北海康成-B(01228.HK)披露基因治療脊髓性肌萎縮症藥物研究初步數據
北海康成-B(01228.HK)公布,已在第25屆ASGCT年會上公布來自其與UMass Chan醫學院Horae基因治療中心基因治療研究協議的初步數據。
高光坪實驗室的Jun Xie團隊經研究得出結論:與基準載體scAAV9-CMVen/CB-hSMN1,從內源性hSMN1啟動子表達co-hSMN1的新型第二代scAAV9基因治療在脊髓性肌萎縮症(SMA)小鼠模型中顯示更強的效力、有效性和安全性。該基準載體和目前美國獲批用於治療SMA基因療法的載體類似。
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