References
1. National Institutes of Health. Genetics Home Reference. Help me understand genetics. Accessed May 3, 2021. https://medlineplus.gov/download/genetics/understanding/primer.pdf2. FDA Commissioner. What is gene therapy? How does it work? US Food and Drug Administration. Accessed July 1, 2021. https://www.fda.gov/consumers/consumer-updates/what-gene-therapy-how-does-it-work3. Biology Online Dictionary. Monogenic disease. Accessed April 19, 2021. https://www.biologyonline.com/dictionary/monogenic-disease4. Griffiths AJF, Miller JH, Suzuki DT, et al. An Introduction to Genetic Analysis, 7th edition. W.H. Freeman; 2000.5. STAT Reports. The STAT guide to viral vectors, the linchpin of gene therapy. STAT News; 2019.6. Collins M, Thrasher A. Gene therapy: progress and predictions. Proc Biol Sci. 2015;282(1821):770-788.7. Guha TK, Wai A, Hausner G. Programmable genome editing tools and their regulation for efficient genome engineering. Comput Struct Biotechnol J. 2017;15:146-160.38. Koshravi MA, Abbasalipour M, Concordet J-P, et al. Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: a promising approach for gene therapy of beta thalassemia disease. Eur J Pharmacol. 2019;854:398-405.9. Ceasar SA, Rajan V, Prykhozhij SV, Berman J, Ignacimuthu S. Insert, remove or replace: A highly advanced genome editing system using CRISPR/Cas9. Biochimica et Biophysica Acta. 201610. Choi J. Huntingtons Outreach Project for Education, at Stanford: Gene Silencing. Accessed July 1, 2021. https://hopes.stanford.edu/gene-silencing/11. Balasubramanian S, Habegger L, Frankish A, MacArthur DG, et al. Gene inactivation and its implications for annotation in the era of personal genomics. Genes Dev. 2011;25(1):1-10.12. Grant SG, Campbell CE, Duff C, et al. Gene inactivation as a mechanism for the expression of recessive phenotypes. Am J Hum Genet. 1989;45:619-634.13. Ellebrecht CT, Bhoj VG, Nace A, et al. Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease. Science. 2016;353(6295):179-184.14. Chandler RJ, Venditti CP. Gene therapy for metabolic diseases. Transl Sci Rare Dis. 016;1(1):73-89.15. Keeler AM, ElMallah MK, Flotte TR. Gene therapy 2017: progress and future directions. Clin Transl Sci. 2017:10(4):242-248.16. Rees H, Liu D. Base editing: prescision chemistry on the genome and transcriptome of living cells. Nat Rev Genet. 2018;19(12):770-788.17. American Society of Gene & Cell therapy (ASGCT). Gene and cell therapy FAQs. Accessed July 1, 2021. https://www.asgct.org/education/more-resources/gene-and-cell-therapy-faqs18. NIH National Cancer Institute. NCI dictionary of cancer terms. CAR T-cell therapy. Accessed July 1, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/car-t-cell-therapy19. Almasbak H, Aarvak T, Vemuri MC. CAR T cell therapy: a game changer in cancer treatment. J Immunol Res. 2016;2016:5474602.20. National Cancer Institute. NCI dictionary of cancer terms: CRISPR-Cas9. Accessed July 1, 2021. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/crispr-cas921. Encyclopedia Britannica. Gene editing. Accessed July 1, 2021. https://www.britanica.com/science/gene-editing
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How does Gene Therapy Work | Types of Gene Therapy
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