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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.
Afiliación
  • Al-Namnam NM; Department Oral Surgery, Faculty of Dentistry, University of Sana'a, Sana'a, Yemen. nis_moh2007@yahoo.com.
  • Jayash SN; School of Dentistry, University of Birmingham, 5 Mill Pool Way, Edgbaston, Birmingham, UK.
  • Hariri F; Department of Oral and Maxillofacial Clinical Sciences, Faculty of Dentistry, University of Malaya, 50603, Kuala Lumpur, Malaysia.
  • Rahman ZAA; Department of Oral and Maxillofacial Clinical Sciences, Faculty of Dentistry, University of Malaya, 50603, Kuala Lumpur, Malaysia.
  • Alshawsh MA; Department of Pharmacology, Faculty of Medicine, University of Malaya, 50603, Kuala Lumpur, Malaysia. alshaweshmam@um.edu.my.
Gene Ther ; 28(10-11): 620-633, 2021 11.
Article en En | MEDLINE | ID: mdl-33619359
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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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Acrocefalosindactilia Tipo de estudio: Systematic_reviews Límite: Humans Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Yemen Pais de publicación: Reino Unido
Texto completo
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XML
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Acrocefalosindactilia Tipo de estudio: Systematic_reviews Límite: Humans Idioma: En Revista: Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Yemen Pais de publicación: Reino Unido