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Efficacy of AAV9-mediated SGPL1 gene transfer in a mouse model of S1P lyase insufficiency syndrome
by
Zhao, Piming
,
Tassew, Gizachew B
,
Lee, Joanna Y
,
Oskouian, Babak
,
Muñoz, Denise P
,
Hodgin, Jeffrey B
,
Watson, Gordon L
,
Tang, Felicia
,
Wang, Jen-Yeu
,
Luo, Jinghui
,
Yang, Yingbao
,
King, Sarah
,
Krauss, Ronald M
,
Keller, Nancy
,
Saba, Julie D
Published in
JCI insight
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AAV-SPL 2.0, a Modified Adeno-Associated Virus Gene Therapy Agent for the Treatment of Sphingosine Phosphate Lyase Insufficiency Syndrome
by
Khan, Ranjha
,
Oskouian, Babak
,
Lee, Joanna Y
,
Hodgin, Jeffrey B
,
Yang, Yingbao
,
Tassew, Gizachew
,
Saba, Julie D
Published in
International journal of molecular sciences
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