Gene therapy study starts for bone marrow failure in dyskeratosis congenita (DC)

Dr Kasiani Myers, from Cincinnati has treated the first patient with DC-related bone marrow failure, with an experimental new gene therapy (EXG34217). The trial is in its very early, safety testing stages but we hope that the treatment will help treat bone marrow failure. The trial is open to adults (18 or older) with DC[…]

Scientists cure DC mice with gene therapy

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[…]

Gene-editing breakthrough – what does this mean for DC?

Photo credit: The Times   The Times reports that a pioneering gene-editing treatment is likely to have been successful in treating a little girl suffering from leukaemia. This follows an article last year, which first reported on the procedure.   Two-year old Layla could potentially be the first person in the world to have been saved[…]

Gene therapy for dyskeratosis congenita? Scientists report first success for dyskeratosis congenita mice

Spanish scientists have reported successful gene therapy treatment for mice with dyskeratosis congenita-like problems. Aplastic anaemia (very low blood counts due to bone marrow failure) was reversed by introduction of a gene therapy virus, which delivered a top up of the TERT gene. TERT gene abnormalities cause dyskeratosis congenita in both mice and humans. Interestingly,[…]