共 35 条
[1]
A PEDIGREE STUDY OF METACHROMATIC LEUKODYSTROPHY . BIOCHEMICAL IDENTIFICATION OF CARRIER STATE
[J].
BASS, NH
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WITMER, EJ
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DREIFUSS, FE
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NEUROLOGY,
1970, 20 (01)
:52-&

BASS, NH
论文数: 0 引用数: 0
h-index: 0

WITMER, EJ
论文数: 0 引用数: 0
h-index: 0

DREIFUSS, FE
论文数: 0 引用数: 0
h-index: 0
[2]
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
[J].
Biffi, Alessandra
;
Montini, Eugenio
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Lorioli, Laura
;
Cesani, Martina
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Fumagalli, Francesca
;
Plati, Tiziana
;
Baldoli, Cristina
;
Martino, Sabata
;
Calabria, Andrea
;
Canale, Sabrina
;
Benedicenti, Fabrizio
;
Vallanti, Giuliana
;
Biasco, Luca
;
Leo, Simone
;
Kabbara, Nabil
;
Zanetti, Gianluigi
;
Rizzo, William B.
;
Mehta, Nalini A. L.
;
Cicalese, Maria Pia
;
Casiraghi, Miriam
;
Boelens, Jaap J.
;
Del Carro, Ubaldo
;
Dow, David J.
;
Schmidt, Manfred
;
Assanelli, Andrea
;
Neduva, Victor
;
Di Serio, Clelia
;
Stupka, Elia
;
Gardner, Jason
;
von Kalle, Christof
;
Bordignon, Claudio
;
Ciceri, Fabio
;
Rovelli, Attilio
;
Roncarolo, Maria Grazia
;
Aiuti, Alessandro
;
Sessa, Maria
;
Naldini, Luigi
.
SCIENCE,
2013, 341 (6148)
:864-U58

Biffi, 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, Italy

Montini, Eugenio
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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

Lorioli, 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, Italy

Cesani, 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, Italy

Fumagalli, Francesca
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Plati, Tiziana
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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

Baldoli, Cristina
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Martino, Sabata
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Calabria, 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, Italy

Canale, Sabrina
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Benedicenti, Fabrizio
论文数: 0 引用数: 0
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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

Vallanti, Giuliana
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Biasco, 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, Italy

Leo, Simone
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Kabbara, Nabil
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Zanetti, Gianluigi
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Rizzo, William B.
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Mehta, Nalini A. L.
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Cicalese, Maria Pia
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Casiraghi, 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, Italy

Boelens, Jaap J.
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Del Carro, Ubaldo
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Dow, 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, Italy

Schmidt, 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, Italy

Assanelli, 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

Neduva, Victor
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Di Serio, Clelia
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Stupka, 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, Italy

Gardner, 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, Italy

von 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, Italy

Bordignon, Claudio
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Ciceri, Fabio
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Rovelli, Attilio
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Roncarolo, 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, Italy

Aiuti, 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, Italy

Sessa, Maria
论文数: 0 引用数: 0
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机构: Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy TIGET, I-20132 Milan, Italy

Naldini, 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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Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report
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Boucher, Alexander A.
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Miller, Weston
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Shanley, Ryan
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Ziegler, Richard
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Lund, Troy
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Raymond, Gerald
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Orchard, Paul J.
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ORPHANET JOURNAL OF RARE DISEASES,
2015, 10

Boucher, Alexander A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA

Miller, Weston
论文数: 0 引用数: 0
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机构:
Div Pediat Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA

Shanley, Ryan
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Minnesota, Mason Canc Ctr, Biostat Core, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA

Ziegler, Richard
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Minnesota, Div Pediat Clin Neurosci, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA

Lund, Troy
论文数: 0 引用数: 0
h-index: 0
机构:
Div Pediat Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA

Raymond, Gerald
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Minnesota, Div Pediat Clin Neurosci, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA

Orchard, Paul J.
论文数: 0 引用数: 0
h-index: 0
机构:
Div Pediat Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Internal Med & Pediat, Minneapolis, MN 55455 USA
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Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
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Chan, Ken Y.
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Jang, Min J.
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Yoo, Bryan B.
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Greenbaum, Alon
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Ravi, Namita
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Wu, Wei-Li
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Sanchez-Guardado, Luis
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Lois, Carlos
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Mazmanian, Sarkis K.
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Deverman, Benjamin E.
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Gradinaru, Viviana
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NATURE NEUROSCIENCE,
2017, 20 (08)
:1172-+

Chan, Ken Y.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Jang, Min J.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Yoo, Bryan B.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Greenbaum, Alon
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Ravi, Namita
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Wu, Wei-Li
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Sanchez-Guardado, Luis
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Lois, Carlos
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Mazmanian, Sarkis K.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Deverman, Benjamin E.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Gradinaru, Viviana
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA
[5]
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate
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Colle, Marie-Anne
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Piguet, Francoise
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Bertrand, Lise
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Raoul, Sylvie
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Bieche, Ivan
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Dubreil, Laurence
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Sloothaak, Didi
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Bouquet, Celine
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Moullier, Philippe
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Aubourg, Patrick
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Cherel, Yan
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Cartier, Nathalie
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Sevin, Caroline
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HUMAN MOLECULAR GENETICS,
2010, 19 (01)
:147-158

