What is it about?
Hemophilia A is a hemorrhage disorder and is a genetic disorder. The disorder affects 1 in 4,000 to 10,000 males. Permanent changes in the F8 gene result in hemophilia A. These changes result in an abnormal version of a coagulation factor VIII. Currently, to treat hemophilia A, recombinant factor VIII can be used. However, the charge is very high. It causes heavy burdens on the patients, their relations, and the health care system. Therefore, advanced therapy such as iPSCs and CRISPR/Cas9, has attracted the interest of investigators to research for treating this disorder. Research outcomes in animal models have shown significant improvements.
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Why is it important?
In this article, the author draws the advances in the study of hemophilia A focusing on the genetic aspects. It comprises the F8 gene, mutations in the gene, gene editing techniques, and the iPSCs technology. It draws gene correction using iPSCs in combination with gene editing (CRISPR/Cas9). It is a future treatment for the hemophilia A disorder.
Perspectives
The iPSCs technique in combination with CRISPR/Cas9 system can edit erroneous segments in the F8 gene to the normal segments. In addition, these techniques can cause normal segments of the F8 gene to erroneous segments and then make it back to the WT situation. It shows that hemophilia A came from mutations or DNA changes in the F8 gene in the past. To overcome the disadvantages in iPSCs technologies for the cure of hemophilia A, TALENs and CRISPR/Cas9 systems can be used.
Martin Nelwan
Nelwan Institution for Human Resource Development
Read the Original
This page is a summary of: Hemophilia A and Induced Pluripotent Stem Cells, Journal of Advances in Biology & Biotechnology, January 2017, Sciencedomain International,
DOI: 10.9734/jabb/2017/35111.
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