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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
by
Bradbury, Allison M
,
Bagel, Jessica H
,
Nguyen, Duc
,
Lykken, Erik A
,
Pesayco Salvador, Jill
,
Jiang, Xuntian
,
Swain, Gary P
,
Assenmacher, Charles A
,
Hendricks, Ian J
,
Miyadera, Keiko
,
Hess, Rebecka S
,
Ostrager, Arielle
,
ODonnell, Patricia
,
Sands, Mark S
,
Ory, Daniel S
,
Shelton, G Diane
,
Bongarzone, Ernesto R
,
Gray, Steven J
,
Vite, Charles H
Published in
The Journal of clinical investigation
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A CNTNAP1 Missense Variant Is Associated with Canine Laryngeal Paralysis and Polyneuropathy
by
Letko, Anna
,
Minor, Katie M
,
Friedenberg, Steven G
,
Shelton, G Diane
,
Salvador, Jill Pesayco
,
Mandigers, Paul J J
,
Leegwater, Peter A J
,
Winkler, Paige A
,
Petersen-Jones, Simon M
,
Stanley, Bryden J
,
Ekenstedt, Kari J
,
Johnson, Gary S
,
Hansen, Liz
,
Jagannathan, Vidhya
,
Mickelson, James R
,
Drögemüller, Cord
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Genes
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