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

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

X-linked hyper-immunoglobulin M (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 expressio...

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Bibliographic Details
Main Authors: Caroline Y. Kuo, Joseph D. Long, Beatriz Campo-Fernandez, Satiro de Oliveira, Aaron R. Cooper, Zulema Romero, Megan D. Hoban, Alok V. Joglekar, Georgia R. Lill, Michael L. Kaufman, Sorel Fitz-Gibbon, Xiaoyan Wang, Roger P. Hollis, Donald B. Kohn
Format: Article
Language:English
Published: Elsevier 2018-05-01
Series:Cell Reports
Subjects:
X-linked hyper-IgM syndrome
gene editing
gene therapy
primary immunodeficiency
CD40 ligand
hematopoietic stem cell
CRISPR/Cas9
TALEN
Online Access:http://www.sciencedirect.com/science/article/pii/S2211124718307010

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http://www.sciencedirect.com/science/article/pii/S2211124718307010

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