Article

Exploiting toxin internalization receptors to enhance delivery of proteins to lysosomes for enzyme replacement therapy

2020-01-23

Abstract excerpt

Lysosomal storage diseases are a group of over 70 inherited genetic diseases caused by a defect or deficiency in a lysosomal protein. Enzyme replacement therapy, in which a functional copy of the defective enzyme is injected either systemically or directly into the brain of affected individuals, has proven to be an effective strategy for treating certain lysosomal storage diseases; however, the inefficient uptake...

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Identifiers and source

Literature Corpus work: e919f9e7-961d-52fe-9899-1d6bc6431129
DOI: 10.1101/2020.01.22.915298

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