Intravenous gene therapy improves lifespan and clinical outcomes in feline Sandhoff Disease
静脉基因疗法可延长猫桑德霍夫病患者的寿命并改善其临床疗效。
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doi:10.1101/2024.11.15.623838
Maguire, Anne S; Ta, Linh; Gross, Amanda L; Osterhoudt, Devin E; Cannon, Jessica S; Hall, Paige I; Sandey, Maninder; Seyfried, Thomas N; Gray-Edwards, Heather L; Sena-Esteves, Miguel; Martin, Douglas R