Gentamicin-mediated suppression of Hurler syndrome stop mutations restores a low level of alpha-L-iduronidase activity and reduces lysosomal glycosaminoglycan accumulation

Human Molecular Genetics - Tập 10 Số 3 - Trang 291-299 - 2001
Kim M. Keeling1, Doug A. Brooks, John J. Hopwood, Jia Li, Jerry N. Thompson, David M. Bedwell
1Department of Human Genetics, University of Alabama at Birmingham, Birmingham, AL 35294-2170, USA.

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