Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome

X-linked hyper-IgM syndrome (XHIM) is a primary immunodeficiency due to mutations in CD40 ligand that affect immunoglobulin class switch recombination and somatic hypermutation. The disease is amenable to gene therapy using retroviral vectors, but dysregulated gene expression resulted in abnormal ly...

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Detalhes bibliográficos
Publicado no:Cell Rep
Main Authors: Kuo, Caroline Y., Long, Joseph D., Campo-Fernandez, Beatriz, de Oliveira, Satiro, Cooper, Aaron R., Romero, Zulema, Hoban, Megan D., Joglekar, Alok V., Lill, Georgia R., Kaufman, Michael L., Fitz-Gibbon, Sorel, Wang, Xiaoyan, Hollis, Roger P., Kohn, Donald B.
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6181643/
https://ncbi.nlm.nih.gov/pubmed/29847792
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.celrep.2018.04.103
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