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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6731271/
https://www.ncbi.nlm.nih.gov/pubmed/31492863
http://dx.doi.org/10.1038/s41467-019-11962-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6731271/
https://www.ncbi.nlm.nih.gov/pubmed/31492863
http://dx.doi.org/10.1038/s41467-019-11962-8

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