Ontology highlight
ABSTRACT:
SUBMITTER: Sugiman-Marangos SN
PROVIDER: S-EPMC7732195 | biostudies-literature | 2020 Dec
REPOSITORIES: biostudies-literature
Sugiman-Marangos Seiji N SN Beilhartz Greg L GL Zhao Xiaochu X Zhou Dongxia D Hua Rong R Kim Peter K PK Rini James M JM Minassian Berge A BA Melnyk Roman A RA
Science advances 20201211 50
Enzyme replacement therapy, in which a functional copy of an 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. The inefficient uptake of recombinant enzymes via the mannose-6-phosphate receptor, however, prohibits the broad utility of replacement therapy. Here, to improve the efficiency and efficacy of lysosomal enzyme uptake, we exploited the strategy used by diphther ...[more]