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Keywords: enzyme replacement therapy
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Biochem J (2021) 478 (12): 2309–2319.
Published: 23 June 2021
...Kai-Wen Cheng; Feng Wang; George A. Lopez; Srikanth Singamsetty; Jill Wood; Patricia I. Dickson; Tsui-Fen Chou Enzyme replacement therapy (ERT) is a scientifically rational and clinically proven treatment for lysosomal storage diseases. Most enzymes used for ERT are purified from the culture...
Includes: Supplementary data
Biochem J (2020) 477 (3): 727–745.
Published: 14 February 2020
... tripeptidyl-peptidase 1 (TPP1). Progression of LINCL can be slowed or halted by enzyme replacement therapy, where recombinant human TPP1 is administered to patients. In this study, we utilized protein engineering techniques to increase the stability of recombinant TPP1 with the rationale that this may...
Includes: Supplementary data
Biochem J (2014) 458 (2): 281–289.
Published: 14 February 2014
...Shih-hsin Kan; Larisa A. Troitskaya; Carolyn S. Sinow; Karyn Haitz; Amanda K. Todd; Ariana Di Stefano; Steven Q. Le; Patricia I. Dickson; Brigette L. Tippin Enzyme replacement therapy for MPS IIIB (mucopolysaccharidosis type IIIB; also known as Sanfilippo B syndrome) has been hindered by inadequate...
Biochem J (2013) 456 (3): 373–383.
Published: 22 November 2013
... by inducing globotriaosylceramide synthesis. Globotriaosylceramide accumulation is a primary pathogenic factor in the symptomatic phenotype. The new mouse model is useful for studying the pathogenesis of Fabry disease and therapeutic approaches. enzyme replacement therapy Fabry disease...
Biochem J (2005) 389 (3): 619–628.
Published: 26 July 2005
... demonstrate that remodelling the carbohydrate of rhGAA to improve its affinity for the CI-MPR represents a feasible approach to enhance the efficacy of enzyme replacement therapy for Pompe disease. 1 To whom correspondence should be addressed (email seng.cheng@genzyme.com ). 2 3 2005 16 4...