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References
1.
Mendell
JR
,
Al-Zaidy
SA
,
Rodino-Klapac
LR
, et al. Current clinical applications of in vivo gene therapy with AAVs . Mol Ther , 2021 ; 29 :464 –488 .
2.
Vanier
MT
. Niemann-Pick disease type C . Orphanet J Rare Dis , 2010 ; 5 :16 .
3.
Kurokawa
Y
,
Osaka
H
,
Kouga
T
, et al. Gene therapy in a mouse model of Niemann-Pick disease type C1 . Hum Gene Ther , 2021 ; 32 :589 –598 .
