基因治療醫學(xué)
AAV治療脊髓性肌萎縮(SMA)
Spinal muscular atroph弟聽y (SMA) is a rare genetic neuro紙醫muscular disorder c業女haracterized by the los鄉就s of motor neuro術體ns in the spinal cord and brainstem, 友雜leading to progressive muscle weaknes土校s and atrophy. SMA is caused by mut讀物ations in the survival 就習motor n...
Gutless腺病毒治療杜興型肌營養不良能師(DMD)
Cargo size is key
Duchenne商對 Muscular Dystrophy (DMD) is a geneti們放c disease that affects 花了~1 in every 5,000 people. Th作河e disease results in muscle weakness海舊 and loss, even鐵自tually resulting制民 in death due to a genetic資著 mutation in the dystro熱答phin gene. Dystrophi...
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