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Cystic Fibrosis: Cystic Fibrosis Center
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Hutchinson Center research on cystic fibrosis largely focuses on developing effective treatments that are based on the delivery of healthy copies of CF genes into patients, whose cells possess two abnormal copies of the gene. This approach, known as gene therapy, often involves engineering harmless viruses to serve as delivery agents, or vectors, of the healthy gene. Hutchinson Center researchers helped to pioneer this method, which led to the first successful applications of gene therapy in humans.
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This comprehensive revision of the well-received first edition provides everything that a clinician treating patients with cystic fibrosis will need in a single volume. A substantial amount of new information has accumulated during the last five years, ranging from advances in the basic scientific understanding of the condition, through novel treatment options to improvements in the delivery of care both in the hospital and the home. To reflect these developments, new chapters have been added on diagnostic genotype/phenotype correlations, screening, reproductive issues, surgery, quality of life and health economics, while other areas such as the organization of home care have been expanded. Edited by two senior physicians from one of the largest cystic fibrosis centers in the world (London's Brompton Hospital) and with increased input from leading authorities in North America, Europe and Australia presenting a truly international perspective, and will establish the book as the essential reference in the field.
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In the case of cystic fibrosis, a modified virus based on an adeno-associated virus was discovered to be a potentially effective vector for delivery of healthy genes to the airway cells involved in cystic fibrosis. A particular type of adeno-associated virus identified at the Hutchinson Center works particularly well to transfer genes to mouse lung, and researchers are currently working to extend this work to treatment of human patients.
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