Single CRISPR treatment provides long-term benefits in mice | Duke University

1These images highlight the stark contrast between mouse models of untreated Duchenne muscular dystrophy (left) and those treated with a CRISPR-based genetic therapy after one year (right). On the right, green indicates a higher level of dystrophin gene expression. The dark spots in the inset show T cells from these mice responding to the bacterial Cas9 protein, indicating the presence of an immune response to the therapy. Credit: Charles Gersbach, Duke University

Feb. 18, 2019 (MedicalXpress) -- Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.

The study appears online Feb. 18 in the journal Nature Medicine.