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Gene therapy for metachromatic leukodystrophy: Lead candidate optimization Gingras, Jacinthe; St Martin, Thia; Gall, Katie; Newman, Jennifer; Selby, Donald E.; Adjei, Samuel; Hanscom, Terrence; Prout, Jaime; Przybylski, Dariusz S.; Zheng, Alice; Avila, Nancy; Lotterhand, Jason; Rivas, Israel; Wright, Teresa; Francone, Omar; Seymour, Albert 分享 收藏
HMI-202: A gene therapy development candidate for metachromatic leukodystrophy (MLD) Gingras, Jacinthe; St-Martin, Thia; Gall, Katie; Seabrook, Tania; Behmoiras, Liana; Lotterhand, Jason; Rivas, Israel; Avila, Nancy; Mercaldi, Michael; Wright, Teresa; Newman, Jennifer; Krupa, Shiva; Francone, Omar; Seymour, Albert 分享 收藏
HMI-202: Investigational gene therapy for treatment of metachromatic leukodystrophy (MLD) Gingras, Jacinthe; St-Martin, Thia; Gall, Katherine; Seabrook, Tania A.; Lotterhand, Jason; Avila, Nancy; Rivas, Jose I.; Mercaldi, Michael; Hennessey, Laura; Wright, Teresa; Krupa, Shiva; Glyman, Steven; Francone, Omar; Seymour, Albert 分享 收藏
Single intravenous dose of AAVHSC15 vector with human phenylalanine hydroxylase transgene results in sustained correction of phenylketonuria in the PAHenu2 mouse model Francone, Omar L.; Ahmed, Seemin S.; Ellsworth, Jeff L.; Faulkner, Deiby; Sengooba, Arnold; Rubin, Hillard; Dollive, Serena; Lamppu, Diana; Wright, Teresa; Seymour, Albert 分享 收藏
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Intrathecal delivery of recombinant human iduronate-2-sulfatase (idursulfase-IT) Calias, P.; Pan, J.; Huang, Y.; Alessandrini, M.; Liu, N.; Pfeifer, R. W.; Wright, T. L.; Felice, B. R.; Powell, J.; Savioli, N.; Lotterhand, J.; Heartlein, M. W. 分享 收藏
Multiple variants in toll-like receptor 4 gene modulate risk of liver fibrosis in Caucasians with chronic hepatitis C infection Li, Yonghong; Chang, Monica; Abar, Olivia; Garcia, Veronica; Rowland, Charles; Catanese, Joseph; Ross, David; Broder, Samuel; Shiffman, Mitchell; Cheung, Ramsey; Wright, Teresa; Friedman, Scott L.; Sninsky, John 分享 收藏
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