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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Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2019
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Article
Acesso em linha: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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