CRISPR/Cas9-generated p47phox-deficient cell line for Chronic Granulomatous Disease gene therapy vector development
Author:
Publisher
Springer Science and Business Media LLC
Subject
Multidisciplinary
Link
http://www.nature.com/articles/srep44187.pdf
Reference24 articles.
1. Seger, R. A. Advances in the diagnosis and treatment of chronic granulomatous disease. Curr Opin Hematol 18, 36–41, doi: 10.1097/MOH.0b013e32834115e7 (2011).
2. Gungor, T. et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet 383, 436–448, doi: 10.1016/S0140-6736(13)62069-3 (2014).
3. Ott, M. G. et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 12, 401–409, doi: 10.1038/nm1393 (2006).
4. Siler, U. et al. Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing. Curr Gene Ther 15, 416–427 (2015).
5. Kaufmann, K. B. et al. Gene therapy for chronic granulomatous disease: current status and future perspectives. Curr Gene Ther 14, 447–460 (2014).
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