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Hope in Translation: Bringing Gene Therapy to the Clinic
In 1987, scientists first uncovered the gene that is the target of mutations that causes Duchenne muscular dystrophy (DMD), a lethal muscle-wasting disease that affects about one in every 5,000 newborn boys. While the discovery of the dystrophin gene sparked hope that scientists could someday replace the mutated gene with a healthy one, turning this concept into a safe and effective treatment has been a decades-long challenge.
In drug discovery, the process of converting early biological insights into a therapy that can be tested in the clinic is known as translational research. It’s rigorous and very often painstaking work that happens behind the scenes, but the nonetheless is a critical bridge to bringing life-transforming treatments to patients. And in the journey of developing a gene therapy for DMD, scientists have had to overcome significant hurdles due to the complexity of the disease and the various challenges of engineering vector delivery systems.
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Jerry Mendell, M.D., Presented Positive Updated Results from the Four Children Dosed in the Gene Therapy Micro-dystr...Sarepta Therapeutics, Inc., a commercial...
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Molecular therapy in a novel translational large animal model for Duchenne muscular dystrophyThe development of efficient and safe st...
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Small Mutation Screening in the DMD Gene by Whole Exome Sequencing of an Argentine Duchenne/Becker Muscular Dystroph...Dystrophinopathies are neuromuscular X-l...
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Gene Editing CRISPR/Cas9 Therapy for Duchenne Muscular Dystrophyhttps://www.youtube.com/watch?v=8xNh1qr4...
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FDA and NIH to loosen oversight of gene therapy developmentCiting the need to safely expedite the d...
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CureDuchenne Ventures Commits $500,000 to 4D Molecular Therapeutics to Advance Next-Generation AAV Delivery of Gene ...CureDuchenne, a national venture phila...
