Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions

Gene therapy (GT) has been reported to improve bone marrow function in individuals with Fanconi anemia (FA); however, its clinical application is still in the initial stages. We conducted this systematic review, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA...

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Autores principales:	Shameel Shafqat, Eleze Tariq, Aric D. Parnes, Majed J. Dasouki, Syed O. Ahmed, Shahrukh K. Hashmi
Formato:	article
Lenguaje:	EN
Publicado:	Elsevier 2021
Materias:	Bone marrow transplant Fanconi anemia Gene therapy Diseases of the blood and blood-forming organs RC633-647.5 Neoplasms. Tumors. Oncology. Including cancer and carcinogens RC254-282
Acceso en línea:	https://doaj.org/article/2da66f4428d8477185ab90a33c9ffe48
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spelling	oai:doaj.org-article:2da66f4428d8477185ab90a33c9ffe482021-12-02T05:00:02ZRole of gene therapy in Fanconi anemia: A systematic and literature review with future directions1658-387610.1016/j.hemonc.2021.02.001https://doaj.org/article/2da66f4428d8477185ab90a33c9ffe482021-12-01T00:00:00Zhttp://www.sciencedirect.com/science/article/pii/S1658387621000261https://doaj.org/toc/1658-3876Gene therapy (GT) has been reported to improve bone marrow function in individuals with Fanconi anemia (FA); however, its clinical application is still in the initial stages. We conducted this systematic review, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, to assess the long-term safety and clinical outcomes of GT in FA patients. Electronic searches from PubMed, Web of Science, Cochrane Library, and Google Scholar were conducted and full texts of articles meeting our inclusion criteria were reviewed. Three clinical trials were included, with a total of nine patients and mean age of 10.7 ± 5.7 years. All patients had lentiviral-mediated GT. A 1-year follow-up showed stabilization in blood lineages, without any serious adverse effects from GT. A metaregression analysis could not be conducted, as very little long-term follow-up data of patients was observed, and the median survival rate could not be calculated. Thus, we can conclude that GT seems to be a safe procedure in FA; however, further research needs to be conducted on the longitudinal clinical effects of GT in FA, for a better insight into its potential to become a standard form of treatment.Shameel ShafqatEleze TariqAric D. ParnesMajed J. DasoukiSyed O. AhmedShahrukh K. HashmiElsevierarticleBone marrow transplantFanconi anemiaGene therapyDiseases of the blood and blood-forming organsRC633-647.5Neoplasms. Tumors. Oncology. Including cancer and carcinogensRC254-282ENHematology/Oncology and Stem Cell Therapy, Vol 14, Iss 4, Pp 290-301 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Bone marrow transplant Fanconi anemia Gene therapy Diseases of the blood and blood-forming organs RC633-647.5 Neoplasms. Tumors. Oncology. Including cancer and carcinogens RC254-282
spellingShingle	Bone marrow transplant Fanconi anemia Gene therapy Diseases of the blood and blood-forming organs RC633-647.5 Neoplasms. Tumors. Oncology. Including cancer and carcinogens RC254-282 Shameel Shafqat Eleze Tariq Aric D. Parnes Majed J. Dasouki Syed O. Ahmed Shahrukh K. Hashmi Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
description	Gene therapy (GT) has been reported to improve bone marrow function in individuals with Fanconi anemia (FA); however, its clinical application is still in the initial stages. We conducted this systematic review, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, to assess the long-term safety and clinical outcomes of GT in FA patients. Electronic searches from PubMed, Web of Science, Cochrane Library, and Google Scholar were conducted and full texts of articles meeting our inclusion criteria were reviewed. Three clinical trials were included, with a total of nine patients and mean age of 10.7 ± 5.7 years. All patients had lentiviral-mediated GT. A 1-year follow-up showed stabilization in blood lineages, without any serious adverse effects from GT. A metaregression analysis could not be conducted, as very little long-term follow-up data of patients was observed, and the median survival rate could not be calculated. Thus, we can conclude that GT seems to be a safe procedure in FA; however, further research needs to be conducted on the longitudinal clinical effects of GT in FA, for a better insight into its potential to become a standard form of treatment.
format	article
author	Shameel Shafqat Eleze Tariq Aric D. Parnes Majed J. Dasouki Syed O. Ahmed Shahrukh K. Hashmi
author_facet	Shameel Shafqat Eleze Tariq Aric D. Parnes Majed J. Dasouki Syed O. Ahmed Shahrukh K. Hashmi
author_sort	Shameel Shafqat
title	Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
title_short	Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
title_full	Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
title_fullStr	Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
title_full_unstemmed	Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions
title_sort	role of gene therapy in fanconi anemia: a systematic and literature review with future directions
publisher	Elsevier
publishDate	2021
url	https://doaj.org/article/2da66f4428d8477185ab90a33c9ffe48
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Role of gene therapy in Fanconi anemia: A systematic and literature review with future directions

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