FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2790249820> ?p ?o ?g. }

• W2790249820 endingPage "1136" @default.
• W2790249820 startingPage "1127" @default.
• W2790249820 abstract "Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal disorder caused by deficiency of iduronate 2-sulfatase (IDS), leading to accumulation of glycosaminoglycans (GAGs) in tissues of affected individuals, progressive disease, and shortened lifespan. Currently available enzyme replacement therapy (ERT) requires lifelong infusions and does not provide neurologic benefit. We utilized a zinc finger nuclease (ZFN)-targeting system to mediate genome editing for insertion of the human IDS (hIDS) coding sequence into a “safe harbor” site, intron 1 of the albumin locus in hepatocytes of an MPS II mouse model. Three dose levels of recombinant AAV2/8 vectors encoding a pair of ZFNs and a hIDS cDNA donor were administered systemically in MPS II mice. Supraphysiological, vector dose-dependent levels of IDS enzyme were observed in the circulation and peripheral organs of ZFN+donor-treated mice. GAG contents were markedly reduced in tissues from all ZFN+donor-treated groups. Surprisingly, we also demonstrate that ZFN-mediated genome editing prevented the development of neurocognitive deficit in young MPS II mice (6–9 weeks old) treated at high vector dose levels. We conclude that this ZFN-based platform for expression of therapeutic proteins from the albumin locus is a promising approach for treatment of MPS II and other lysosomal diseases." @default.
• W2790249820 created "2018-03-29" @default.
• W2790249820 creator A5002283680 @default.
• W2790249820 creator A5011032269 @default.
• W2790249820 creator A5016591941 @default.
• W2790249820 creator A5036401265 @default.
• W2790249820 creator A5051543327 @default.
• W2790249820 creator A5064245536 @default.
• W2790249820 creator A5064433009 @default.
• W2790249820 creator A5065159714 @default.
• W2790249820 creator A5069563469 @default.
• W2790249820 creator A5070829500 @default.
• W2790249820 creator A5073932029 @default.
• W2790249820 creator A5079503723 @default.
• W2790249820 creator A5083375634 @default.
• W2790249820 creator A5087254418 @default.
• W2790249820 creator A5089507101 @default.
• W2790249820 creator A5091741284 @default.
• W2790249820 date "2018-04-01" @default.
• W2790249820 modified "2023-10-15" @default.
• W2790249820 title "Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing" @default.
• W2790249820 cites W1480125634 @default.
• W2790249820 cites W1481961971 @default.
• W2790249820 cites W1559407499 @default.
• W2790249820 cites W1969271703 @default.
• W2790249820 cites W1980449150 @default.
• W2790249820 cites W1985676321 @default.
• W2790249820 cites W1988426813 @default.
• W2790249820 cites W1993518098 @default.
• W2790249820 cites W1995497292 @default.
• W2790249820 cites W2005101164 @default.
• W2790249820 cites W2008784634 @default.
• W2790249820 cites W2038866304 @default.
• W2790249820 cites W2041607260 @default.
• W2790249820 cites W2045158993 @default.
• W2790249820 cites W2051833330 @default.
• W2790249820 cites W2055909681 @default.
• W2790249820 cites W2059243802 @default.
• W2790249820 cites W2074231493 @default.
• W2790249820 cites W2082721633 @default.
• W2790249820 cites W2091134968 @default.
• W2790249820 cites W2097514370 @default.
• W2790249820 cites W2116369307 @default.
• W2790249820 cites W2120305385 @default.
• W2790249820 cites W2147640566 @default.
• W2790249820 cites W2149353660 @default.
• W2790249820 doi "https://doi.org/10.1016/j.ymthe.2018.03.002" @default.
• W2790249820 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/6080131" @default.
• W2790249820 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/29580682" @default.
• W2790249820 hasPublicationYear "2018" @default.
• W2790249820 type Work @default.
• W2790249820 sameAs 2790249820 @default.
• W2790249820 citedByCount "97" @default.
• W2790249820 countsByYear W27902498202018 @default.
• W2790249820 countsByYear W27902498202019 @default.
• W2790249820 countsByYear W27902498202020 @default.
• W2790249820 countsByYear W27902498202021 @default.
• W2790249820 countsByYear W27902498202022 @default.
• W2790249820 countsByYear W27902498202023 @default.
• W2790249820 crossrefType "journal-article" @default.
• W2790249820 hasAuthorship W2790249820A5002283680 @default.
• W2790249820 hasAuthorship W2790249820A5011032269 @default.
• W2790249820 hasAuthorship W2790249820A5016591941 @default.
• W2790249820 hasAuthorship W2790249820A5036401265 @default.
• W2790249820 hasAuthorship W2790249820A5051543327 @default.
• W2790249820 hasAuthorship W2790249820A5064245536 @default.
• W2790249820 hasAuthorship W2790249820A5064433009 @default.
• W2790249820 hasAuthorship W2790249820A5065159714 @default.
• W2790249820 hasAuthorship W2790249820A5069563469 @default.
• W2790249820 hasAuthorship W2790249820A5070829500 @default.
• W2790249820 hasAuthorship W2790249820A5073932029 @default.
• W2790249820 hasAuthorship W2790249820A5079503723 @default.
• W2790249820 hasAuthorship W2790249820A5083375634 @default.
• W2790249820 hasAuthorship W2790249820A5087254418 @default.
• W2790249820 hasAuthorship W2790249820A5089507101 @default.
• W2790249820 hasAuthorship W2790249820A5091741284 @default.
• W2790249820 hasBestOaLocation W27902498201 @default.
• W2790249820 hasConcept C104317684 @default.
• W2790249820 hasConcept C141231307 @default.
• W2790249820 hasConcept C142724271 @default.
• W2790249820 hasConcept C144501496 @default.
• W2790249820 hasConcept C207001950 @default.
• W2790249820 hasConcept C2779134260 @default.
• W2790249820 hasConcept C35802184 @default.
• W2790249820 hasConcept C54355233 @default.
• W2790249820 hasConcept C70721500 @default.
• W2790249820 hasConcept C71924100 @default.
• W2790249820 hasConcept C86803240 @default.
• W2790249820 hasConcept C95444343 @default.
• W2790249820 hasConceptScore W2790249820C104317684 @default.
• W2790249820 hasConceptScore W2790249820C141231307 @default.
• W2790249820 hasConceptScore W2790249820C142724271 @default.
• W2790249820 hasConceptScore W2790249820C144501496 @default.
• W2790249820 hasConceptScore W2790249820C207001950 @default.
• W2790249820 hasConceptScore W2790249820C2779134260 @default.
• W2790249820 hasConceptScore W2790249820C35802184 @default.
• W2790249820 hasConceptScore W2790249820C54355233 @default.
• W2790249820 hasConceptScore W2790249820C70721500 @default.

• next