Scientists in Madrid have successfully treated mice with a form of Dyskeratosis Congenita (TERT gene mutations) and lung fibrosis with gene therapy targeted at the lungs.
All mice treated with the gene therapy showed improvement and some were completely cured of fibrosis after 7 weeks. In contrast, mice treated with a sham gene therapy still had severe lung fibrosis.
These encouraging results follow on from studies showing that gene therapy can treat aplastic anaemia in mice with DC.
Many more studies are required before this gene therapy could be available as a treatment for humans. However, the type of gene therapy used has already proved very successful in men with haemophilia, another genetic condition. A promising start!
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