FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W3015420073> ?p ?o ?g. }

• W3015420073 endingPage "2642" @default.
• W3015420073 startingPage "2642" @default.
• W3015420073 abstract "Pompe disease is an autosomal recessive lysosomal storage disorder (LSD) caused by deficiency of lysosomal acid alpha-glucosidase (GAA). The result of the GAA deficiency is a ubiquitous lysosomal and non-lysosomal accumulation of glycogen. The most affected tissues are heart, skeletal muscle, liver, and the nervous system. Replacement therapy with the currently approved enzyme relies on M6P-mediated endocytosis. However, therapeutic outcomes still leave room for improvement, especially with regard to skeletal muscles. We tested the uptake, activity, and effect on glucose metabolism of a non-phosphorylated recombinant human GAA produced in moss (moss-GAA). Three variants of moss-GAA differing in glycosylation pattern have been analyzed: two with terminal mannose residues in a paucimannosidic (Man3) or high-mannose (Man 5) configuration and one with terminal N-acetylglucosamine residues (GnGn). Compared to alglucosidase alfa the moss-GAA GnGn variant showed increased uptake in differentiated myotubes. Moreover, incubation of immortalized muscle cells of Gaa−/− mice with moss-GAA GnGn led to similarly efficient clearance of accumulated glycogen as with alglucosidase alfa. These initial data suggest that M6P-residues might not always be necessary for the cellular uptake in enzyme replacement therapy (ERT) and indicate the potential of moss-GAA GnGn as novel alternative drug for targeting skeletal muscle in Pompe patients." @default.
• W3015420073 created "2020-04-17" @default.
• W3015420073 creator A5001990831 @default.
• W3015420073 creator A5003746621 @default.
• W3015420073 creator A5009199568 @default.
• W3015420073 creator A5022121355 @default.
• W3015420073 creator A5035612494 @default.
• W3015420073 creator A5037913853 @default.
• W3015420073 creator A5039404447 @default.
• W3015420073 creator A5071615321 @default.
• W3015420073 creator A5088000243 @default.
• W3015420073 date "2020-04-10" @default.
• W3015420073 modified "2023-10-18" @default.
• W3015420073 title "Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease" @default.
• W3015420073 cites W1898679873 @default.
• W3015420073 cites W1968391529 @default.
• W3015420073 cites W1998733027 @default.
• W3015420073 cites W2000400288 @default.
• W3015420073 cites W2003880659 @default.
• W3015420073 cites W2011287480 @default.
• W3015420073 cites W2062305874 @default.
• W3015420073 cites W2062886670 @default.
• W3015420073 cites W2073434167 @default.
• W3015420073 cites W2083805206 @default.
• W3015420073 cites W2084210525 @default.
• W3015420073 cites W2089909382 @default.
• W3015420073 cites W2098331662 @default.
• W3015420073 cites W2103986567 @default.
• W3015420073 cites W2122377406 @default.
• W3015420073 cites W2127608615 @default.
• W3015420073 cites W2135689611 @default.
• W3015420073 cites W2312883876 @default.
• W3015420073 cites W2469663448 @default.
• W3015420073 cites W2538968033 @default.
• W3015420073 cites W2562247590 @default.
• W3015420073 cites W2611287306 @default.
• W3015420073 cites W2766582923 @default.
• W3015420073 cites W2767691022 @default.
• W3015420073 cites W2789403551 @default.
• W3015420073 cites W2789917827 @default.
• W3015420073 cites W2886388595 @default.
• W3015420073 cites W2914730328 @default.
• W3015420073 cites W2941370568 @default.
• W3015420073 doi "https://doi.org/10.3390/ijms21072642" @default.
• W3015420073 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/7177967" @default.
• W3015420073 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/32290314" @default.
• W3015420073 hasPublicationYear "2020" @default.
• W3015420073 type Work @default.
• W3015420073 sameAs 3015420073 @default.
• W3015420073 citedByCount "15" @default.
• W3015420073 countsByYear W30154200732020 @default.
• W3015420073 countsByYear W30154200732021 @default.
• W3015420073 countsByYear W30154200732022 @default.
• W3015420073 countsByYear W30154200732023 @default.
• W3015420073 crossrefType "journal-article" @default.
• W3015420073 hasAuthorship W3015420073A5001990831 @default.
• W3015420073 hasAuthorship W3015420073A5003746621 @default.
• W3015420073 hasAuthorship W3015420073A5009199568 @default.
• W3015420073 hasAuthorship W3015420073A5022121355 @default.
• W3015420073 hasAuthorship W3015420073A5035612494 @default.
• W3015420073 hasAuthorship W3015420073A5037913853 @default.
• W3015420073 hasAuthorship W3015420073A5039404447 @default.
• W3015420073 hasAuthorship W3015420073A5071615321 @default.
• W3015420073 hasAuthorship W3015420073A5088000243 @default.
• W3015420073 hasBestOaLocation W30154200731 @default.
• W3015420073 hasConcept C104317684 @default.
• W3015420073 hasConcept C126322002 @default.
• W3015420073 hasConcept C134018914 @default.
• W3015420073 hasConcept C170493617 @default.
• W3015420073 hasConcept C181199279 @default.
• W3015420073 hasConcept C2775887612 @default.
• W3015420073 hasConcept C2776735236 @default.
• W3015420073 hasConcept C2777499176 @default.
• W3015420073 hasConcept C2778264360 @default.
• W3015420073 hasConcept C2779134260 @default.
• W3015420073 hasConcept C2779468605 @default.
• W3015420073 hasConcept C2779959927 @default.
• W3015420073 hasConcept C2779969927 @default.
• W3015420073 hasConcept C2780888338 @default.
• W3015420073 hasConcept C28005876 @default.
• W3015420073 hasConcept C40767141 @default.
• W3015420073 hasConcept C55493867 @default.
• W3015420073 hasConcept C59822182 @default.
• W3015420073 hasConcept C71924100 @default.
• W3015420073 hasConcept C86803240 @default.
• W3015420073 hasConceptScore W3015420073C104317684 @default.
• W3015420073 hasConceptScore W3015420073C126322002 @default.
• W3015420073 hasConceptScore W3015420073C134018914 @default.
• W3015420073 hasConceptScore W3015420073C170493617 @default.
• W3015420073 hasConceptScore W3015420073C181199279 @default.
• W3015420073 hasConceptScore W3015420073C2775887612 @default.
• W3015420073 hasConceptScore W3015420073C2776735236 @default.
• W3015420073 hasConceptScore W3015420073C2777499176 @default.
• W3015420073 hasConceptScore W3015420073C2778264360 @default.
• W3015420073 hasConceptScore W3015420073C2779134260 @default.
• W3015420073 hasConceptScore W3015420073C2779468605 @default.
• W3015420073 hasConceptScore W3015420073C2779959927 @default.
• W3015420073 hasConceptScore W3015420073C2779969927 @default.

• next