Cautious welcome for gene editing of Duchenne muscular dystrophy in animal modelBMJ 2016; 351 doi: https://doi.org/10.1136/bmj.h7033 (Published 04 January 2016) Cite this as: BMJ 2016;351:h7033
- Nigel Hawkes
The gene defect responsible for Duchenne muscular dystrophy has been corrected in a mouse model, raising hopes for a cure for this fatal disease.
The success, published in Science by a team from several US research centres, was greeted as “very exciting indeed” by a UK expert, Peter Braude, emeritus professor of obstetrics and gynaecology at King’s College London. So far, only proof of principle has been established, and more work will be needed before clinical use in human patients could be attempted.
Duchenne muscular dystrophy is the commonest and most severe form of the disease among boys and causes progressive muscle degeneration and weakness. Its cause has long been known to be mutations in a gene carried on the X chromosome that encode the protein dystrophin, vital to …