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Showing 1 to 1 of 1 for “"Niemann Pick's disease"”.
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Factors affecting transduction efficiency of pseudotyped viral vectors incorporating alphaviral glycoproteins
… deficient (ASMase-/-) cells derived from Niemann Pick’s disease-model mice more efficiently than the cells from healthy mice (ASMase+/+). Thus alphavirus-glycoprotein pseudotypes hold a potential as suitable vectors for gene therapy/ transfer in Niemann Pick’s Disease-Type A, a genetic …