MCIDAS gene therapy

MCIDAS gene therapy is an experimental gene replacement approach designed to treat primary ciliary dyskinesia (PCD) caused by mutations in the MCIDAS gene. MCIDAS is a transcriptional co-regulator essential for multiciliogenesis, the process by which respiratory epithelial cells develop multiple motile cilia. The therapy utilizes a viral vector, such as a lentivirus, to deliver a functional copy of the MCIDAS gene into patient-derived cells. In preclinical models using human induced pluripotent stem cell (iPSC)-derived basal cells and nasal epithelial cells, this approach has demonstrated the ability to rescue the multiciliated cell phenotype and restore the expression of markers like FOXJ1 and DNAH5. It is currently in the research and proof-of-concept stage and has not yet entered clinical development.

Other names

MCIDAS viral deliverylentiviral MCIDAS therapyMCIDAS gene replacement therapy