Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review
Apert syndrome is a genetic disorder characterised by craniosynostosis and structural discrepancy of the craniofacial region as well as the hands and feet. This condition is closely linked with fibroblast growth factor receptor-2 (FGFR2) gene mutations. Gene therapies are progressively being tested...
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my.um.eprints.279962022-07-06T01:51:33Z http://eprints.um.edu.my/27996/ Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review Al-Namnam, Nisreen Mohammed Jayash, Soher Nagi Hariri, Firdaus Rahman, Zainal Ariff Abdul Alshawsh, Mohammed Abdullah QD Chemistry QH301 Biology QR Microbiology R Medicine (General) Apert syndrome is a genetic disorder characterised by craniosynostosis and structural discrepancy of the craniofacial region as well as the hands and feet. This condition is closely linked with fibroblast growth factor receptor-2 (FGFR2) gene mutations. Gene therapies are progressively being tested in advanced clinical trials, leading to a rise of its potential clinical indications. In recent years, research has made great progress in the gene therapy of craniosynostosis syndromes and several studies have investigated its influences in preventing/diminishing the complications of Apert syndrome. This article reviewed and exhibited different techniques of gene therapy and their influences in Apert syndrome progression. A systematic search was executed using electronic bibliographic databases including PubMed, EMBASE, ScienceDirect, SciFinder and Web of Science for all studies of gene therapy for Apert syndrome. The primary outcomes measurements vary from protein to gene expressions. According to the findings of included studies, we conclude that the gene therapy using FGF in Apert syndrome was critical in the regulation of suture fusion and patency, occurred via alterations in cellular proliferation. The superior outcome could be brought by biological therapies targeting the FGF/FGFR signalling. More studies in molecular genetics in Apert syndrome are recommended. This study reviews the current literature and provides insights to future possibilities of genetic therapy as intervention in Apert syndrome. Springernature 2021-11 Article PeerReviewed Al-Namnam, Nisreen Mohammed and Jayash, Soher Nagi and Hariri, Firdaus and Rahman, Zainal Ariff Abdul and Alshawsh, Mohammed Abdullah (2021) Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review. Gene Therapy, 28 (10-11). pp. 620-633. ISSN 0969-7128, DOI https://doi.org/10.1038/s41434-021-00238-w <https://doi.org/10.1038/s41434-021-00238-w>. 10.1038/s41434-021-00238-w |
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QD Chemistry QH301 Biology QR Microbiology R Medicine (General) Al-Namnam, Nisreen Mohammed Jayash, Soher Nagi Hariri, Firdaus Rahman, Zainal Ariff Abdul Alshawsh, Mohammed Abdullah Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review |
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Apert syndrome is a genetic disorder characterised by craniosynostosis and structural discrepancy of the craniofacial region as well as the hands and feet. This condition is closely linked with fibroblast growth factor receptor-2 (FGFR2) gene mutations. Gene therapies are progressively being tested in advanced clinical trials, leading to a rise of its potential clinical indications. In recent years, research has made great progress in the gene therapy of craniosynostosis syndromes and several studies have investigated its influences in preventing/diminishing the complications of Apert syndrome. This article reviewed and exhibited different techniques of gene therapy and their influences in Apert syndrome progression. A systematic search was executed using electronic bibliographic databases including PubMed, EMBASE, ScienceDirect, SciFinder and Web of Science for all studies of gene therapy for Apert syndrome. The primary outcomes measurements vary from protein to gene expressions. According to the findings of included studies, we conclude that the gene therapy using FGF in Apert syndrome was critical in the regulation of suture fusion and patency, occurred via alterations in cellular proliferation. The superior outcome could be brought by biological therapies targeting the FGF/FGFR signalling. More studies in molecular genetics in Apert syndrome are recommended. This study reviews the current literature and provides insights to future possibilities of genetic therapy as intervention in Apert syndrome. |
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Article |
author |
Al-Namnam, Nisreen Mohammed Jayash, Soher Nagi Hariri, Firdaus Rahman, Zainal Ariff Abdul Alshawsh, Mohammed Abdullah |
author_facet |
Al-Namnam, Nisreen Mohammed Jayash, Soher Nagi Hariri, Firdaus Rahman, Zainal Ariff Abdul Alshawsh, Mohammed Abdullah |
author_sort |
Al-Namnam, Nisreen Mohammed |
title |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review |
title_short |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review |
title_full |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review |
title_fullStr |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review |
title_full_unstemmed |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review |
title_sort |
insights and future directions of potential genetic therapy for apert syndrome: a systematic review |
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Springernature |
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2021 |
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http://eprints.um.edu.my/27996/ |
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