Finally, the researchers looked for this same pattern in actual patients. They analyzed blood plasma samples from autistic children alongside samples from neurotypical children. Some of the autistic children had specific Shank3 genetic mutations, while others had autism with no known genetic cause.

The human blood tests mirrored the laboratory experiments perfectly. The samples from the autistic children contained much lower levels of the TSC2 brake protein. Their blood also showed clear signs of an overactive mTOR growth pathway.

For those interested, here’s the link to the peer reviewed journal article:

https://www.nature.com/articles/s41380-026-03514-6