共 50 条
- [31] Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress-NCT04201405BLOOD, 2020, 136Kinsella, Jane L.论文数: 0 引用数: 0 h-index: 0Wynn, Robert F.论文数: 0 引用数: 0 h-index: 0Bigger, Brian论文数: 0 引用数: 0 h-index: 0Thrasher, Adrian J.论文数: 0 引用数: 0 h-index: 0Booth, Claire论文数: 0 引用数: 0 h-index: 0Buckland, Karen论文数: 0 引用数: 0 h-index: 0Izotova, Natalia论文数: 0 引用数: 0 h-index: 0Rust, Stewart论文数: 0 引用数: 0 h-index: 0Weisberg, Daniel论文数: 0 引用数: 0 h-index: 0Church, Heather J.论文数: 0 引用数: 0 h-index: 0Brammeier, Kathryn L.论文数: 0 引用数: 0 h-index: 0Tylee, Karen L.论文数: 0 引用数: 0 h-index: 0Jones, Ceri论文数: 0 引用数: 0 h-index: 0Lee, Helena论文数: 0 引用数: 0 h-index: 0Ford, Laura论文数: 0 引用数: 0 h-index: 0Sadegholnejat, Atusa论文数: 0 引用数: 0 h-index: 0Ellison, Stuart论文数: 0 引用数: 0 h-index: 0Jones, Simon A.论文数: 0 引用数: 0 h-index: 0
- [32] INTERIM ANALYSIS ON NEUROLOGICAL OUTCOMES IN HURLER SYNDROME PATIENTS TREATED WITH EX-VIVO HAEMATOPOIETIC STEM CELL GENE THERAPYBONE MARROW TRANSPLANTATION, 2024, 59 : 67 - 68Consiglieri, Giulia论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyFumagalli, Francesca论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyTucci, Francesca论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyFilisetti, Chiara论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyCossutta, Matilde论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyScarparo, Stefano论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalySarzana, Marina论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyPuricelli, Cristina论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyCiotti, Francesca论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyBarbera, Maurizio论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyDarin, Silvia论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyParini, Rossella论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyBaldoli, Cristina论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyGentner, Bernhard论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyAiuti, Alessandro论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, ItalyBernardo, Maria Ester论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Milan, Italy IRCCS San Raffaele Sci Inst, Milan, Italy
- [33] Clinical trial design for HMI-203 investigational gene therapy for mucopolysaccharidosis type II (MPS II) informed by cross-correction potential and KOL inputMOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S46 - S46Gingras, Jacinthe论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USAHaroldson, Jeff论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USASmith, Laura J.论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USAPatel, Kruti论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USASalstrom, Jen论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USAJordan, Julie论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USACohn, Gabriel M.论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USAFrancone, Omar论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USASeymour, Albert论文数: 0 引用数: 0 h-index: 0机构: Homol Med, Bedford, MA USA Homol Med, Bedford, MA USA
- [34] An innovative platform approach for the preclinical development of ex-vivo gene therapy for lysosomal storage disorders with skeletalHUMAN GENE THERAPY, 2024, 35 (3-4) : A246 - A246Crippa, S.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyScala, S.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyAlberti, G.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyForni, C.论文数: 0 引用数: 0 h-index: 0机构: Telethon Fdn ETS, Rome, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyBerti, M.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyQuaranta, P.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalySanti, L.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyDe Ponti, G.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyJofra-Hernandez, R.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyBasso-Ricci, L.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyRilievo, A. A.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyVisigalli, I.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, GLP San Raffaele Telethon Inst Gene Therapy SR TI, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyCecere, F.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, GLP San Raffaele Telethon Inst Gene Therapy SR TI, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyZonari, E.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyConsiglieri, G.论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyTucci, F.论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyCossutta, M.论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyAlbertini, P.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, GLP San Raffaele Telethon Inst Gene Therapy SR TI, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyParini, R.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyGentner, B.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyBernardo, M. E.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy Univ Vita Salute San Raffaele, Fac Med, Milan, Italy IRCCS San Raffaele Sci Inst, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyAiuti, A.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy Univ Vita Salute San Raffaele, Fac Med, Milan, Italy IRCCS San Raffaele Sci Inst, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy IRCCS Osped San Raffaele, San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy
- [35] Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-HurlerMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2022, 25 : 370 - 381Jin, Xiu论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaSu, Jing论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaZhao, Qinyu论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaLi, Ruiting论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaXiao, Jianlu论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaZhong, Xiaomei论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaSong, Li论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaLiu, Yi论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaShe, Kaiqin论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Dept Ophthalmol, Chengdu, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaDeng, Hongxin论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaWei, Yuquan论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R ChinaYang, Yang论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, Canc Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Collaborat Innovat Ctr, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Ke Yuan Rd 4,1 Gao Peng St, Chengdu 610041, Sichuan, Peoples R China
