Found 4 datasets matching "duchenne-muscular-dystrophy".
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Recombinant adeno-associated virus (rAAV) vectors mediate long-term gene transfer without any known toxicity. The primary limitation of rAAV has been the small size of the virion (20 nm), which...
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Background Currently molecular diagnostic laboratories focus only on the identification of large deletion and duplication mutations (spanning one exon or more) for Duchenne Muscular...
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AMONDYS 45 (casimersen) is an intravenous solution used to treat Duchenne muscular dystrophy in patients with a specific genetic mutation. It works as an antisense oligonucleotide to increase...
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Background Aside from muscle, brain is also a major expression site for dystrophin, the protein whose abnormal expression is responsible for Duchenne muscular dystrophy. Cognitive...
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