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,[…]