Candotti Publications

National Human Genome Research Institute

National Institutes of Health
U.S. Department of Health and Human Services

Fabio Candotti, M.D.

Senior Investigator, Genetics and Molecular Biology Branch
Head, Disorders of Immunity Section

Selected Publications

Muul L.M., Tuschong L.M., Soenen S.L., Jagadeesh G.J., Ramsey W.J., Long Z., Carter C.S., Garabedian E.K., Alleyne M., Brown M., Bernstein W., Schurman S.H., Fleisher T.A., Leitman S.F., Dunbar C.E., Blaese R.M., Candotti F. Persistence and expression of the adenosine deaminase gene for twelve years and immune reaction to gene transfer components: Long-term results of the first clinical gene therapy trial. Blood, 101:2563-2569. 2003. [PubMed]

Wada T., Konno A., Schurman S.H., Garabedian E.K., Anderson S.M., Kirby M., Nelson D.L., Candotti F.: Second-site mutation in the Wiskott-Aldrich syndrome (WAS) protein gene causes somatic mosaicism in two WAS siblings. J Clin Invest, 111:1389-1397. 2003. [PubMed]

Konno A., Wada T., Schurman S.H., Garabedian E.K., Kirby M., Anderson S.M., Candotti F. Differential contribution of Wiskott-Aldrich syndrome protein to selective advantage in T- and B-cell lineages. Blood, 103(2):676-8. 2004. [PubMed]

Wada T., Schurman S.H., Jagadeesh G.J., Garabedian E.K., Nelson D.L., Candotti F. Multiple patients with revertant mosaicism in a single Wiskott-Aldrich syndrome family. Blood, 104(5):1270-2. 2004. [PubMed]

Fieschi C., Bosticardo M., de Beaucoudrey L., Dupuis S., Feinberg J., dos Santos O., Bustamante J., Levy J., Candotti F., Casanova J.L. A novel form of complete IL-12/IL-23 receptor 1-deficiency with cell surfaceexpressed non-functional receptors. Blood, 104:2095-2101. 2004. [PubMed]

Wada T., Schurman S.H., Garabedian E.K., Yachie A., Candotti F. Analysis of T-cell repertoire diversity in Wiskott-Aldrich syndrome. Blood, 106(12):3895-7. 2005. [PubMed]

Puck, J.M., Candotti, F., Lessons from the Wiskott-Aldrich syndrome. N Engl J Med, 355(17):1759-61. 2006. [FullText]

Konno, A., Kirby, M., Anderson, S.A., Schwartzberg, P.L., Candotti, F. The expression of Wiskott-Aldrich syndrome protein (WASP) is dependent on WASP-interacting protein (WIP). Int Immunol, 19(2):185-92. 2007. [PubMed]

Adriani, M., Aoki, J., Horai, R., Thornton, A.M., Konno, A., Kirby, M., Anderson, S.M., Siegel, R.M., Candotti, F., Schwartzberg, P.L. Impaired in vitro regulatory T cell function associated with Wiskott-Aldrich syndrome. Clin Immunol, 124(1):41-8. 2007 . [PubMed]

Lagresle-Peyrou C., Six E.M., Picard C., Rieux-Laucat F., Michel V., Ditadi A., Demerens-de Chappedelaine C., Morillon E., Valensi F., Simon-Stoos K.L., Mullikin J.C., Noroski L.M., Besse C., Wulffraat N.M., Ferster A., Abecasis M.M., Calvo F., Petit C., Candotti F., Abel L., Fischer A., Cavazzana-Calvo M. Human adenylate kinase 2 deficiency causes a profound hematopoietic defect associated with sensorineural deafness. Nat Genet, 41:106-11. 2009. [PubMed]

Kohn D.B., Candotti F. Gene therapy fulfilling its promise. N Engl J Med, 360:518-521.43. 2009. [PubMed]

Bosticardo M, Ghosh A, Du Y, Jenkins NA, Copeland NG, Candotti F. Self-inactivating retroviral vector-mediated gene transfer induces oncogene activation and immortalization of primary murine bone marrow cells. Mol Ther, 17(11):1910-8. 2009. [PubMed]

Davis BR, Yan Q, Bui JH, Felix K, Moratto D, Muul LM, Prokopishyn NL, Blaese RM, Candotti F. Somatic mosaicism in the Wiskott-Aldrich syndrome: molecular and functional characterization of genotypic revertants. Clin Immunol, 135(1):72-83. 2010. [PubMed]

Nikolov NP, Shimizu M, Cleland S, Bailey D, Aoki J, Strom T, Schwartzberg PL, Candotti F, Siegel RM. Systemic autoimmunity and defective Fas ligand secretion in the absence of the Wiskott-Aldrich syndrome protein. Blood, 2010 May 10. Epub ahead of print. [PubMed]

Taylor MD, Sadhukhan S, Kottangada P, Ramgopal A, Sarkar K, D'Silva S, Selvakumar A, Candotti F, Vyas YM. Nuclear role of WASp in the pathogenesis of dysregulated TH1 immunity in human Wiskott-Aldrich syndrome. Sci Transl Med, 2(37):37ra44. 2010. [PubMed]

Adriani M., Jones K.A., Uchiyama T., Kirby M., Silvin C., Anderson S., Candotti F. Defective inhibition of B-cell proliferation by Wiskott-Aldrich syndrome protein-deficient regulatory T cells. Blood, 117:6608-11. 2011. [PubMed]

Sokolic R., Maric I., Kesserwan C., Garabedian E., Hanson C.I., Dodds M., Buckley R., Issekutz A., Kamani N., Shaw K., Tan B., Bali P., Hershfield M.S., Kohn D.B., Wayne A.S., Candotti F. Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood, 118:2688-94. 2011. [PubMed]

Uchiyama T., Adriani M., Jagadeesh G.J., Paine A., Candotti F. Foamy virus vector-mediated gene correction of a mouse model of Wiskott-Aldrich syndrome. Mol Ther, 2012. [In Press]

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Last Reviewed: June 26, 2013