Therapeutic efficacy of rscAAVrh74.miniCMV.<i>LIPA</i> gene therapy in a mouse model of lysosomal acid lipase deficiency.

Cd8 expression, however, showed a trend toward increased expression at all times after treatment, consistent with a CD8 response to AAV infection, though such CD8 responses to AAV can be non-functional. 24 We next looked at the extent of liver fibrosis in treated versus untreated Lipa −/− mice.

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