Different Gene Therapies Fortreatment of Hemophilia A

Abstract

According to US Center for Disease Control and Prevention, there are 12 per 100000 males born with hemophilia A in US, and there are 400 male babies born with hemophilia A per year. Hemophilia A is type of disease that is inherited with X-linked recessive that have mutation in Factor VII gene. Recently, there are types of gene therapies success in hepatic vivo gene transferring. In this review, this paper will discuss three types of gene therapies for hemophilia A which are Zinc Finger Nucleases (ZFNs) and Transcription Activator Factors (TALENs) and CRISPER Cas9. ZFNs using the zinc finger DNA binding array in conjunction with FoKI cleavage nucleases domain to target and cleave to trigger the DNA repairing. In TALENs researchers induced pluripotent stem cells (iPSCs) derived by TALENickases to target the FVIII site. The latest research is using CRISPR Cas9 as a gene editing method to treat hemophilia A. The researchers used this method to repair the mutated gene so that the FVIII factor could be expressed normally. The paper also explains the current methods and process of delivery in gene therapy and shows the pros and cons of each. In conclusion, this paper express our prospects for future research about gene therapies.