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Case Study Cystic Fibrosis and Gene Therapy

Kris Allen was diagnosed with cystic fibrosis (CF) shortly after birth. Genetic analysis revealed that he had the most common form of dysfunction of the CF transmembrane conductance regulator gene (CFTR) leading to faulty processing and protein trafficking. His therapy to date has consisted of palliative treatments, such as daily physiotherapy to improve chest and lung function, pancreatic enzyme replacement, and a high calorie diet. Conventional treatment of his recurrent pulmonary disease is less and less effective, and he is interested in gene therapy. What would be a logical strategy for this patient?

Answer: Aerosol delivery of the CFTR gene. Both viruses and liposome-DNA complexes are capable of successful CFTR gene transfer to the nasal and airway epithelia of patients with CF. In fact, gene transfer to the airways is one of the few areas where liposome-DNA complexes match the expression obtained using viral vectors without the viruses' inflammatory side effects. Current trials are aimed at optimizing gene delivery with reduced toxicity to produce sustained correction of the epithelial transport defect.

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