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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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UT researcher, with CureDuchenne support, launches company to treat DMD using CRISPR/Cas9 technologyEric Olson, a molecular biologist, has s...
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Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular...Pfizer Inc. has initiated a Phase 1b cli...
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Development of a CRISPR-based gene therapy approach targeting the large exon 45-exon 55 mutation hotspot in DMD geneGene replacement and exon skipping thera...
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Gene Therapy OverviewIn this brief document, we want to expla...
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Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyThe primary objective of this study is t...