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Gene editing emerges as a new therapeutic strategy for Duchenne muscular dystrophy


Advances in gene editing technology have spurred considerable progress towards a treatment for Duchenne muscular dystrophy (DMD).  Although the disease is rare – affecting roughly 1 in 5,000 male births – its consequences are devastating: patients are confined to wheelchairs at an early age and often succumb to heart or respiratory failure in their twenties or thirties.  No treatments are currently available, but three separate labs have used CRISPR/Cas9, the much-publicized DNA editing system, to address the root cause of the disease.  DMD is caused by mutations that alter the function of a protein called dystrophin, which is required for proper muscle function.  It could be beneficial, therefore, to excise the regions of the gene that harbor those harmful mutations.  Encouragingly, mice with DMD that were treated in this manner saw a rise in functional dystrophin levels and alleviation of disease symptoms.