Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA

New method for quantitative determination of uronic acids

Nelly Blumenkrantz, G. Asboe‐Hansen · Analytical Biochemistry · 1973 · 7.1K citations

Natural Sciences, Biochemistry, Analytical Chemistry +4

Epidemiology of mucopolysaccharidoses

Shaukat Khan, Hira Peracha, Diana Ballhausen et al. · Molecular Genetics and Metabolism · 2017 · 1.1K citations

Studies on glucosaminidase. 4. The fluorimetric assay of <i>N</i>-acetyl-β-glucosaminidase

David H. Leaback, P. G. Walker · Biochemical Journal · 1961 · 453 citations · Full text

Life Sciences, Biochemistry, Enzymology +6

Efficient Gene Delivery and Targeted Expression to Hepatocytes <i>In Vivo</i> by Improved Lentiviral Vectors

Antonia Follenzi, Giovanni Sabatino, Angelo Lombardo et al. · Human Gene Therapy · 2002 · 242 citations

Pharmacy, Biotechnology, Molecular Biology +17

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease

Thomas M. Daly, Carole Vogler, Beth Levy et al. · Proceedings of the National Academy of Sciences · 1999 · 206 citations

Life Sciences, Genetics, Molecular Biology +17