Pharmaco-behavioral profiling identifies suppressors of autism gene-associated phenotypes in zebrafish - PubMed
3 hours ago
- #zebrafish
- #pharmacological screen
- #autism spectrum disorder
- Pharmaco-behavioral profiling identifies suppressors of autism gene-associated phenotypes in zebrafish.
- A database and open-source website of behavioral signatures of 520 FDA-approved drugs was established using high-throughput assays in larval zebrafish.
- Behavioral profiles of 9 large-effect ASD gene mutants identified pharmacological mechanisms that anticorrelate with subgroups of ASD genes.
- Estropipate and paclitaxel were identified as top suppressors in scn1lab and dyrk1a mutants, respectively.
- Levocarnitine (LEVO) was found to suppress behavioral phenotypes in both mutants and rescued brain activity deficits and dysregulated lipid metabolic pathways.
- The study demonstrates conservation of drug rescue across systems, including human pluripotent stem cell-derived neurons.
- A provisional patent application was submitted for methods identifying compounds for ASD treatment.