Developing a Potential Substrate Reduction Therapy for Six Mucopolysaccharidoses by Decreasing NDST1 Activity
Mucopolysaccharidoses result from genetic mutations in lysosomal enzymes required for degradation of glycosaminoglycans. The deficiency in any of eight lysosomal enzymes needed to degrade heparan sulfate leads to an accumulation of both non-degraded and partially degraded polysaccharides within the...
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| Language: | en_ca |
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2013
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| Online Access: | http://hdl.handle.net/1807/42963 |