Kanut Laoharawee on Muck Rack

Kanut Laoharawee

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Articles

Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders

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By Jacob Jensen, Joseph Peterson, Evan Kleinboehl, Kanut Laoharawee
| biorxiv.org
Abstract Enzymopathy disorders are the result of missing or defective enzymes. Amongst these enzymopathies, mucopolysaccharidosis type I, is a rare genetic lysosomal storage disorder caused by mutations in the gene encoding alpha-L-iduronidase (IDUA), ultimately causes toxic build-up of glycosaminoglycans (GAGs). There is currently no cure and standard treatments provide insufficient relief to the skeletal structure and central nervous system (CNS).
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