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- [31] Brain Gene Therapy of Metachromatic Leukodystrophy: From Mice to Human PatientsHUMAN GENE THERAPY, 2009, 20 (06) : 685 - 685Piguet, Francoise论文数: 0 引用数: 0 h-index: 0机构: Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceSondhi, Dolan论文数: 0 引用数: 0 h-index: 0机构: Cornell Univ, Weill Med Coll, New York, NY 10021 USA Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceVanier, Marie论文数: 0 引用数: 0 h-index: 0机构: Fac Med RTH Laennec, INSERM, U820, Lyon, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FrancePiraud, Monique论文数: 0 引用数: 0 h-index: 0机构: Ctr Reference Malad Hereditaires Metab, Lyon, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceAhouansou, Ornella论文数: 0 引用数: 0 h-index: 0机构: Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceFouquet, Francoise论文数: 0 引用数: 0 h-index: 0机构: Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceCrystal, Ronald G.论文数: 0 引用数: 0 h-index: 0机构: Cornell Univ, Weill Med Coll, New York, NY 10021 USA Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceAubourg, Patrick论文数: 0 引用数: 0 h-index: 0机构: Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, FranceCartier, Nathalie论文数: 0 引用数: 0 h-index: 0机构: Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, France Univ Paris 05, Fac Pharmaceut Sci, INSERM, UMR 745, Paris, France论文数: 引用数: h-index:机构:
- [32] Haematopoietic stem cell based gene therapy for the treatment of metachromatic leukodystrophyBONE MARROW TRANSPLANTATION, 2007, 39 : S36 - S36Biffi, A.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalySessa, M.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyCapotondo, A.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyCesani, M.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyFasano, S.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyDel Carro, U.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyTononi, L.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyMarchesini, S.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyBrambilla, R.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyGrompe, M.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyQuattrini, A.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyBordignon, C.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyRoncarolo, M. G.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyRovelli, A.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, ItalyNaldini, L.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Sci Inst, I-20132 Milan, Italy
- [33] 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
- [34] HSC gene therapy trial for Metachromatic Leukodystrophy: first report on gene marking efficiencyHUMAN GENE THERAPY, 2010, 21 (10) : 1363 - 1363Biffi, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Vita Salute San Raffaele, San Raffaele Telethon Inst Gene Therapy HSR TIGET, Pediat Immunohematol & Bone Marrow Transplantat U, San Raffaele Sci Inst, Milan, Italy MolMed SpA, Milan, ItalySessa, M.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, ItalyPlati, T.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, ItalyLorioli, L.论文数: 0 引用数: 0 h-index: 0机构: Univ Vita Salute San Raffaele, San Raffaele Telethon Inst Gene Therapy HSR TIGET, Pediat Immunohematol & Bone Marrow Transplantat U, San Raffaele Sci Inst, Milan, Italy MolMed SpA, Milan, ItalyMontini, E.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, ItalyBenedicenti, F.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, ItalySalomoni, M.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, Italy MolMed SpA, Milan, ItalyVallanti, G.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, Italy MolMed SpA, Milan, ItalyRadrizzani, M.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, Italy MolMed SpA, Milan, ItalyFumagalli, F.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Dept Neurol, Milan, Italy MolMed SpA, Milan, ItalyCasiraghi, M.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, ItalyKabbara, N.论文数: 0 引用数: 0 h-index: 0机构: Rafic Hariri Univ Hosp, Childrens Ctr Canc & Blood Dis, Div Pediat Hematol Oncol 5, Beirut, Lebanon MolMed SpA, Milan, ItalyCiceri, F.论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy HSR TIGET, Bone Marrow Transplantat Unit, Milan, Italy MolMed SpA, Milan, ItalyAiuti, A.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, ItalyRovelli, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Milano Bicocca, Bone Marrow Transplantat Unit 7, Dept Pediat, San Gerardo Hosp, Monza, Italy MolMed SpA, Milan, ItalyRoncarolo, M. G.论文数: 0 引用数: 0 h-index: 0机构: Univ Vita Salute San Raffaele, San Raffaele Telethon Inst Gene Therapy HSR TIGET, Pediat Immunohematol & Bone Marrow Transplantat U, San Raffaele Sci Inst, Milan, Italy MolMed SpA, Milan, ItalyNaldini, L.论文数: 0 引用数: 0 h-index: 0机构: MolMed SpA, Milan, Italy
- [35] A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic LeukodystrophyCRISPR JOURNAL, 2022, 5 (01): : 66 - 79Antony, Justin S.论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyDaniel-Moreno, Alberto论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyLamsfus-Calle, Andres论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyRaju, Janani论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyKaftancioglu, Merve论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyUrena-Bailen, Guillermo论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyRottenberger, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyHou, Yujuan论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanySanthanakumaran, Vidiyaah论文数: 0 引用数: 0 h-index: 0机构: Univ Childrens Hosp, Dept Pediat Neurol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyLee, Jun-Hoe论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Quantitat Biol Ctr QBiC, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyHeumos, Lukas论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Quantitat Biol Ctr QBiC, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyBoehringer, Judith论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Childrens Hosp, Dept Pediat Neurol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyKraegeloh-Mann, Ingeborg论文数: 0 引用数: 0 h-index: 0机构: Univ Childrens Hosp, Dept Pediat Neurol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyHandgretinger, Rupert论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, GermanyMezger, Markus论文数: 0 引用数: 0 h-index: 0机构: Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany Univ Tubingen, Univ Childrens Hosp, Dept Hematol & Oncol, Tubingen, Germany
- [36] Characterization of hematopoietic system reconstitution in vivo in metachromatic leukodystrophy gene therapy patientsHUMAN GENE THERAPY, 2018, 29 (12) : A124 - A125Calabria, A.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, ItalySpinozzi, G.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, ItalyRancoita, P.论文数: 0 引用数: 0 h-index: 0机构: Univ Vita Salute San Raffaele, Univ Ctr Stat Biomed Sci, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyBenedicenti, F.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, ItalyCesana, D.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, ItalyAcquati, S.论文数: 0 引用数: 0 h-index: 0机构: Osped San Raffaele, Pediat Immunohematol & BMT, Segrate, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyRedaelli, D.论文数: 0 引用数: 0 h-index: 0机构: Osped San Raffaele, Pediat Immunohematol & BMT, Segrate, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyAttanasio, V.论文数: 0 引用数: 0 h-index: 0机构: Osped San Raffaele, Pediat Immunohematol & BMT, Segrate, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyFumagalli, F.论文数: 0 引用数: 0 h-index: 0机构: Osped San Raffaele, Pediat Immunohematol & BMT, Segrate, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyDi Serio, C.论文数: 0 引用数: 0 h-index: 0机构: Univ Vita Salute San Raffaele, Univ Ctr Stat Biomed Sci, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyBiffi, A.论文数: 0 引用数: 0 h-index: 0机构: Dana Farber Canc Inst, Boston, MA 02115 USA 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 Osped San Raffaele, Pediat Immunohematol & BMT, Segrate, Italy Univ Vita Salute San Raffaele, Milan, Italy San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, ItalyNaldini, L.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, ItalyMontini, E.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
- [37] 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 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