Journal of Human Genetics and Genomics

Published by: Kowsar

Hemophilia Gene Therapy; Clinical and Molecular Aspects

Mohammad Zaree 1 , Masomeh Sadat Sabzevari 2 , Ayub Ahmadi 3 , Mahboubeh Ramezanzadeh 4 , * and Ali Hosseini Bereshneh 5 , **
Authors Information
1 Department of Internal Medicine, Persian Gulf Hospital, School of Medicine, Bushehr University of Medical Sciences, Bushehr, IR Iran
2 Department of Perinatology, Imam Khomeini Hospital, Tehran University of Medical Sciences, Tehran, IR Iran
3 Persian Gulf Hospital, School of Medicine, Bushehr University of Medical Sciences, Bushehr, IR Iran
4 Department of Genetics and Molecular Medicine, School of Medicine, Bushehr University of Medical Sciences, Busherhr, IR Iran
5 Department of Medical Genetics, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, IR Iran
Corresponding Authors:
Article information
  • Journal of Human Genetics and Genomics: July 2018, 2 (1); e80764
  • Published Online: July 29, 2017
  • Article Type: Review Article
  • Received: May 10, 2017
  • Accepted: July 1, 2017
  • DOI: 10.5812/jhgg.80764

To Cite: Zaree M, Sabzevari M S, Ahmadi A, Ramezanzadeh M, Hosseini Bereshneh A. Hemophilia Gene Therapy; Clinical and Molecular Aspects, J Human Gen Genom. 2018 ; 2(1):e80764. doi: 10.5812/jhgg.80764.

Abstract
Copyright © 2017, Journal of Human Genetics and Genomics. This is an open-access article distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/) which permits copy and redistribute the material just in noncommercial usages, provided the original work is properly cited
1. Background
2. Structure and Function of Hemophilia Associated Genes and Proteins
3. Hemophilia Clinical Signs
4. Diagnosis
5. Treatment
6. Potential of Gene Therapy for Hemophilia
7. Tissue Targets for Hemophilia Gene Therapy
8. Viral Vectors
9. Stem Cell Based Gene Transfer
10. Hepatocyte Based Gen Transfer
11. Myocyte Based Gene Transfer
12. Conclusions
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