Colle, Marie-Anne
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Vet Nantes, INRA, UMR 703, Nantes, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Piguet, Francoise
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Paris, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Bertrand, Lise
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Vet Nantes, INRA, UMR 703, Nantes, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Raoul, Sylvie
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Nantes, CHU Nord, Serv Neurochirurg, Nantes, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Bieche, Ivan
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Paris, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Dubreil, Laurence
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Vet Nantes, INRA, UMR 703, Nantes, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Sloothaak, Didi
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Paris, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Bouquet, Celine
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Paris, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Moullier, Philippe
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Nantes, INSERM, U649, Nantes, France
Univ Florida, Dept Mol Genet & Microbiol, Gainesville, FL USA INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Aubourg, Patrick
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Paris, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Cherel, Yan
论文数: 0 引用数: 0
h-index: 0
机构:
Ecole Vet Nantes, INRA, UMR 703, Nantes, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

Cartier, Nathalie
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Paris 05, Paris, France INSERM, Fac Sci Pharmaceut & Biol Paris 5, UMR 745, F-75279 Paris 06, France

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Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial
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Dali, Christine, I
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Sevin, Caroline
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Giugliani, Roberto
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Sakai, Norio
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Wu, James
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Wasilewski, Margaret
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MOLECULAR GENETICS AND METABOLISM,
2020, 131 (1-2)
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Dali, Christine, I
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h-index: 0
机构:
Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark
Orphazyme AS, Copenhagen, Denmark Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark

Sevin, Caroline
论文数: 0 引用数: 0
h-index: 0
机构:
Bicetre Hosp, Neuropediat Unit, Paris, France Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark

Kraegeloh-Mann, Ingeborg
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Childrens Hosp Tubingen, Dept Neuropediat, Tubingen, Germany Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark

Giugliani, Roberto
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Fed Rio Grande do Sul, Med Genet Serv, HCPA, Dept Genet, Porto Alegre, RS, Brazil
INAGEMP, Porto Alegre, RS, Brazil Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark

论文数: 引用数:
h-index:
机构:

Wu, James
论文数: 0 引用数: 0
h-index: 0
机构:
Takeda Grp Co, Shire, Lexington, MA USA
Ironwood Pharmaceut, Boston, MA USA Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark

Wasilewski, Margaret
论文数: 0 引用数: 0
h-index: 0
机构:
Takeda Grp Co, Shire, Lexington, MA USA
Summit Therapeut PLC, Cambridge, MA USA Copenhagen Univ Hosp, Dept Clin Genet, Rigshosp, Copenhagen, Denmark
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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
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Deverman, Benjamin E.
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Pravdo, Piers L.
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Chan, Ken Y.
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Banerjee, Abhik
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Wu, Wei-Li
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Yang, Bin
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Huber, Nina
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Pasca, Sergiu P.
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Gradinaru, Viviana
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NATURE BIOTECHNOLOGY,
2016, 34 (02)
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Deverman, Benjamin E.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Pravdo, Piers L.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Simpson, Bryan P.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Kumar, Sripriya Ravindra
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Chan, Ken Y.
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Banerjee, Abhik
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Wu, Wei-Li
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Yang, Bin
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Huber, Nina
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Sch Med, Dept Psychiat & Behav Sci, Stanford, CA 94305 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Pasca, Sergiu P.
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Sch Med, Dept Psychiat & Behav Sci, Stanford, CA 94305 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA

Gradinaru, Viviana
论文数: 0 引用数: 0
h-index: 0
机构:
CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA CALTECH, Div Biol & Biol Engn, Pasadena, CA 91125 USA
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Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy
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Fu, Haiyan
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Flanigan, Kevin M.
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McCarty, Douglas M.
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HUMAN GENE THERAPY CLINICAL DEVELOPMENT,
2017, 28 (04)
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Fu, Haiyan
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h-index: 0
机构:
Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

Meadows, Aaron S.
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Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

Pineda, Ricardo J.
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Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

Kunkler, Krista L.
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Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

Truxal, Kristen V.
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Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA
Nationwide Childrens Hosp, Div Mol & Human Genet, Columbus, OH USA
Ohio State Univ, Sch Med, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

McBride, Kim L.
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Nationwide Childrens Hosp, Ctr Cardiovasc Res, Columbus, OH USA
Nationwide Childrens Hosp, Div Mol & Human Genet, Columbus, OH USA
Ohio State Univ, Sch Med, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

Flanigan, Kevin M.
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Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA
Ohio State Univ, Sch Med, Dept Pediat, Columbus, OH 43210 USA
Ohio State Univ, Sch Med, Dept Neurol, Columbus, OH 43210 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA

McCarty, Douglas M.
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Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA
Ohio State Univ, Sch Med, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Ctr Gene Therapy, 700 Childrens Dr, Columbus, OH 43205 USA
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Gieselmann, V.
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机构:
Univ Bonn, Dept Physiol, D-5300 Bonn, Germany Univ Childrens Hosp, Dept Paediat Neurol & Dev Med, D-72076 Tubingen, Germany

Kraegeloh-Mann, I.
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机构:
Univ Childrens Hosp, Dept Paediat Neurol & Dev Med, D-72076 Tubingen, Germany Univ Childrens Hosp, Dept Paediat Neurol & Dev Med, D-72076 Tubingen, Germany
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Gieselmann V., 2006, Scriver's Online Metabolic and Molecular Bases of Inherited Disease, DOI [10.1036/ommbid.179, DOI 10.1036/OMMBID.179]