New developments in gene therapy

  • Katarina Sedej University Medical Centre Ljubljana, Bohoričeva 20, Ljubljana
  • Primož Rožman Blood Transfusion Centre, Šlajmerjeva 6, Ljubljana
Keywords: gene therapy, inherited genetic disease, vectors for gene therapy, hematopoietic stem cells, advanced therapies

Abstract

The article is a review of recent successes in the field of gene therapy of genetic diseases. The emphasis is on genetic diseases where gene therapy has already been applied successfully.

Downloads

Download data is not yet available.

References

Rožman P, Jež M. Matične celice in napredno zdravljenje (zdravljenje s celicami, gensko zdravljenje in tkivno inženirstvo) - Pojmovnik. Ljubljana: Društvo za celično in tkivno inženirstvo Slovenije; 2010.

Selkirk SM. Gene therapy in clinical medicine. Postgrad Med J 2004; 80: 560-70.

Nayerossadat N, Maedeh T, Abas Ali P. Viral and nonviral delivery systems for gene delivery. Adv Biomed Res 2012; 1: 27.

Prud'homme GJ, Glinka Y, Khan AS, Draghia-Akli R. Electroporation-enhanced nonviral gene transfer for the prevention or treatment of immunological, endocrine and neoplastic diseases. Curr Gene Ther 2006; 6(2): 243-73.

Murakami T, Sunada Y. Plasmid DNA gene therapy by electroporation: principles and recent advances. Curr Gene Ther 2011; 11(6): 447-56.

Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, et al. Gene transfer into humans-immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990; 323(9): 570-8.

Sheridan C. Gene therapy finds its niche. Nat Biotechnol 2011; 29(2): 121-8.

Edelstein ML, Abedi MR, Wixon J, Edelstein RM. Gene therapy clinical trials worldwide 1989-2004-an overview. J Gene Med 2004; 6: 597-602.

The Journal of Gene Medicine Clinical Trial. Dosegljivo na: http://www.wiley.co.uk/genmed/clinical .

Ginn SL, Alexander IE, Edelstein ML, Abedi MR, Wixon J. Gene therapy clinical trials worldwide to 2012 - an update. J Gene Med 2013; 15(2): 65-77.

Nijman IJ, van Montfrans JM, Hoogstraat M, Boes ML, van de Corput L, Renner ED. Targeted next-generation sequencing: A novel diagnostic tool for primary immunodeficiencies. J Allergy Clin Immunol okt 2013. Dosegljivo na: www.science direct.com

Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, et al. Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood 2012; 120(18): 3635-46.

Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360(5): 447-58.

Aiuti A, Roncarolo MG. Ten years of gene therapy for primary immune deficiencies. Hematology Am Soc Hematol Educ Program 2009: 682-9.

Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008; 118(9): 3143-50.

Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2010; 363(4): 355-64.

Herzog RW. Gene Therapy for SCID-X1: Round 2. Mol Ther 2010; 18(11): 1891.

Nihuis AW. Development of gene therapy for blood disorders: an update. Blood 2013; 122(9): 1556-64.

Rivat C, Santilli G, Gaspar HB, Thrasher AJ. Gene therapy for primary immunodeficiencies. Hum Gene Ther 2012; 23(7): 668-75.

Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration. Mol Ther 2010; 18(3): 643–650.

Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Bougnères P, Schmidt M, Kalle CV, et al. Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy. Methods Enzymol 2012; 507: 187-98.

Roselli EA, Mezzadra R, Frittoli MC, Maruggi G, Biral E, Mavilio F, et al. Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. EMBO Mol Med 2010; 2(8): 315-28.

Nienhuis AW, Persons DA. Development of gene therapy for thalassemia. Cold Spring Harb Perspect Med 2012. Dosegljivo na: http://perspectivesinmedicine.cshlp.org

Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 2010; 467: 318-22.

Gaudet D, Méthot J, Kastelein J. Gene therapy for lipoprotein lipase deficiency. Curr Opin Lipidol 2012; 23(4): 310-20.

Gruber K. Europe gives gene therapy the green light. Lancet 2012; 380(9855): e10.

Griesenbach U, Alton EW. Progress in gene and cell therapy for cystic fibrosis lung disease. Curr Pharm Des 2012; 18(5): 642-62.

Davies JC, Alton EW. Gene therapy for cystic fibrosis. Proc Am Thorac Soc 2010; 7(6): 408-14.

Knobe K, Berntorp E. New treatments in hemophilia: insights for the clinician. Ther Adv Hematol 2012; 3(3): 165-75.

Scott DW, Lozier JN. Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation. Br J Haematol 2012; 156(3): 295-302.

Nathwani AC, Tuddenham EGD, Rangarajan S, Rosales C, McIntosh J, Linch DC, et al. Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B. N Engl J Med 2011; 365: 2357-65.

Kočevar N, Čemažar M, Serša G. Gensko zdravljenje raka . Farm Vestn 2010; 61: 14-22.

Porter DL, Levine BL, Kalos M, Bagg A, June CH. Chimeric antigen receptor-modified T cells in chronic lymphoid leukemia. N Engl J Med 2011; 365(8): 725-33.

Robbins PF, Morgan RA, Feldman SA, Yang JC, Sherry RM, Dudley ME, et al. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol 2011; 29(7): 917-24.

ClinicalTrials.gov avg 2012. Dosegljivo na: clinicaltrials.gov

Published
2014-12-18
How to Cite
1.
Sedej K, Rožman P. New developments in gene therapy. TEST ZdravVestn [Internet]. 18Dec.2014 [cited 28Apr.2024];83(11). Available from: http://vestnik-dev.szd.si/index.php/ZdravVest/article/view/1060
Section
Review