AAV9-hMAOA

AAV9-hMAOA is an adeno-associated virus serotype 9 (AAV9) based gene therapy designed to treat Brunner syndrome, an X-linked recessive neurodevelopmental disorder caused by loss-of-function mutations in the monoamine oxidase A (MAOA) gene. The therapy delivers a functional copy of the human MAOA gene to restore enzymatic activity, which is critical for the metabolism of neurotransmitters such as serotonin and dopamine. In preclinical mouse models, intra-thalamic administration of AAV9-hMAOA has been shown to reduce serotonin accumulation, restore cortical architecture, and modulate synaptic function. The program is being developed by researchers at The Ohio State University and UT Southwestern Medical Center.

Other names

AAV9-MAOA gene therapyAAV-9-MAOA gene therapyAAV 9-MAOA gene therapy