FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2037220161> ?p ?o ?g. }

• W2037220161 endingPage "687" @default.
• W2037220161 startingPage "679" @default.
• W2037220161 abstract "Farber disease is a rare lysosomal storage disorder (LSD) that manifests due to acid ceramidase (AC) deficiencies and ceramide accumulation. We present a preclinical gene therapy study for Farber disease employing a lentiviral vector (LV-huAC/huCD25) in three enzymatically normal nonhuman primates. Autologous, mobilized peripheral blood (PB) cells were transduced and infused into fully myelo-ablated recipients with tracking for at least 1 year. Outcomes were assessed by measuring the AC specific activity, ceramide levels, vector persistence/integration, and safety parameters. We observed no hematological, biochemical, radiological, or pathological abnormalities. Hematological recovery occurred by approximately 3 weeks. Vector persistence was observed in PB and bone marrow (BM) cells by qualitative and quantitative PCR. We did not observe any clonal proliferation of PB and BM cells. Importantly, AC-specific activity was detected above normal levels in PB and BM cells analyzed post-transplantation and in spleens and livers at the endpoint of the study. Decreases of ceramide in PB cells as well as in spleen and liver tissues were seen. We expect that this study will provide a roadmap for implementation of clinical gene therapy protocols targeting hematopoietic cells for Farber disease and other LSDs." @default.
• W2037220161 created "2016-06-24" @default.
• W2037220161 creator A5008048551 @default.
• W2037220161 creator A5013734332 @default.
• W2037220161 creator A5018270628 @default.
• W2037220161 creator A5024793393 @default.
• W2037220161 creator A5033158463 @default.
• W2037220161 creator A5039160738 @default.
• W2037220161 creator A5041626712 @default.
• W2037220161 creator A5044250345 @default.
• W2037220161 creator A5065002010 @default.
• W2037220161 creator A5072705132 @default.
• W2037220161 creator A5073545182 @default.
• W2037220161 creator A5079519987 @default.
• W2037220161 creator A5090732038 @default.
• W2037220161 date "2011-06-01" @default.
• W2037220161 modified "2023-10-06" @default.
• W2037220161 title "Autologous Transplantation of Lentivector/Acid Ceramidase–Transduced Hematopoietic Cells in Nonhuman Primates" @default.
• W2037220161 cites W145486817 @default.
• W2037220161 cites W1966394929 @default.
• W2037220161 cites W1967750537 @default.
• W2037220161 cites W1975703199 @default.
• W2037220161 cites W1988475564 @default.
• W2037220161 cites W1999015381 @default.
• W2037220161 cites W2006351191 @default.
• W2037220161 cites W2012516911 @default.
• W2037220161 cites W2013519009 @default.
• W2037220161 cites W2048573643 @default.
• W2037220161 cites W2048856089 @default.
• W2037220161 cites W2063781737 @default.
• W2037220161 cites W2066146740 @default.
• W2037220161 cites W2066334259 @default.
• W2037220161 cites W2077649301 @default.
• W2037220161 cites W2086922763 @default.
• W2037220161 cites W2105358704 @default.
• W2037220161 cites W2121882313 @default.
• W2037220161 cites W2137560033 @default.
• W2037220161 cites W2141210116 @default.
• W2037220161 cites W2161347101 @default.
• W2037220161 cites W2179786763 @default.
• W2037220161 cites W2180507994 @default.
• W2037220161 cites W2332428851 @default.
• W2037220161 cites W4245896511 @default.
• W2037220161 cites W2072919270 @default.
• W2037220161 doi "https://doi.org/10.1089/hum.2010.195" @default.
• W2037220161 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3155125" @default.
• W2037220161 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/21280983" @default.
• W2037220161 hasPublicationYear "2011" @default.
• W2037220161 type Work @default.
• W2037220161 sameAs 2037220161 @default.
• W2037220161 citedByCount "32" @default.
• W2037220161 countsByYear W20372201612012 @default.
• W2037220161 countsByYear W20372201612013 @default.
• W2037220161 countsByYear W20372201612014 @default.
• W2037220161 countsByYear W20372201612015 @default.
• W2037220161 countsByYear W20372201612016 @default.
• W2037220161 countsByYear W20372201612017 @default.
• W2037220161 countsByYear W20372201612018 @default.
• W2037220161 countsByYear W20372201612019 @default.
• W2037220161 countsByYear W20372201612020 @default.
• W2037220161 countsByYear W20372201612021 @default.
• W2037220161 countsByYear W20372201612022 @default.
• W2037220161 countsByYear W20372201612023 @default.
• W2037220161 crossrefType "journal-article" @default.
• W2037220161 hasAuthorship W2037220161A5008048551 @default.
• W2037220161 hasAuthorship W2037220161A5013734332 @default.
• W2037220161 hasAuthorship W2037220161A5018270628 @default.
• W2037220161 hasAuthorship W2037220161A5024793393 @default.
• W2037220161 hasAuthorship W2037220161A5033158463 @default.
• W2037220161 hasAuthorship W2037220161A5039160738 @default.
• W2037220161 hasAuthorship W2037220161A5041626712 @default.
• W2037220161 hasAuthorship W2037220161A5044250345 @default.
• W2037220161 hasAuthorship W2037220161A5065002010 @default.
• W2037220161 hasAuthorship W2037220161A5072705132 @default.
• W2037220161 hasAuthorship W2037220161A5073545182 @default.
• W2037220161 hasAuthorship W2037220161A5079519987 @default.
• W2037220161 hasAuthorship W2037220161A5090732038 @default.
• W2037220161 hasBestOaLocation W20372201612 @default.
• W2037220161 hasConcept C104317684 @default.
• W2037220161 hasConcept C109159458 @default.
• W2037220161 hasConcept C111599444 @default.
• W2037220161 hasConcept C126322002 @default.
• W2037220161 hasConcept C142724271 @default.
• W2037220161 hasConcept C190283241 @default.
• W2037220161 hasConcept C203014093 @default.
• W2037220161 hasConcept C2777408962 @default.
• W2037220161 hasConcept C2777851122 @default.
• W2037220161 hasConcept C2778264360 @default.
• W2037220161 hasConcept C2779134260 @default.
• W2037220161 hasConcept C2779994418 @default.
• W2037220161 hasConcept C2780007613 @default.
• W2037220161 hasConcept C2780674200 @default.
• W2037220161 hasConcept C2780931953 @default.
• W2037220161 hasConcept C28328180 @default.
• W2037220161 hasConcept C2911091166 @default.
• W2037220161 hasConcept C32470452 @default.
• W2037220161 hasConcept C40767141 @default.
• W2037220161 hasConcept C502942594 @default.

• next