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The DOI matches its Crossref record; 8 of the 8 references it cites were found in a registry or an index.

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

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.·2018·Cell Reports
DOI 10.1016/j.celrep.2018.04.103PMC PMC6181643
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Show all 8 references

Research works printed but not linked: 33

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  6. Cooper, 2011, Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration, J. Virol. Methods, 177, 1, 10.1016/j.jviromet.2011.06.019 · DOI
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  9. de la Morena, 2017, Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation, J. Allergy Clin. Immunol., 139, 1282, 10.1016/j.jaci.2016.07.039 · DOI
  10. De Oliveira, 2013, Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy, Hum. Gene Ther., 24, 824, 10.1089/hum.2012.202 · DOI
  11. De Ravin, 2016, Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease, Nat. Biotechnol., 34, 424, 10.1038/nbt.3513 · DOI · Paper page
  12. Doyle, 2012, TAL Effector-Nucleotide Targeter (TALE-NT) 2.0: tools for TAL effector design and target prediction, Nucleic Acids Res., 40, W117, 10.1093/nar/gks608 · DOI
  13. Dull, 1998, A third-generation lentivirus vector with a conditional packaging system, J. Virol., 72, 8463, 10.1128/JVI.72.11.8463-8471.1998 · DOI · Paper page
  14. Epinat, 2003, A novel engineered meganuclease induces homologous recombination in yeast and mammalian cells, Nucleic Acids Res., 31, 2952, 10.1093/nar/gkg375 · DOI
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  16. Gabriel, 2011, An unbiased genome-wide analysis of zinc-finger nuclease specificity, Nat. Biotechnol., 29, 816, 10.1038/nbt.1948 · DOI · Paper page
  17. Gennery, 2004, Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002, Blood, 103, 1152, 10.1182/blood-2003-06-2014 · DOI
  18. Gwiazda, 2016, High efficiency CRISPR/Cas9-mediated gene editing in primary human T-cells using mutant adenoviral E4orf6/E1b55k “helper” proteins, Mol. Ther., 24, 1570, 10.1038/mt.2016.105 · DOI
  19. Hayward, 1997, Cholangiopathy and tumors of the pancreas, liver, and biliary tree in boys with X-linked immunodeficiency with hyper-IgM, J. Immunol., 158, 977, 10.4049/jimmunol.158.2.977 · DOI
  20. Hoban, 2015, Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells, Blood, 125, 2597, 10.1182/blood-2014-12-615948 · DOI · Paper page
  21. Hollenbaugh, 1994, The random inactivation of the X chromosome carrying the defective gene responsible for X-linked hyper IgM syndrome (X-HIM) in female carriers of HIGM1, J. Clin. Invest., 94, 616, 10.1172/JCI117377 · DOI
  22. Hubbard, 2016, Targeted gene editing restores regulated CD40L expression and function in X-linked hyper-IgM syndrome, Blood, 127, 2513, 10.1182/blood-2015-11-683235 · DOI
  23. Levy, 1997, Clinical spectrum of X-linked hyper-IgM syndrome, J. Pediatr., 131, 47, 10.1016/S0022-3476(97)70123-9 · DOI
  24. Mitsui-Sekinaka, 2015, Clinical features and hematopoietic stem cell transplantations for CD40 ligand deficiency in Japan, J. Allergy Clin. Immunol., 136, 1018, 10.1016/j.jaci.2015.02.020 · DOI
  25. Petrovic, 2009, Hematopoietic stem cell transplantation for pediatric patients with primary immunodeficiency diseases at All Children’s Hospital/University of South Florida, Immunol. Res., 44, 169, 10.1007/s12026-009-8111-z · DOI
  26. Romero, 2011, A tissue-specific, activation-inducible, lentiviral vector regulated by human CD40L proximal promoter sequences, Gene Ther., 18, 364, 10.1038/gt.2010.144 · DOI
  27. Sacco, 2000, Lymphoid abnormalities in CD40 ligand transgenic mice suggest the need for tight regulation in gene therapy approaches to hyper immunoglobulin M (IgM) syndrome, Cancer Gene Ther., 7, 1299, 10.1038/sj.cgt.7700232 · DOI
  28. Saifuddin, 2013, Phosphorylation and the regulation of CD40L mRNA stability by polypyrimidine tract binding protein (P1394), J. Immunol., 190, 14, 10.4049/jimmunol.190.Supp.203.14 · DOI
  29. Satomura, 2017, Precise genome-wide base editing by the CRISPR Nickase system in yeast, Sci. Rep., 7, 2095, 10.1038/s41598-017-02013-7 · DOI
  30. Schiroli, 2017, Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1, Sci. Transl. Med., 9, eaan0820, 10.1126/scitranslmed.aan0820 · DOI · Paper page
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Cited by

Metadata

Authors
Kuo, C. Y., Long, J. D., Campo-Fernandez, B., de Oliveira, S., Cooper, A. R., Romero, Z., Hoban, M. D., Joglekar, A. V., Lill, G. R., Kaufman, M. L., Fitz-Gibbon, S., Wang, X., Hollis, R. P., & Kohn, D. B.
ORCID
Kuo, Caroline Y.
Venue
Cell Reports
Publisher
Elsevier BV
Published
2018-05
Volume
23
Issue
9
Pages
2606-2616
ISSN
2211-1247
DOI
10.1016/j.celrep.2018.04.103
PMC
PMC6181643
Cited by
139
References
8
Language
en
Licence
Funders
  • University of California, Los Angeles (K12HD34610-20, K12HD34610-19)
  • California Institute for Regenerative Medicine (DISC2-10124)
  • Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California Los Angeles
  • Australasian Society of Clinical Immunology and Allergy

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