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Found 2 dataset(s) matching "duchenne-muscular-dystrophy".

Size does matter: overcoming the adeno-associated virus packaging limit
- Organization: U.S. Department of Health & Human Services
- Updated: 2025-09-06 | Harvested: September 06, 2025 at 09:42 AM
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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Detection of mutations in the dystrophin gene via automated DHPLC screening and direct sequencing
- Organization: U.S. Department of Health & Human Services
- Updated: 2025-09-06 | Harvested: September 06, 2025 at 10:28 AM
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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