Courtney Young, 32, was a senior in high school when her two-year-old nephew, Christopher, was diagnosed with Duchenne muscular dystrophy, a fatal genetic disorder. The diagnosis rocked Young’s family and sent her on a journey to find a cure. Her recent work with gene editing offers one of the most promising advances in decades.
Muscular dystrophy results from a mutation that prevents the body from producing proteins needed to create and sustain healthy muscle. Using CRISPR-Cas9, Young and her team at MyoGene Bio can change a patient’s DNA, restoring the ability to create the necessary proteins.
Young and her team can target a commonly mutated portion of a gene and remove it, after which the DNA can repair itself naturally. Although CRISPR-Cas9 has been used to address genetic mutations for a decade, Young’s research has pushed the boundaries, proving that far larger deletions are possible than previously thought.
“Current approved therapies for Duchenne muscular dystrophy address downstream side effects or offer only modest benefits,” says Young. “Our approach targets the underlying cause of the disease.”
Young envisions packaging CRISPR in a harmless virus that can be injected into the bloodstream. The virus could then infiltrate the muscle cells, allowing the editing technology to do its work on the patient’s DNA. Young thinks clinical trials could be as near as two years away.
“The clock is ticking,” she says. “Once approved, this therapy could help up to 10,000 new Duchenne patients every year.”
