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- [2] Gene therapy of metachromatic leukodystrophy by AAV vectorsJOURNAL OF GENE MEDICINE, 2006, 8 (03): : 393 - 393Kurai, T论文数: 0 引用数: 0 h-index: 0机构: Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, Japan Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, JapanKitagawa, R论文数: 0 引用数: 0 h-index: 0机构: Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, Japan Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, JapanHisayasu, S论文数: 0 引用数: 0 h-index: 0机构: Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, Japan Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, JapanHirai, Y论文数: 0 引用数: 0 h-index: 0机构: Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, Japan Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, JapanShimada, T论文数: 0 引用数: 0 h-index: 0机构: Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, Japan Nippon Med Coll, Dept Biochem & Mol Biol, Tokyo 1138602, Japan
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- [4] In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected miceNature Medicine, 2001, 7 : 310 - 316Antonella Consiglio论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyAngelo Quattrini论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologySabata Martino论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyJean Charles Bensadoun论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyDiego Dolcetta论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyAlessandra Trojani论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyGiuliana Benaglia论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologySergio Marchesini论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyVincenzo Cestari论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyAlberto Oliverio论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyClaudio Bordignon论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of NeurologyLuigi Naldini论文数: 0 引用数: 0 h-index: 0机构: Scientific Institute H.S. Raffaele HSR-TIGET,Telethon Institute for Gene Therapy and Department of Neurology
- [5] In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors:: correction of neuropathology and protection against learning impairments in affected miceNATURE MEDICINE, 2001, 7 (03) : 310 - 316Consiglio, A论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyQuattrini, A论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyMartino, S论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyBensadoun, JC论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyDolcetta, D论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyTrojani, A论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyBenaglia, G论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyMarchesini, S论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyCestari, V论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyOliverio, A论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyBordignon, C论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, Italy Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, ItalyNaldini, L论文数: 0 引用数: 0 h-index: 0机构: Sci Inst HS Raffaele HSR TIGET, Telethon Inst Gene Therapy, Milan, Italy
- [6] Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic LeukodystrophySCIENCE, 2013, 341 (6148) : 864 - U58Biffi, Alessandra论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyMontini, Eugenio论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyLorioli, Laura论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyCesani, Martina论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyFumagalli, Francesca论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyPlati, Tiziana论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyBaldoli, Cristina论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyMartino, Sabata论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyCalabria, Andrea论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyCanale, Sabrina论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyBenedicenti, Fabrizio论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyVallanti, Giuliana论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyBiasco, Luca论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyLeo, Simone论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyKabbara, Nabil论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyZanetti, Gianluigi论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyRizzo, William B.论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyMehta, Nalini A. L.论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyCicalese, Maria Pia论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyCasiraghi, Miriam论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyBoelens, Jaap J.论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyDel Carro, Ubaldo论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyDow, David J.论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalySchmidt, Manfred论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyAssanelli, Andrea论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyNeduva, Victor论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyDi Serio, Clelia论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyStupka, Elia论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyGardner, Jason论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italyvon Kalle, Christof论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyBordignon, Claudio论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyCiceri, Fabio论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyRovelli, Attilio论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyRoncarolo, Maria Grazia论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyAiuti, Alessandro论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalySessa, Maria论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, ItalyNaldini, Luigi论文数: 0 引用数: 0 h-index: 0机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy
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- [10] Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrationsMOLECULAR THERAPY NUCLEIC ACIDS, 2025, 36 (01):Tricoli, Lucas论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USASase, Sunetra论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Neurol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAHacker, Julia L.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Neurol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAPham, Vi论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Human Genet, Dept Pediat, Philadelphia, PA USA Univ Penn, Perelman Sch Med, Philadelphia, PA USA Univ Penn, Cell & Mol Biol Affin Grp CAMB, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAChappell, Maxwell论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USABreda, Laura论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAHurwitz, Stephanie N.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Philadelphia, PA USA CHOP, Comprehens Bone Marrow Failure Ctr, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USATanaka, Naoto论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USACastracani, Carlo Castruccio论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAGuerra, Amaliris论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAHou, Zhongqi论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Neurol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USASchlotawa, Lars论文数: 0 引用数: 0 h-index: 0机构: Univ Med Ctr Gottingen, Gottingen, Germany Fraunhofer Inst Translat Med & Pharmacol, Gottingen, Germany Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA论文数: 引用数: h-index:机构:Hogenauer, Matthew论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Dept Microbiol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USARoche, Aoife论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Dept Microbiol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAEverett, John论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Dept Microbiol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USABushman, Frederic论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Dept Microbiol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAKurre, Peter论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAAhrens-Nicklas, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Human Genet, Dept Pediat, Philadelphia, PA USA Univ Penn, Perelman Sch Med, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAAdang, Laura A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Neurol, Philadelphia, PA USA Univ Penn, Perelman Sch Med, Dept Neurol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USAVanderver, Adeline L.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Neurol, Philadelphia, PA USA Univ Penn, Perelman Sch Med, Dept Microbiol, Philadelphia, PA USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USARivella, Stefano论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Philadelphia, PA USA Hosp Univ Penn, Philadelphia, PA USA Univ Penn, Inst RNA Innovat, Philadelphia, PA USA Univ Penn, Inst Regenerat Med, Philadelphia, PA USA CHOP, Penn Ctr Musculoskeletal Disorders, Philadelphia, PA USA CHOP, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Div Hematol, Philadelphia, PA 19104 USA