Gene therapy and gene editing in human disease: CRISPR and beyond
DOI:
https://doi.org/10.58445/rars.484Keywords:
Gene Therapy, Gene Editing, CRISPRAbstract
Exploring genetic diseases and disorders and new technologies in gene therapy and gene editing treating these disorders.
References
Giorgio Ottaviano et al. Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia.Sci. Transl. Med.14,eabq3010(2022).DOI:10.1126/scitranslmed.abq3010
Health Direct staff. (2021, September). Williams Syndrome. Health Direct. Retrieved September 18, 2023, from https://www.healthdirect.gov.au/williams-syndrome
Montaño, A., Forero-Castro, M., Hernández-Rivas, J. M., García-Tuñón, I., & Benito, R. (2018). Targeted genome editing in acute lymphoblastic leukemia: a review. BMC biotechnology, 18(1), 45. https://doi.org/10.1186/s12896-018-0455-9
Morris, C. (2023, April 13). Williams Syndrome. National Library of Medicine. Retrieved September 18, 2023, from https://www.ncbi.nlm.nih.gov/books/NBK1249/
Vajen, B., Ronez, J., Rathje, W., Heinisch, L., Ebeling, S., Gebhard, U., Hößle, C., & Schlegelberger, B. (2021). Students' attitudes towards somatic genome editing versus genome editing of the germline using an example of familial leukemia. Journal of community genetics, 12(3), 397–406. https://doi.org/10.1007/s12687-021-00528-1
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