AAV gene replacement therapy for CCM3

AAV gene replacement therapy for CCM3 is an experimental gene therapy designed to treat familial cerebral cavernous malformations (CCM) caused by mutations in the PDCD10 (CCM3) gene. Developed by researchers at Duke University and the University of Chicago, the therapy utilizes engineered AAV capsids (AAV.cc47 and AAV.cc84) derived from AAV9 to deliver a functional copy of the CCM3 gene to the central nervous system vasculature. By restoring therapeutic levels of the CCM3 protein, the therapy aims to halt the formation and progression of hemorrhagic lesions in the brain and spinal cord, which otherwise predispose patients to stroke and seizures. Preclinical studies in mouse models have demonstrated significant reductions in lesion burden and improved vascular integrity.

Other names

PDCD10 gene therapyPDCD-10 gene therapyPDCD 10 gene therapyCCM3 gene therapyCCM-3 gene therapyCCM 3 gene therapy