Test therapy

The cystic fibrosis thread focussed on the development of gene therapy and clinical trials now underway under the direction of Professor Eric Alton of the UK Cystic Fibrosis Gene Therapy Consortium . These gene therapy trials use lipid spheres to transport working copies of the CFTR gene – defective in cystic fibrosis – to the lungs of patients, and the particular lipid formulation used in these trials, known as GL67A was selected after careful evaluation against other candidates, first in CF mice and then in sheep (1). Mice models of cystic fibrosis have helped researchers to understand more about the disease and to assess therapies , but until very recently research has been hampered by the lack of a large animal model of cystic fibrosis that models the lung pathology of cystic fibrosis. This situation finally changed in 2008 when scientists at the Universities of Iowa and Missouri produced genetically modified pigs that lack the CFTR gene and develop all the pathologies that are characteristic of cystic fibrosis in humans. This new animal model for cystic fibrosis will be very useful for evaluating the safety and efficiency of new gene therapy techniques as the science advances.

Test therapy

test therapy

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