Ap­ply­ing CRISPR to Duchenne, Duke re­searchers spot­light 1-year mouse da­ta in Sarep­ta-spon­sored study

As Sarep­ta hus­tles its gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy in­to a piv­otal study, one of its aca­d­e­m­ic col­lab­o­ra­tors has some longterm mouse da­ta to re­port on an at­tempt to treat the dis­ease through gene edit­ing.

One year af­ter a sin­gle dose of the CRISPR-based treat­ment, the adult mice in the study main­tained a high lev­el of dy­s­trophin ex­pres­sion, ac­cord­ing to re­searchers led by Pro­fes­sor Charles Gers­bach at Duke Uni­ver­si­ty.

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