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ris Allen was diagnosed with cystic fibrosis ANSWER: Aerosol delivery of the CFTR gene. Both K(CF) shortly after birth. Genetic analysis re- viruses and liposome DNA complexes are capable vealed that he had the most common form of dys- of successful CFTR gene transfer to the nasal and function of the CF transmembrane conductance airway epithelia of patients with CF. In fact, gene regulator gene (CFTR) leading to faulty processing transfer to the airways is one of the few areas where and protein trafficking. His therapy to date has con- liposome DNA complexes match the expression sisted of palliative treatments, such as daily physio- obtained using viral vectors without the viruses in- therapy to improve chest and lung function, pancre- flammatory side effects. Current trials are aimed at atic enzyme replacement, and a high calorie diet. optimizing gene delivery with reduced toxicity to Conventional treatment of his recurrent pulmonary produce sustained correction of the epithelial trans- disease is less and less effective, and he is interested port defect. in gene therapy. What would be a logical strategy for this patient? [ Pobierz całość w formacie PDF ] |