AAV9-NEXMIF

AAV9-NEXMIF is an adeno-associated virus serotype 9 (AAV9)-mediated gene replacement therapy designed to treat X-Linked Intellectual Developmental Disorder-98 (XLID98). XLID98 is a neurodevelopmental syndrome caused by mutations in the Neurite Extension and Migration Factor (NEXMIF) gene, which results in intellectual disability, autism spectrum disorders, and drug-resistant epilepsy. The therapy utilizes an AAV9 vector to deliver a functional copy of the NEXMIF transgene to the central nervous system. Preclinical studies in mouse models have shown that intracerebroventricular administration of the vector can rescue cognitive impairments, reduce epileptic activity, and achieve widespread biodistribution in the nervous system and peripheral organs. The program is currently in preclinical development at Queen's University.

Other names

AAV9-mediated NEXMIF gene replacement therapyAAV-9-mediated NEXMIF gene replacement therapyAAV 9-mediated NEXMIF gene replacement therapy