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收藏Phase 1/2 study update of an AAV9-based gene therapy for Gaucher disease type 2 (PROVIDE trial)
Neuhaus, Sarah; Lewis, Travis B.; Tamburri, Paul A.; Whitley, Chester B.; Rajan, Deepa; Gallagher, Renata C.; Jones, Simon; Donald, Aimee; Escolar, Maria L.; Beckerman, Yael; Mahoney, Erin; Hatch, Daniel A.; Shaughnessy, Lee; Sondergaard, Patricia; Uspenskaya, Olga; Sevigny, Jeffrey
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