Tehran University of Medical Sciences

Science Communicator Platform

Stay connected! Follow us on X network (Twitter):
Share this content! On (X network) By
Recent Advances in Gene Therapy and Modeling of Chronic Granulomatous Disease Publisher Pubmed



Jafarian A4 ; Shokri G1 ; Barough MS2, 4 ; Moin M4 ; Pourpak Z4 ; Soleimani M3
Authors
Show Affiliations
Authors Affiliations
  1. 1. Stem Cell Technology Research Center, Tehran, Iran
  2. 2. Department of Cancer Immunotherapy and Regenerative Medicine, Breast Cancer Research Center, Motamed Cancer Institute, Academic Center for Education, Culture and Research (ACECR), Tehran, Iran
  3. 3. Department of Hematology, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, 14115-111, Iran
  4. 4. Immunology, Asthma and Allergy Research Institute, Tehran University of Medical Sciences, Tehran, 14194, Iran

Source: Iranian Journal of Allergy# Asthma and Immunology Published:2019


Abstract

The Chronic granulomatous disease (CGD) is a primary immunodeficiency that characterized by mutations in phagocyte nicotinamide adenine dinucleotide phosphate (NADPH) oxidase, resulting in deficient antimicrobial activity of phagocytic cells and recurrent childhood infections. Hematopoietic stem cell transplantation (HSCT) is a curative option for patients with human leukocyte antigen (HLA) matched donor, when conventional cares and therapies fail. However, in many cases when the patients have not an HLA-matched donor, they need to a method to recapitulate the function of the affected gene within the patient’s own cells. Gene therapy is a promising approach for CGD. While, the success of retroviral or lentiviral vectors in gene therapy for CGD has been hampered by random integration and insertional activation of proto-oncogenes. These serious adverse events led to improvement and generations of viral vectors with increased safety characteristics. Gene therapy continues to progress and the advent of new technologies, such as engineered endonucleases that have shown a great promise for the treatment of genetic disease. This review focuses on the application of gene therapy for the CGD, the limitations encountered in current clinical trials, advantages and disadvantages of endonucleases in gene correction and modeling with CRISPR/Cas9 approach. © Copyright© April 2019, Iran J Allergy Asthma Immunol. All rights reserved.
Related Docs
View other Related Docs
1. Characterization of 4 New Mutations in the Cybb Gene in 10 Iranian Families With X-Linked Chronic Granulomatous Disease, Journal of Pediatric Hematology/Oncology (2018)
2. Technology Developments in Biological Tools for Targeted Genome Surgery, Biotechnology Letters (2015)
3. Genetic and Molecular Findings of 38 Iranian Patients With Chronic Granulomatous Disease Caused by P47-Phox Defect, Scandinavian Journal of Immunology (2019)
4. Crispr/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases As Therapeutic Delivery; Current Applications and Challenges, Current Gene Therapy (2025)
5. Recent Advances in Gene Therapy: Genetic Bullets to the Root of the Problem, Clinical and Experimental Medicine (2023)
6. Three Novel Mutations in Cyba Among 22 Iranians With Chronic Granulomatous Disease, International Journal of Immunogenetics (2017)
7. Implications of Crispr-Cas9 Genome Editing Methods in Atherosclerotic Cardiovascular Diseases, Current Problems in Cardiology (2023)
8. Induced Pluripotent Stem Cell Meets Severe Combined Immunodeficiency, Cell Journal (2020)
Experts (# of related papers)
View all Related Experts
Zahra Pourpak (4)
Amir Ali Hamidieh (4)
Other Related Docs
9. Viral-Based Gene Therapy Clinical Trials for Immune Deficiencies and Blood Disorders From 2013 Until 2023 - an Overview, Regenerative Therapy (2025)
10. Bcgitis As the Primary Manifestation of Chronic Granulomatous Disease, IDCases (2021)
11. Paecilomyces Formosus Infection in an Adult Patient With Undiagnosed Chronic Granulomatous Disease, Journal of Clinical Immunology (2017)
12. Ocular Manifestations of Chronic Granulomatous Disease: First Report of Coats’ Disease and Literature Review, Journal of Clinical Immunology (2020)
13. The Crispr Growth Spurt: From Bench to Clinic on Versatile Small Rnas, Journal of Microbiology and Biotechnology (2017)
14. In Vitro Study of Hax1 Gene Therapy by Retro Viral Transduction As a Therapeutic Target in Severe Congenital Neutropenia, European Cytokine Network (2018)
15. Bi-Functionalized Aminoguanidine-Pegylated Periodic Mesoporous Organosilica Nanoparticles: A Promising Nanocarrier for Delivery of Cas9-Sgrna Ribonucleoproteine, Journal of Nanobiotechnology (2021)
16. Exploring Non-Viral Methods for the Delivery of Crispr-Cas Ribonucleoprotein to Hematopoietic Stem Cells, Stem Cell Research and Therapy (2024)
17. A Fludarabine and Melphalan Reduced-Intensity Conditioning Regimen for Hsct in Fifteen Chronic Granulomatous Disease Patients and a Literature Review, Annals of Hematology (2022)
18. Dyskeratosis Congenita Future: Hematopoietic Stem Cell Transplantation or Gene Therapy?, Gene Reports (2025)
19. A Novel Cybb Mutation in Chronic Granulomatous Disease in Iran, Iranian Journal of Allergy# Asthma and Immunology (2016)
20. Improving the Function of Neutrophils From Chronic Granulomatous Disease Patients Using Mesenchymal Stem Cells’ Exosomes, Human Immunology (2020)
21. Genetic Modification of Cystic Fibrosis With Δf508 Mutation of Cftr Gene Using the Crispr System in Peripheral Blood Mononuclear Cells, Iranian Journal of Basic Medical Sciences (2020)