References
If you want to know more here you will find all the references, reviews, websites, videos and links that I used to find all information about Gene Therapy and Severe Combined Immunodeficiency.
Bibliography
[1] Kildebeck, E. et al. Gene therapy for primary immunodeficiencies. Curr Opin Pediatr. 2012 Dec; 24(6):731-8.
[2] Lev, A. et al. Characterizing T cells in SCID patients presenting with reactive or residual T lymphocytes. Clin Dev Immunol. 2012; 2012:261470.
[3] Barzaghi, F. et al. Immune dysregulation, polyendocrinopathy, enteropathy, x-linked syndrome: a paradigm of immunodeficiency with autoimmunity. Front Immunol. 2012; 3:211.
[4] Sauer, AV. et al. Autoimmune dysregulation and purine metabolism in adenosine deaminase deficiency. Front Immunol. 2012; 3:265.
[5] Barese, CN. et al. Contributions of gene marking to cell and gene therapies. Hum Gene Ther. 2011 Jun; 22(6):659-68.
[6] Agostinelli, C. et al. Peripheral T cell lymphoma, not otherwise specified: the stuff of genes, dreams and therapies. J Clin Pathol. 2008 Nov; 61(11):1160-7.
[7] Romano, G. et al. Development of safer gene delivery systems to minimize the risk of insertional mutagenesis-related malignancies: a critical issue for the field of gene therapy. ISRN Oncol. 2012; 2012:616310.
[8] Baum, C. et al. Gene therapy for SCID-X1: focus on clinical data. Mol Ther. 2011 Dec; 19(12):2103-4.
[9] Wunderli, W. et al. Astrovirus infection in hospitalized infants with severe combined immunodeficiency after allogeneic hematopoietic stem cell transplantation. PLoS One. 2011; 6(11):e27483.
[10] Allenspach, E. et al. X-Linked Severe Combined Immunodeficiency. GeneReviews. 2003 Aug.
[11] Kaneda, Y. et al. Progress of human gene therapy. Nihon Rinsho. 2009 Jun; 67(6):1180-4.
more...
[12] Blaese, RM. et al. What is the status of gene therapy for primary immunodeficiency? Immunol Res. 2007; 38(1-3):274-84.
[13] Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000 Apr 28; 288(5466):669-72.
[14] Aloj, G. et al. Severe combined immunodeficiences: new and old scenarios. Int Rev Immunol. 2012 Feb; 31(1):43-65.
[15] Fischer, A. et al. 20 years of gene therapy for SCID. Nat Immunol. 2010 Jun; 11(6):457-60.
[16] Hacein-Bey-Abina, S. et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 2008; 118:3132–3142.
[17] Howe, SJ. et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest. 2008 Sep; 118(9):3143-50.
[18] Naldini L. Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet. 2011 May; 12(5):301-15.
[19] Gaspar, H. B. et al. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol. Ther. 2006; 14:505–513.
[20] Hacein-Bey-Abina, S. et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 2010; 363:355–364.
[21] Aiuti, A. et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002 Jun.
[22] John T, Gray. et al. The Effect of the B2 Mutation on Hematopoietic Reconstitution in the French X-SCID Gene Therapy Trial. Molecular Therapy 2006; 13:S254.
Links and websites
The National Patient Organization for Primary Immunodeficiency Diseases
Boston Children’s Hospital
http://www.childrenshospital.org/az/Site1577/mainpageS1577P0.html
Institute of child health:
http://www.ucl.ac.uk/ich/research-ich/immunobiology
Severe Combined Immunodeficiency
http://www.scid.net/foundations.htm
Kids Health
http://kidshealth.org/parent/medical/allergies/severe_immunodeficiency.html#
