Cilia-related gene therapy

Cilia-related gene therapy aims to treat disorders caused by defective cilia—tiny hair-like structures on cells vital for functions like movement, sensing, and signaling. By delivering correct copies of faulty genes or editing existing ones, this therapy seeks to restore normal cilia function. It often involves using vectors, such as viruses, to carry the healthy genes into affected cells. This approach offers hope for conditions like ciliopathies, which can involve issues like vision loss, kidney problems, and respiratory issues, by targeting the root genetic causes to improve cellular health and overall function.