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基因治療醫(yī)學(xué)

AAV治療脊髓性肌萎縮(SMA)
AAV治療脊髓性肌萎縮(SMA)
Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder characterized by the loss of motor neurons in the spinal cord and brainstem, leading to progressive muscle weakness and atrophy. SMA is caused by mutations in the survival motor n...
Gutless腺病毒治療杜興型肌營養(yǎng)不良(DMD)
Gutless腺病毒治療杜興型肌營養(yǎng)不良(DMD)
Cargo size is key Duchenne Muscular Dystrophy (DMD) is a genetic disease that affects ~1 in every 5,000 people. The disease results in muscle weakness and loss, eventually resulting in death due to a genetic mutation in the dystrophin gene. Dystrophi...
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