One drug blocks mTOR signaling, and the other stops the blocker from acting anywhere in the body but the brain, lowering the potential for side effects.
Spectrum: Autism Research News
Tag: fragile X syndrome
After a brain transplant of reprogrammed human cells, the animals can for the first time recapitulate some neuronal changes seen in people with fragile X syndrome.
People with the autism-linked syndrome lack a protein implicated in several cancers, but it’s unclear whether — or how — they are protected from malignancies.
Non-neuronal brain cells called astrocytes secrete proteins that seem to hamper the growth of neurons in people with autism-related syndromes. These proteins could be new drug targets, Allen says.
A 341-repeat mutation from a person with fragile X does not lead to the syndrome’s traits or function the same way in mice, highlighting a need for different animal models.
An overabundance of ribosomes drives an imbalance of proteins produced from long and short genetic transcripts in a mouse model of fragile X syndrome.
Long cast in supporting roles in the brain, astrocytes are now emerging as primary players in certain characteristics of autism and related conditions.
Swiss biotech Stalicla hopes to bring precision medicine to autism. Experts praise efforts to identify autism subgroups, but evidence to support the company’s claims has yet to be seen.