A Review on Gene Therapy of Genetic Disorders
Abstract
Numerous intricate and untreatable ailments are brought on by genetic diseases. Each person's unique DNA sequence is encoded, and the mitochondrial, nuclear, and microbial metagenomes all contain a wealth of disease-causing genes. The use of next-generation DNA sequencing has brought these disorders' diagnoses together. Nonetheless, one of the key objectives is still to convert particular genetic diagnosis into tailored genetic treatments. Genetic therapies can be divided into three main categories thus far: replacing the damaged genetic compartments in large quantities with a new exogenous genome; adding extra genetic material to compensate for genetic errors without targeting it; and, most recently, using gene editing to directly correct the alterations that are causing the problems. The development of new generations of curative genetic medicines will be accelerated by the use of generalized techniques for diagnosis, treatment, and reagent administration into each genetic compartment.
Keywords:
Genetic disease, gene therapies, gene editing, genetic diagnostics, clinical genetics
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References
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2. Soler A, Morales C, Mademont-Soler I, Margarit E, Borrell A, Borobio V, Muñoz M, Sánchez A. Overview of chromosome abnormalities in first trimester miscarriages: a series of 1,011 consecutive chorionic villi sample karyotypes. Cytogenetic and genome research. 2017 Feb 1;152(2):81-9.
3. Baird PA, Anderson TW, Newcombe HB, Lowry R. Genetic disorders in children and young adults: a population study. American journal of human genetics. 1988 May;42(5):677.
4. Slack J, Evans KA. The increased risk of death from ischaemic heart disease in first degree relatives of 121 men and 96 women with ischaemic heart disease. Journal of medical genetics. 1966 Dec;3(4):239.
5. Ingram VM. A specific chemical difference between the globins of normal human and sickle-cell anaemia haemoglobin. Nature. 1956 Oct 13;178(4537):792-4.
6. Pauling L, Itano Ha, Et Al. Sickle Cell Anemia A Molecular Disease. Science. 1949 Nov 25;110(2865):543-8. Doi: 10.1126/Science.110.2865.543. Pmid: 15395398.
7. LEJEUNE J, GAUTIER M, Turpin RA. Étude des Chromosomes Somatiques de Neuf Enfants Mongoliens (Study of the somatic chromosomes of nine mongoloid children). Landmarks in Medical Genetics: Classic Papers with Commentaries. 2004 Mar 11(51):73.
8. Vennela B, Shirisha V, Rao CB, Gullapalli V, Batsala M. Current and Future Strategies for Therapy of Pancreatic Cancer. International Journal of Research in Pharmacy and Medicine. 2012;2(3):728-40.
9. Katakam P, Dey B, Assaleh FH, Hwisa NT, Adiki SK, Chandu BR, Mitra A. Top-down and bottom-up approaches in 3D printing technologies for drug delivery challenges. Critical Reviews™ in Therapeutic Drug Carrier Systems. 2015;32(1).
10. Jenkins MK, Chu HH, McLachlan JB, Moon JJ. On the composition of the preimmune repertoire of T cells specific for peptide–major histocompatibility complex ligands. Annual review of immunology. 2009 Apr 23;28:275-94.
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12. Vennela M, Srinivas B, Reddy VR, Balram N. Studies on correlation and path coefficient analysis in hybrid rice (Oryza sativa L.) for yield and quality traits. International Journal of Bio-resource and Stress Management. 2021;12(5):496-505.
13. Gowri SP, Lakshmi HV, Bhanu VN, Brahmaiah B, Nama S, Rao CB. Proniosome: a novel approach to vesicular drug delivery system. The Pharma Innovation. 2013 May 1;2(3, Part A):166.
14. Qi Q, Liu Y, Cheng Y, Glanville J, Zhang D, Lee JY, Olshen RA, Weyand CM, Boyd SD, Goronzy JJ. Diversity and clonal selection in the human T-cell repertoire. Proceedings of the National Academy of Sciences. 2014 Sep 9;111(36):13139-44.
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21. Friedmann T, Roblin R. Gene Therapy for Human Genetic Disease? Proposals for genetic manipulation in humans raise difficult scientific and ethical problems. Science.1972Mar3;175(4025):949-55. https://www.science.org/doi/abs/10.1126/science.175.4025.949
22. Merril CR, Geier MR, Petricciani JC. Bacterial virus gene expression in human cells. Nature. 1971 Oct 8;233(5319):398-400.
https://www.nature.com/articles/233398a0
23. Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, Lotze MT, Yang JC, Topalian SL, Merino MJ. Gene transfer into humans—immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. New England journal of medicine. 1990 Aug 30;323(9):570-8.
24. Cline MJ, Stang H, Mercola K, Morse L, Ruprecht R, Browne J, Salser W. Gene transfer in intactanimals.Nature. 1980 Apr 3;284(5755):422-5.
https://www.nature.com/articles/284422a0
25. Neelapu SS, Locke FL, Bartlett NL, Lekakis LJ, Miklos DB, Jacobson CA, Braunschweig I, Oluwole OO, Siddiqi T, Lin Y, Timmerman JM. Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma. New England Journal of Medicine. 2017 Dec 28;377(26):2531-44.
26. Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. New England Journal of Medicine. 2018 Feb 1;378(5):439-48.
Published
18/08/2024
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How to Cite
Adavi, S. “A Review on Gene Therapy of Genetic Disorders”. International Journal of Health Care and Biological Sciences, Vol. 5, no. 3, Aug. 2024, pp. 16-20, doi:10.46795/ijhcbs.v5i3.619.
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Review Articles
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