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2009 3
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Page 1
How I treat ADA deficiency.
Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD. Gaspar HB, et al. Blood. 2009 Oct 22;114(17):3524-32. doi: 10.1182/blood-2009-06-189209. Epub 2009 Jul 28. Blood. 2009. PMID: 19638621 Free PMC article. Review.
Gene therapy for adenosine deaminase deficiency.
Cappelli B, Aiuti A. Cappelli B, et al. Immunol Allergy Clin North Am. 2010 May;30(2):249-60. doi: 10.1016/j.iac.2010.02.003. Immunol Allergy Clin North Am. 2010. PMID: 20493400 Review.
ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency.
Sauer AV, Mrak E, Hernandez RJ, Zacchi E, Cavani F, Casiraghi M, Grunebaum E, Roifman CM, Cervi MC, Ambrosi A, Carlucci F, Roncarolo MG, Villa A, Rubinacci A, Aiuti A. Sauer AV, et al. Blood. 2009 Oct 8;114(15):3216-26. doi: 10.1182/blood-2009-03-209221. Epub 2009 Jul 24. Blood. 2009. PMID: 19633200 Free article. Clinical Trial.