More Progress Toward Gene Editing for Kids with Muscular DystrophyPosted on February 26th, 2019 by Dr. Francis CollinsCaption: Muscles of untreated mouse model of Duchenne muscular dystrophy (left) compared to muscles of similar mice one year after gene-editing treatment (right). This “in vivo” approach to gene editing successfully restored production of functional dystrophin proteins, strengthening animals’ muscles within weeks of treatment. It’s important to emphasize that this gene editing research aimed at curing DMD is being done in non-reproductive (somatic) cells, primarily muscle tissue. The NIH does not support the use of gene editing technologies in human embryos or human reproductive (germline) cells, which would change the genetic makeup of future offspring. As such, the Duke researchers’ CRISPR/Cas9 system is designed to work optimally in a range of muscle and muscle-progenitor cells.