Cystic fibrosis (CF) is a genetic disorder caused by mutations in the CFTR gene, leading to the production of thick, sticky mucus that can obstruct airways and lead to severe respiratory and digestive issues. Gene therapy aims to address the root cause of CF by delivering a functional copy of the CFTR gene into the affected cells.
Key Approaches:
Viral Vectors: One of the most common methods involves using viral vectors, such as adeno-associated viruses (AAV), to deliver the corrected CFTR gene directly into the lungs. This allows for the production of normal CFTR protein, which is essential for regulating salt and water balance in epithelial cells.
Lipid Nanoparticles: Another promising approach is the use of lipid-based delivery systems, which encapsulate the genetic material and facilitate its uptake by target cells. This method has shown potential in improving the efficiency of gene delivery and expression.
Ongoing Research: Numerous clinical trials are underway to refine these delivery methods and enhance targeting to specific organs affected by CF, particularly the lungs. Researchers are also exploring combination therapies that may include gene therapy alongside other treatment modalities to provide a more comprehensive approach to managing CF.
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