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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2017-11-01 Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell, W. Dave Arnold, Louise R. Rodino-Klapac, Thomas W. Prior, Linda Lowes, Lindsay Alfano, Katherine Berry, Kathleen Church, John T. Kissel, Sukumar Nagendran, James L’Italien, Douglas M. Sproule, Courtney Wells, Jessica A. Cardenas, Marjet D. Heitzer, Allan Kaspar, Sarah Corcoran, Lyndsey Braun, Shibi Likhite, Carlos Miranda, Kathrin Meyer, K.D. Foust, Arthur H.M. Burghes, Brian K. Kaspar
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2017-11-01 Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell, W. Dave Arnold, Louise R. Rodino-Klapac, Thomas W. Prior, Linda Lowes, Lindsay Alfano, Katherine Berry, Kathleen Church, John T. Kissel, Sukumar Nagendran, James L’Italien, Douglas M. Sproule, Courtney Wells, Jessica A. Cardenas, Marjet D. Heitzer, Allan Kaspar, Sarah Corcoran, Lyndsey Braun, Shibi Likhite, Carlos Miranda, Kathrin Meyer, K.D. Foust, Arthur H.M. Burghes, Brian K. Kaspar
In patients with SMA1, a single intravenous infusion of adeno-associated viral vector containing DNA coding for SMN resulted in longer survival, superior achievement of motor milestones, and better motor function than in historical cohorts. Further studies are necessary to confirm the safety and efficacy of this gene therapy. (Funded by AveXis and others; ClinicalTrials.gov number, NCT02122952.)
中文翻译：
脊髓性肌萎缩症的单剂量基因替代疗法
在SMA1患者中，单次静脉输注含有编码SMN的DNA的腺相关病毒载体，与历史研究组相比，具有更长的生存期，更好的运动里程碑实现和更好的运动功能。需要进一步的研究来证实这种基因疗法的安全性和有效性。（由AveXis等资助； ClinicalTrials.gov编号，NCT02122952。）
更新日期：2017-11-02
中文翻译：
脊髓性肌萎缩症的单剂量基因替代疗法
在SMA1患者中，单次静脉输注含有编码SMN的DNA的腺相关病毒载体，与历史研究组相比，具有更长的生存期，更好的运动里程碑实现和更好的运动功能。需要进一步的研究来证实这种基因疗法的安全性和有效性。（由AveXis等资助； ClinicalTrials.gov编号，NCT02122952。）
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