Matches in SemOpenAlex for { <https://semopenalex.org/work/W2971567285> ?p ?o ?g. }
- W2971567285 abstract "Abstract Lysosomal enzyme deficiencies comprise a large group of genetic disorders that generally lack effective treatments. A potential treatment approach is to engineer the patient’s own hematopoietic system to express high levels of the deficient enzyme, thereby correcting the biochemical defect and halting disease progression. Here, we present an efficient ex vivo genome editing approach using CRISPR-Cas9 that targets the lysosomal enzyme iduronidase to the CCR5 safe harbor locus in human CD34+ hematopoietic stem and progenitor cells. The modified cells secrete supra-endogenous enzyme levels, maintain long-term repopulation and multi-lineage differentiation potential, and can improve biochemical and phenotypic abnormalities in an immunocompromised mouse model of Mucopolysaccharidosis type I. These studies provide support for the development of genome-edited CD34+ hematopoietic stem and progenitor cells as a potential treatment for Mucopolysaccharidosis type I. The safe harbor approach constitutes a flexible platform for the expression of lysosomal enzymes making it applicable to other lysosomal storage disorders." @default.
- W2971567285 created "2019-09-12" @default.
- W2971567285 creator A5004317823 @default.
- W2971567285 creator A5007644395 @default.
- W2971567285 creator A5019574388 @default.
- W2971567285 creator A5027181279 @default.
- W2971567285 creator A5030118016 @default.
- W2971567285 creator A5030695798 @default.
- W2971567285 creator A5035110445 @default.
- W2971567285 creator A5038816770 @default.
- W2971567285 creator A5043551591 @default.
- W2971567285 creator A5044985085 @default.
- W2971567285 creator A5051621625 @default.
- W2971567285 creator A5067192778 @default.
- W2971567285 creator A5071597606 @default.
- W2971567285 creator A5072324344 @default.
- W2971567285 creator A5077315209 @default.
- W2971567285 creator A5077591555 @default.
- W2971567285 creator A5081896361 @default.
- W2971567285 date "2019-09-06" @default.
- W2971567285 modified "2023-10-18" @default.
- W2971567285 title "Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I" @default.
- W2971567285 cites W1481961971 @default.
- W2971567285 cites W1906870688 @default.
- W2971567285 cites W1972144548 @default.
- W2971567285 cites W1977955083 @default.
- W2971567285 cites W1980888760 @default.
- W2971567285 cites W1988809467 @default.
- W2971567285 cites W1994657204 @default.
- W2971567285 cites W1995497292 @default.
- W2971567285 cites W1995979918 @default.
- W2971567285 cites W1999203943 @default.
- W2971567285 cites W2001556991 @default.
- W2971567285 cites W2008147758 @default.
- W2971567285 cites W2013609666 @default.
- W2971567285 cites W2016745966 @default.
- W2971567285 cites W2024018223 @default.
- W2971567285 cites W2024999114 @default.
- W2971567285 cites W2035445362 @default.
- W2971567285 cites W2040036069 @default.
- W2971567285 cites W2041626130 @default.
- W2971567285 cites W2058352256 @default.
- W2971567285 cites W2064497500 @default.
- W2971567285 cites W2066099080 @default.
- W2971567285 cites W2072299899 @default.
- W2971567285 cites W2081409454 @default.
- W2971567285 cites W2089319449 @default.
- W2971567285 cites W2090630729 @default.
- W2971567285 cites W2091140839 @default.
- W2971567285 cites W2097300250 @default.
- W2971567285 cites W2100174615 @default.
- W2971567285 cites W2109879947 @default.
- W2971567285 cites W2110978695 @default.
- W2971567285 cites W2116008939 @default.
- W2971567285 cites W2123587476 @default.
- W2971567285 cites W2126590211 @default.
- W2971567285 cites W2136255603 @default.
- W2971567285 cites W2140757379 @default.
- W2971567285 cites W2141570423 @default.
- W2971567285 cites W2142776719 @default.
- W2971567285 cites W2145254159 @default.
- W2971567285 cites W2154986930 @default.
- W2971567285 cites W2163248265 @default.
- W2971567285 cites W2165934664 @default.
- W2971567285 cites W2166658660 @default.
- W2971567285 cites W2209402024 @default.
- W2971567285 cites W2230199130 @default.
- W2971567285 cites W2275463106 @default.
- W2971567285 cites W2293120410 @default.
- W2971567285 cites W2317788533 @default.
- W2971567285 cites W2408400092 @default.
- W2971567285 cites W2471587598 @default.
- W2971567285 cites W2527156470 @default.
- W2971567285 cites W2547418311 @default.
- W2971567285 cites W2592778503 @default.
- W2971567285 cites W2759699238 @default.
- W2971567285 cites W2761956671 @default.
- W2971567285 cites W2764213443 @default.
- W2971567285 cites W2767634557 @default.
- W2971567285 cites W2777434339 @default.
- W2971567285 cites W2784423079 @default.
- W2971567285 cites W2791745527 @default.
- W2971567285 cites W2806820493 @default.
- W2971567285 cites W2807198924 @default.
- W2971567285 cites W2808076791 @default.
- W2971567285 cites W2886819502 @default.
- W2971567285 cites W2902840690 @default.
- W2971567285 cites W2912280590 @default.
- W2971567285 cites W2924385776 @default.
- W2971567285 cites W2947535260 @default.
- W2971567285 doi "https://doi.org/10.1038/s41467-019-11962-8" @default.
- W2971567285 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/6731271" @default.
- W2971567285 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/31492863" @default.
- W2971567285 hasPublicationYear "2019" @default.
- W2971567285 type Work @default.
- W2971567285 sameAs 2971567285 @default.
- W2971567285 citedByCount "79" @default.
- W2971567285 countsByYear W29715672852020 @default.
- W2971567285 countsByYear W29715672852021 @default.
- W2971567285 countsByYear W29715672852022 @default.