How Does Gene Therapy Work for Respiratory Diseases?
Updated: Oct 5
Gene therapy treats respiratory diseases by targeting their genetic causes. This approach typically involves delivering therapeutic genes to the airway epithelium, which is crucial for lung function.
Key Mechanisms:
Gene Delivery: Gene therapy uses viral vectors like adeno-associated viruses (AAV) to introduce functional genes into affected lung cells.
Restoring Function: The introduced genes produce essential proteins, such as the CFTR protein in cystic fibrosis, which helps regulate ion transport and mucus clearance.
Research and Development: Ongoing studies aim to improve delivery methods and ensure the safety and effectiveness of gene therapies for respiratory conditions.
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