Stem cells for cystic fibrosis research

UK researchers have developed a human embryonic stem cell (ES cell) line which carries the genetic defect underlying cystic fibrosis (CF). The scientists, based at King's College London, say the cell line will be useful for studying how the CF gene mutation affects cells, as well as for testing out gene therapy and other new treatments. Speaking at the British Association for the Advancement of Science Festival of Science last week, team leader Stephen Minger said there was 'a significant shortage' of high quality human ES cell lines. 'It is essential for the development of human stem cell technology, and the larger goal of cellular replacement therapy for human disease, that additional human cell lines are generated', he added.

Cystic fibrosis is a serious inherited lung disorder, which affects people who inherit two faulty CFTR genes. The King's College team isolated the ES cells from an embryo left over from PGD (preimplantation genetic diagnosis) testing for the disease. The cells have two copies of the delta-508 CFTR gene mutation, which is the most common mutation that causes the disease. The scientists are now trying to grow ES cells affected by other genetic conditions, such as Huntington's disease. ES cells are the body's 'master cells', which are capable of growing into any type of body tissue. Researchers hope that studying isolated cells affected by genetic disorders will help them understand how the symptoms of the condition develop in the body.

Minger, whose group announced their success in growing the UK's first human embryo stem cell line in August 2003, predicts that the pace of stem cell research is set to accelerate. 'The number of research groups using stem cells in the UK has already grown from a handful a few years ago to now about 60. The input of Government money and enlightened regulation processes will ensure that this work continues', he said in a press release. Earlier this year, scientists at the Reproductive Genetics Institute in Chicago announced that they had developed 12 new ES stem cell lines each affected by one of several genetic disorders, but not CF. However, none of the new cell lines can be used by federally funded ES cell researchers in the US, who are only permitted to use ES cell-lines created before 9 August 2001.