- [36] Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defectsHUMAN GENE THERAPY, 2021, 32 (19-20) : A77 - A77Santi, L.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy论文数: 引用数: h-index:机构:Crippa, S.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyCapo, V.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy Univ Vita Salute San Raffaele, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy论文数: 引用数: h-index:机构:Berti, M.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyMancino, M.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyTucci, F.论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyRiminucci, M.论文数: 0 引用数: 0 h-index: 0机构: Sapienza Univ, Dept Mol Med, Rome, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyCorsi, A.论文数: 0 引用数: 0 h-index: 0机构: Sapienza Univ, Dept Mol Med, Rome, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy论文数: 引用数: h-index:机构:Klein, T.论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Basel, Tissue Engn Dept Biomed, Basel, Switzerland San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyLopa, S.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Galeazzi Orthopaed Inst, Cell & Tissue Engn Lab, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyMartin, I.论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Basel, Tissue Engn Dept Biomed, Basel, Switzerland San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyMoretti, M.论文数: 0 引用数: 0 h-index: 0机构: IRCCS Galeazzi Orthopaed Inst, Cell & Tissue Engn Lab, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyGentner, B.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyAiuti, A.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy Ist Sci San Raffaele, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy Univ Vita Salute San Raffaele, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyVilla, A.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy CNR, Ist Ric Genet & Biomed, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyBernardo, M. E.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy Ist Sci San Raffaele, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy
- [37] Dissecting Bone Remodelling Mechanisms and Hematopoietic Stem Cell Gene Therapy Impact in Mucopolysaccharidosis Type I Hurler Bone DefectsMOLECULAR THERAPY, 2021, 29 (04) : 295 - 296Santi, Ludovica论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyPenna, Sara论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyCrippa, Stefania论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyCapo, Valentina论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyDe Ponti, Giada论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyBerti, Margherita论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyMancino, Marilena论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyTucci, Francesca论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyRiminucci, Mara论文数: 0 引用数: 0 h-index: 0机构: Sapienza Univ, Dept Mol Med, Rome, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyCorsi, Alessandro论文数: 0 引用数: 0 h-index: 0机构: Sapienza Univ, Dept Mol Med, Rome, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy论文数: 引用数: h-index:机构:Klein, Thibaut论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Basel, Tissue Engn Dept Biomed, Basel, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyMartin, Ivan论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Basel, Tissue Engn Dept Biomed, Basel, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyGentner, Bernhard论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyAiuti, Alessandro论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyVilla, Anna论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, ItalyBernardo, Maria Ester论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR Tiget, Milan, Italy
- [38] Ex vivo gene therapy improves the accumulation of glycosaminoglycans in brain from murine model of mucopolysaccharidosis type IIGLYCOBIOLOGY, 2014, 24 (11) : 1214 - 1214Wakabayashi, Taichi论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, Japan Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, JapanShimada, Yohta论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Res Ctr Med Sci, Div Gene Therapy, Minato, Tokushima, Japan Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, JapanHiguchi, Takashi论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Res Ctr Med Sci, Div Gene Therapy, Minato, Tokushima, Japan Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, JapanKobayashi, Hiroshi论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Res Ctr Med Sci, Div Gene Therapy, Minato, Tokushima, Japan Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, JapanIda, Hiroyuki论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, Japan Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, JapanOhashi, Toya论文数: 0 引用数: 0 h-index: 0机构: Jikei Univ, Sch Med, Res Ctr Med Sci, Div Gene Therapy, Minato, Tokushima, Japan Jikei Univ, Sch Med, Dept Pediat, Minato, Tokyo, Japan
- [39] Mucopolysaccharidosis Type I Ex Vivo Gene Therapy Based on MLV-Mesenchymal Stem Cell Transduction: Is There a Way To Be Attenuated When Older?MOLECULAR THERAPY, 2009, 17 : S345 - S345Silva, Flavia H.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilPereira, Vanessa F.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilYasumura, Eduardo论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilTenorio, Ligia Z.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilCarvalho, Leonardo P.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilLisboa, Bianca C.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilCalegare, Bruno F.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilBrandao, Leticia D.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilD'Almeida, Vania论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilFilippo, Thais R.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilPorcionatto, Marimelia论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilToma, Leny论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilNader, Helena B.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilCamassola, Melissa论文数: 0 引用数: 0 h-index: 0机构: ULBRA, Porto Alegre, RS, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilNardi, Nance B.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil ULBRA, Porto Alegre, RS, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, BrazilHan, Sang W.论文数: 0 引用数: 0 h-index: 0机构: Univ Fed Sao Paulo, CINTERGEN, Sao Paulo, Brazil Univ Fed Rio Grande do Sul, Porto Alegre, RS, Brazil
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