Gene therapy for hemophilia 由 Peter J. Lenting, Sylvia Fong

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The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice 由 Ashrafali Mohamed Ismail, Evan Witt, Taren Bouwman, Wyatt Clark, Bridget Yates, Matteo Franco, Sylvia Fong

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Adeno-associated virus-based gene therapy for hemophilia–addressing the gaps 由 Wolfgang Miesbach, Paul Batty, Pratima Chowdary, Sylvia Fong, Radoslaw Kaczmarek, Frank W.G. Leebeek, Brian Long, Johnny Mahlangu, Mike Makris, Glenn F. Pierce, Steven W. Pipe, Alok Srivastava, Jan Voorberg, Flora Peyvandi

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Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model. 由 Manuel E Lopez, Daniel Wendt, Roger Lawrence, Kerui Gong, Hoonsan Ong, Bryan Yip, Joseph Chen, Linley Mangini, Britta Handyside, Alexander Giaramita, Aashish Lamichhane, Melanie Lo, Vishal Agrawal, Jeremy Van Vleet, Amanda Abolhesn, Jessica B Felix, Isaac Villalpando, Vikas Bhat, Rolando De Angelis, Yuanbin Ru, Ayesha Khan, Sylvia Fong, Terri Christianson, Sherry Bullens, Brett E Crawford, Stuart Bunting, Mika Aoyagi-Scharber

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