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Full-Text Articles in Enzymes and Coenzymes
Interconversion Of The Specificities Of Human Lysosomal Enzymes, Ivan B. Tomasic
Interconversion Of The Specificities Of Human Lysosomal Enzymes, Ivan B. Tomasic
Masters Theses 1911 - February 2014
Fabry disease (FD) is an X-linked recessive lysosomal storage disorder (LSD) known to affect approximately 1 in every 40,000 males, and a smaller number of females. FD results from a deficiency of functional α-galactosidase (α-GAL), which leads to the accumulation of terminally α-galactosylated substrates in the lysosome. The predominant treatment is Enzyme Replacement Therapy (ERT), requiring the regular infusion of recombinant human α-GAL. More than half of individuals receiving ERT experience a range of adverse infusion reactions, and it has been reported that as many as 88% of patients receiving ERT develop neutralizing IgG antibodies against the drug.
In aim …