Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome
Piming Zhao
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Gizachew B. Tassew
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Joanna Y. Lee
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Babak Oskouian
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Denise P. Muñoz
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...
2015 ◽
Vol 23
(11)
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pp. 1712-1721
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Tomomi Kiyota
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Gang Zhang
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Christine M Morrison
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Megan E Bosch
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Robert A Weir
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...
2019 ◽
Vol 13
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pp. 1-13
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Bence György
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Elise J. Meijer
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Maryna V. Ivanchenko
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Kelly Tenneson
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Frederick Emond
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...
2013 ◽
Vol 9
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pp. P187-P187
Antonella Caccamo
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Salvatore Oddo
2003 ◽
Vol 7
(5)
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pp. S130-S131
2015 ◽
Vol 23
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pp. S202-S203
Eric R. Pozsgai
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Danielle A. Griffin
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Kristin N. Heller
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Jerry R. Mendell
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Louise R. Rodino-Klapac
2008 ◽
Vol 7
(10)
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pp. 3389-3398
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François Lamoureux
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Gaëlle Picarda
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Julie Rousseau
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Clothilde Gourden
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Séverine Battaglia
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...
2015 ◽
Vol 24
(13)
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pp. 3651-3661
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Takashi Iizuka
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Kazusaku Kamiya
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Satoru Gotoh
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Yoshinobu Sugitani
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Masaaki Suzuki
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...
2012 ◽
Vol 20
(4)
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pp. 778-787
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Jung Hwa Lim
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Hyo Jung Shin
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Kyeong-Su Park
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Chan Hee Lee
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Cho-Rok Jung
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...
2003 ◽
Vol 111
(2)
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pp. S309
S.S. Mohapatra
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M. Kumar
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A. Behera
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G. Hellerman
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R.F. Lockey
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...
2011 ◽
Vol 19
(5)
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pp. 870-875
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Eduardo Salido
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Marisol Rodriguez-Pena
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Alfredo Santana
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Stuart G. Beattie
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Harald Petry
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...