Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

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 halti...

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Udgivet i:Nat Commun
Main Authors: Gomez-Ospina, Natalia, Scharenberg, Samantha G., Mostrel, Nathalie, Bak, Rasmus O., Mantri, Sruthi, Quadros, Rolen M., Gurumurthy, Channabasavaiah B., Lee, Ciaran, Bao, Gang, Suarez, Carlos J., Khan, Shaukat, Sawamoto, Kazuki, Tomatsu, Shunji, Raj, Nitin, Attardi, Laura D., Aurelian, Laure, Porteus, Matthew H.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6731271/
https://ncbi.nlm.nih.gov/pubmed/31492863
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-019-11962-8
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