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- [1] Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype[J]. BLOOD, 2010, 115 (26) : 5329 - 5337van Til, Niek P.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsStok, Merel论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsKaya, Fatima S. F. Aerts论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlandsde Waard, Monique C.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Expt Cardiol, Dept Cardiol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsFarahbakhshian, Elnaz论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsVisser, Trudi P.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsKroos, Marian A.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Clin Genet, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsJacobs, Edwin H.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Cell Biol & Genet, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsWillart, Monique A.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Pulm Med, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlandsvan der Wegen, Pascal论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Cell Biol & Genet, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsScholte, Bob J.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Cell Biol & Genet, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsLambrecht, Bart N.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Pulm Med, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsDuncker, Dirk J.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Expt Cardiol, Dept Cardiol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlandsvan der Ploeg, Ans T.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Med Ctr Sophia, Dept Pediat, Div Metab Dis & Genet, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsReuser, Arnold J. J.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Clin Genet, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsVerstegen, Monique M.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, NetherlandsWagemaker, Gerard论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, NL-3015 GE Rotterdam, Netherlands
- [2] LENTIVIRAL GENE THERAPY OF HEMATOPOIETIC STEM CELLS RESULTS IN PHENOTYPE CORRECTION IN A MOUSE MODEL OF POMPE DISEASE[J]. HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 2008, 93 : 169 - 169Stok, M.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, Netherlandsvan Til, N.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsKaya, F. S. F. Aerts论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsVisser, T. P.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsKroos, M.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, Netherlandsde Waard, M.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsJacobs, E.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsWillart, M.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, Netherlandsvan der Wegen, P.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsScholte, B.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsLambrecht, B.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsDuncker, D.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, Netherlandsvan der Ploeg, A.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsReuser, A.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsVerstegen, M.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, NetherlandsWagemaker, G.论文数: 0 引用数: 0 h-index: 0机构: Erasmus MC, Rotterdam, Netherlands Erasmus MC, Rotterdam, Netherlands
- [3] Lentiviral gene therapy of hematopoietic stem cells results in phenotype correction in a mouse model of Pompe disease[J]. HUMAN GENE THERAPY, 2007, 18 (10) : 987 - 987Stok, Merel论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsVan der Wegen, Pascal论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsDe Waard, Monique论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsDuncker, Dirk论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsReuser, Arnold论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsVan der Ploeg, Ans论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsScholte, Bob J.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsVan Til, Niek论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsWagemaker, Gerard论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, NetherlandsVerstegen, Monique M. A.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Rotterdam, Netherlands
- [4] Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease[J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 : 1014 - 1025Stok, Merel论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Pediat, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Ctr Lysosomal & Metab Dis, Rotterdam, Netherlands ProPharma Grp, Leiden, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlandsde Boer, Helen论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Clin Genet, Mol Stem Cell Biol, Rotterdam, Netherlands Merus NV, Utrecht, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsHuston, Marshall W.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Mol Genet, Rotterdam, Netherlands Sangamo Therapeut, Richmond, CA USA Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsJacobs, Edwin H.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Mol Genet, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Clin Genet, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsRoovers, Onno论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsVisser, Trudi P.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsJahr, Holger论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Orthopaed, Rotterdam, Netherlands Univ Hosp RWTH, Dept Orthopaed, Aachen, Germany Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsDuncker, Dirk J.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Cardiol, Div Expt Cardiol, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlandsvan Deel, Elza D.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Cardiol, Div Expt Cardiol, Rotterdam, Netherlands Rotterdam Univ Appl Sci, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsReuser, Arnold J. J.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Ctr Lysosomal & Metab Dis, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Clin Genet, Mol Stem Cell Biol, Rotterdam, Netherlands Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlandsvan Til, Niek P.论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Univ Med Ctr Utrecht, Ctr Translat Immunol, Cambridge, MA USA AvroBio, Cambridge, MA USA Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, NetherlandsWagemaker, Gerard论文数: 0 引用数: 0 h-index: 0机构: Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands Hacettepe Med Univ, Stem Cell Res & Dev Ctr, Ankara, Turkey First Pavlov State Med Univ, Raisa Gorbacheva Mem Res Inst Pediat Oncol & Hema, St Petersburg, Russia Erasmus Univ, Med Ctr, Dept Hematol, POB 37048, NL-3005 LA Rotterdam, Netherlands
- [5] Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease[J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 18 : 558 - 570Piras, Giuseppa论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandMontiel-Equihua, Claudia论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandChan, Yee-Ka Agnes论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandWantuch, Slawomir论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandStuckey, Daniel论文数: 0 引用数: 0 h-index: 0机构: UCL, Ctr Adv Biomed Imaging, London WC1E 6DD, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandBurke, Derek论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Hos, Enzyme & Metab Lab, London WC1N 3JH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandPrunty, Helen论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Hos, Enzyme & Metab Lab, London WC1N 3JH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandPhadke, Rahul论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, MRC Ctr Neuromuscular Dis, Dubowitz Neuromuscular Ctr, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandChambers, Darren论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, MRC Ctr Neuromuscular Dis, Dubowitz Neuromuscular Ctr, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandPartida-Gaytan, Armando论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England Fdn Mexicana Ninas & Ninos Con Inmunodeficiencias, Ciudad De Mexico, Mexico UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandLeon-Rico, Diego论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandPanchal, Neelam论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandWhitmore, Kathryn论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandCalero, Miguel论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandBenedetti, Sara论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England NIHR Great Ormond St Hosp Biomed Res Ctr, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandSantilli, Giorgia论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England NIHR Great Ormond St Hosp Biomed Res Ctr, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandThrasher, Adrian J.论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, EnglandGaspar, H. Bobby论文数: 0 引用数: 0 h-index: 0机构: UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England Orchard Therapeut Ltd, London EC4N 6EU, England UCL, UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, Infect Immun & Inflammat Program, 30 Guilford St, London WC1N 1EH, England
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