scholarly journals Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome

JCI Insight ◽  
2021 ◽  
Vol 6 (8) ◽  
Author(s):  
Piming Zhao ◽  
Gizachew B. Tassew ◽  
Joanna Y. Lee ◽  
Babak Oskouian ◽  
Denise P. Muñoz ◽  
...  
2015 ◽  
Vol 23 (11) ◽  
pp. 1712-1721 ◽  
Author(s):  
Tomomi Kiyota ◽  
Gang Zhang ◽  
Christine M Morrison ◽  
Megan E Bosch ◽  
Robert A Weir ◽  
...  

Author(s):  
Bence György ◽  
Elise J. Meijer ◽  
Maryna V. Ivanchenko ◽  
Kelly Tenneson ◽  
Frederick Emond ◽  
...  

2015 ◽  
Vol 23 ◽  
pp. S202-S203
Author(s):  
Eric R. Pozsgai ◽  
Danielle A. Griffin ◽  
Kristin N. Heller ◽  
Jerry R. Mendell ◽  
Louise R. Rodino-Klapac

2008 ◽  
Vol 7 (10) ◽  
pp. 3389-3398 ◽  
Author(s):  
François Lamoureux ◽  
Gaëlle Picarda ◽  
Julie Rousseau ◽  
Clothilde Gourden ◽  
Séverine Battaglia ◽  
...  

2015 ◽  
Vol 24 (13) ◽  
pp. 3651-3661 ◽  
Author(s):  
Takashi Iizuka ◽  
Kazusaku Kamiya ◽  
Satoru Gotoh ◽  
Yoshinobu Sugitani ◽  
Masaaki Suzuki ◽  
...  

2012 ◽  
Vol 20 (4) ◽  
pp. 778-787 ◽  
Author(s):  
Jung Hwa Lim ◽  
Hyo Jung Shin ◽  
Kyeong-Su Park ◽  
Chan Hee Lee ◽  
Cho-Rok Jung ◽  
...  

2011 ◽  
Vol 19 (5) ◽  
pp. 870-875 ◽  
Author(s):  
Eduardo Salido ◽  
Marisol Rodriguez-Pena ◽  
Alfredo Santana ◽  
Stuart G. Beattie ◽  
Harald Petry ◽  
...  

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