Exon Skipping Therapies May Be Effective for Some Mutations

Exon Skipping Therapies May Be Effective for Some Mutations

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Exon Skipping Therapies May Be Effective for Some Mutations

Using exon skipping therapies may be an easy, affordable, and effective approach for people with epidermolysis bullosa (EB) with amenable mutations, an early study suggests. Such therapies would promote the production of a slightly shorter, but working version of the skin-related protein that is missing in people with EB, researchers said. This conclusion was based on the fact that this type of therapy effectively skipped the mutated portion, or exon, of the disease-causing gene and allowed the…

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