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Page 1
Gene Therapy Approaches to Immunodeficiency.
Ghosh S, Gaspar HB. Ghosh S, et al. Hematol Oncol Clin North Am. 2017 Oct;31(5):823-834. doi: 10.1016/j.hoc.2017.05.003. Epub 2017 Jun 29. Hematol Oncol Clin North Am. 2017. PMID: 28895850 Free article. Review.
Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency.
Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Carbonaro DA, et al. Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20. Mol Ther. 2014. PMID: 24256635 Free PMC article.
Gene therapy for primary immunodeficiencies.
Rivat C, Santilli G, Gaspar HB, Thrasher AJ. Rivat C, et al. Hum Gene Ther. 2012 Jul;23(7):668-75. doi: 10.1089/hum.2012.116. Hum Gene Ther. 2012. PMID: 22691036 Free PMC article. Review.
The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity.
Montiel-Equihua CA, Zhang L, Knight S, Saadeh H, Scholz S, Carmo M, Alonso-Ferrero ME, Blundell MP, Monkeviciute A, Schulz R, Collins M, Takeuchi Y, Schmidt M, Fairbanks L, Antoniou M, Thrasher AJ, Gaspar HB. Montiel-Equihua CA, et al. Mol Ther. 2012 Jul;20(7):1400-9. doi: 10.1038/mt.2012.50. Epub 2012 Mar 20. Mol Ther. 2012. PMID: 22434141 Free PMC article.