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- W2519937269 abstract "Abstract Lysosomal storage diseases (LSDs) are a group of 40-50 rare monogenic disorders that result in disrupted lysosomal function and subsequent lysosomal pathology. Depending on the protein or enzyme deficiency associated with each disease, LSDs affect an array of organ systems and elicit a complex set of secondary disease mechanisms that make many of these disorders difficult to fully treat. The etiology of most LSDs is known and the innate biology of lysosomal enzymes favors therapeutic intervention, yet most attempts at treating LSDs with enzyme replacement strategies fall short of being curative. Even with the advent of more sophisticated approaches, like substrate reduction therapy, pharmacologic chaperones, gene therapy or stem cell therapy, comprehensive treatments for LSDs have yet to be achieved. Given the limitations with individual therapies, recent research has focused on using a combination approach to treat LSDs. By coupling protein-, cell-, and gene- based therapies with small molecule drugs, researchers have found greater success in eradicating the clinical features of disease. This review seeks to discuss the positive and negatives of singular therapies used to treat LSDs, and discuss how, in combination, studies have demonstrated a more holistic benefit on pathological and functional parameters. By optimizing routes of delivery, therapeutic timing, and targeting secondary disease mechanisms, combination therapy represents the future for LSD treatment." @default.
- W2519937269 created "2016-09-23" @default.
- W2519937269 creator A5066821454 @default.
- W2519937269 date "2016-06-01" @default.
- W2519937269 modified "2023-09-23" @default.
- W2519937269 title "Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem." @default.
- W2519937269 cites W1498704261 @default.
- W2519937269 cites W1509821238 @default.
- W2519937269 cites W1599067171 @default.
- W2519937269 cites W1964210819 @default.
- W2519937269 cites W1965227495 @default.
- W2519937269 cites W1965350697 @default.
- W2519937269 cites W1966394929 @default.
- W2519937269 cites W1967567480 @default.
- W2519937269 cites W1969492366 @default.
- W2519937269 cites W1970030078 @default.
- W2519937269 cites W1971217038 @default.
- W2519937269 cites W1974742668 @default.
- W2519937269 cites W1977439828 @default.
- W2519937269 cites W1982869943 @default.
- W2519937269 cites W1984841297 @default.
- W2519937269 cites W1992646508 @default.
- W2519937269 cites W1993728901 @default.
- W2519937269 cites W1995863785 @default.
- W2519937269 cites W1996007115 @default.
- W2519937269 cites W1998598037 @default.
- W2519937269 cites W2004893995 @default.
- W2519937269 cites W2005623482 @default.
- W2519937269 cites W2012506180 @default.
- W2519937269 cites W2014022896 @default.
- W2519937269 cites W2018078101 @default.
- W2519937269 cites W2020008627 @default.
- W2519937269 cites W2025541547 @default.
- W2519937269 cites W2026584056 @default.
- W2519937269 cites W2031826717 @default.
- W2519937269 cites W2034955397 @default.
- W2519937269 cites W2039857764 @default.
- W2519937269 cites W2043929943 @default.
- W2519937269 cites W2043965507 @default.
- W2519937269 cites W2044785609 @default.
- W2519937269 cites W2047162457 @default.
- W2519937269 cites W2054412016 @default.
- W2519937269 cites W2056265469 @default.
- W2519937269 cites W2058658653 @default.
- W2519937269 cites W2058725606 @default.
- W2519937269 cites W2060496255 @default.
- W2519937269 cites W2062018052 @default.
- W2519937269 cites W2062600620 @default.
- W2519937269 cites W2064398966 @default.
- W2519937269 cites W2066284508 @default.
- W2519937269 cites W2075367650 @default.
- W2519937269 cites W2075891229 @default.
- W2519937269 cites W2078015980 @default.
- W2519937269 cites W2082475723 @default.
- W2519937269 cites W2082991267 @default.
- W2519937269 cites W2083048616 @default.
- W2519937269 cites W2083455100 @default.
- W2519937269 cites W2084225245 @default.
- W2519937269 cites W2084764150 @default.
- W2519937269 cites W2089627637 @default.
- W2519937269 cites W2091141794 @default.
- W2519937269 cites W2093722439 @default.
- W2519937269 cites W2094919922 @default.
- W2519937269 cites W2101746758 @default.
- W2519937269 cites W2102542024 @default.
- W2519937269 cites W2105748332 @default.
- W2519937269 cites W2107128771 @default.
- W2519937269 cites W2109305605 @default.
- W2519937269 cites W2117767504 @default.
- W2519937269 cites W2118314348 @default.
- W2519937269 cites W2123858283 @default.
- W2519937269 cites W2126439062 @default.
- W2519937269 cites W2126901292 @default.
- W2519937269 cites W2132754272 @default.
- W2519937269 cites W2135424735 @default.
- W2519937269 cites W2138948114 @default.
- W2519937269 cites W2141964082 @default.
- W2519937269 cites W2144499710 @default.
- W2519937269 cites W2148072738 @default.
- W2519937269 cites W2149102934 @default.
- W2519937269 cites W2155822340 @default.
- W2519937269 cites W2161537647 @default.
- W2519937269 cites W2161638299 @default.
- W2519937269 cites W2166819680 @default.
- W2519937269 cites W2166854663 @default.
- W2519937269 cites W2169237458 @default.
- W2519937269 cites W2169276902 @default.
- W2519937269 cites W2177323402 @default.
- W2519937269 cites W2263098028 @default.
- W2519937269 cites W2330486247 @default.
- W2519937269 cites W2395282085 @default.
- W2519937269 cites W75161736 @default.
- W2519937269 cites W2115864709 @default.
- W2519937269 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5374980" @default.
- W2519937269 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/27491211" @default.
- W2519937269 hasPublicationYear "2016" @default.
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