Articles | Open Access |

ADVANCEMENTS IN GENE THERAPY: NOVEL AAV-DERIVED VECTORS AND SIN CHANNELS

Kehinde Adefope , Faculty of Biological Sciences, University of Nigeria, Nsukka, Nigeria

Abstract

Gene therapy holds promise for treating various genetic disorders, but its efficacy has been hindered by limitations in delivery mechanisms. Recent strides in adeno-associated virus (AAV)-derived vectors and self-inactivating (SIN) channels have revolutionized gene therapy by enhancing delivery efficiency and reducing immunogenicity. This paper explores the latest innovations in AAV-derived vectors and SIN channels, highlighting their potential to overcome barriers and elevate the efficacy of gene therapy. By unlocking the potential of these technologies, researchers aim to address critical challenges and pave the way for more effective treatments of genetic diseases.

Keywords

Gene therapy, genetic disorders, delivery mechanisms

References

Sheridan C. Gene therapy finds its niche. Nat Biotechnol 2011;29:121–8.

Bryant LM, Christopher DM, Giles AR, Hinderer C, Rodriguez JL, Smith JB, et al. Lessons learned from the clinical development and market authorization of glybera. Hum Gene Ther Clin Dev 2013;24:55–64.

Elsner M, Terbish T, Jörns A, Naujok O, Wedekind D, Hedrich H-J, et al. Reversal of diabetes through gene therapy of diabetic rats by hepatic insulin expression via lentiviral transduction. Mol Ther 2012;20:918–26.

Russell SJ, Peng K-W, Bell JC. Oncolytic virotherapy. Nat Biotechnol 2012;30:658–70.

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 tumorinfiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990;323:570–8.

Scholler J, Brady TL, Binder-Scholl G, Hwang WT, Plesa G, Hege KM, et al. Decade-long safety and function of retroviral-modified chimeric antigen receptor T cells. Sci Transl Med 2012;4:132–53.

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;365:725–33.

Vago L, Oliveira G, Bondanza A, Noviello M, Soldati C, Ghio D, et al. T-cell suicide gene therapy prompts thymic renewal in adults after hematopoietic stem cell transplantation. Blood 2012;120:1820–30.

Kershaw MH, Westwood JA, Darcy PK. Gene-engineered T cells for cancer therapy. Nat Rev Cancer 2013;13:525–41.

Grossman M, Raper SE, Kozarsky K, Stein EA, Engelhardt JF, Muller D, et al. Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia. Nat Genet 1994;6:335–41.

Roos A-K, Eriksson F, Timmons JA, Gerhardt J, Nyman U, Gudmundsdotter L, et al. Skin electroporation: effects on transgene expression, DNA persistence and local tissue environment. PLoS One 2009;4:e7226.

Kaufman HL, Kohlhapp FJ, Zloza A. Oncolytic viruses: a new class of immunotherapy drugs. Nat Rev Drug Discov 2015;14:642–62.

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ADVANCEMENTS IN GENE THERAPY: NOVEL AAV-DERIVED VECTORS AND SIN CHANNELS. (2024). International Journal of Modern Medicine, 3(02), 01-05. http://www.intjmm.com/index.php/ijmm/article/view/16