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CRISPR/Cas9-based double-strand oligonucleotide insertion strategy corrects metabolic abnormalities in murine glycogen storage disease type-Ia.
Samanta, Ananya; George, Nelson; Arnaoutova, Irina; Chen, Hung-Dar; Mansfield, Brian C; Hart, Christopher; Carlo, Troy; Chou, Janice Y.
Affiliation
  • Samanta A; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
  • George N; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
  • Arnaoutova I; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
  • Chen HD; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
  • Mansfield BC; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
  • Chou JY; Section on Cellular Differentiation, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
J Inherit Metab Dis ; 46(6): 1147-1158, 2023 11.
Article in En | MEDLINE | ID: mdl-37467014
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Glycogen Storage Disease Type I Limits: Animals Language: En Journal: J Inherit Metab Dis Year: 2023 Document type: Article Affiliation country: United States Country of publication: United States
Fulltext
Add to My VHL
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligonucleotides / Glycogen Storage Disease Type I Limits: Animals Language: En Journal: J Inherit Metab Dis Year: 2023 Document type: Article Affiliation country: United States Country of publication: United States