PMID- 31066249
OWN - NLM
STAT- MEDLINE
DCOM- 20190827
LR  - 20191210
IS  - 1735-5249 (Electronic)
IS  - 1735-1502 (Linking)
VI  - 18
IP  - 2
DP  - 2019 Apr 1
TI  - Recent Advances in Gene Therapy and Modeling of Chronic Granulomatous Disease.
PG  - 131-142
AB  - 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.
FAU - Jafarian, Arefeh
AU  - Jafarian A
AD  - Immunology, Asthma and Allergy Research Institute, Tehran University of Medical 
      Sciences, Tehran, Iran. a-jafarian@sina.tums.ac.ir.
FAU - Shokri, Gelareh
AU  - Shokri G
AD  - Stem Cell Technology Research Center, Tehran, Iran. gelareh.shokri@gmail.com.
FAU - Shokrollahi Barough, Mahdieh
AU  - Shokrollahi Barough M
AD  - Immunology, Asthma and Allergy Research Institute, Tehran University of Medical 
      Sciences, Tehran, Iran AND Department of Cancer Immunotherapy and Regenerative 
      Medicine, Breast Cancer Research Center, Motamed Cancer Institute, Academic 
      Center for Education, Culture and Research (ACECR), Tehran, Iran. 
      mahdie_shokrollahy@yahoo.com.
FAU - Moin, Mostafa
AU  - Moin M
AD  - Immunology, Asthma and Allergy Research Institute, Tehran University of Medical 
      Sciences, Tehran, Iran. mmoin@sina.tums.ac.ir.
FAU - Pourpak, Zahra
AU  - Pourpak Z
AD  - Immunology, Asthma and Allergy Research Institute, Tehran University of Medical 
      Sciences, Tehran, Iran. pourpakz@sina.tums.ac.ir.
FAU - Soleimani, Masoud
AU  - Soleimani M
AD  - Department of Hematology, Faculty of Medical Sciences, Tarbiat Modares 
      University, Tehran, Iran. soleim_m@modares.ac.ir.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20190401
PL  - Iran
TA  - Iran J Allergy Asthma Immunol
JT  - Iranian journal of allergy, asthma, and immunology
JID - 101146178
RN  - EC 1.6.3.- (NADPH Oxidases)
SB  - IM
MH  - Animals
MH  - CRISPR-Cas Systems
MH  - Clinical Trials as Topic
MH  - *Genetic Therapy
MH  - Genetic Vectors
MH  - Granulomatous Disease, Chronic/genetics/*therapy
MH  - Hematopoietic Stem Cell Transplantation
MH  - Humans
MH  - Infections/genetics/*therapy
MH  - Mutation/*genetics
MH  - NADPH Oxidases/*genetics
MH  - Phagocytosis/genetics
OTO - NOTNLM
OT  - Chronic granulomatous disease
OT  - Endonucleases
OT  - Gene editing
OT  - NADPH oxidases
EDAT- 2019/05/09 06:00
MHDA- 2019/08/28 06:00
CRDT- 2019/05/09 06:00
PHST- 2018/02/12 00:00 [received]
PHST- 2018/07/15 00:00 [accepted]
PHST- 2019/05/09 06:00 [entrez]
PHST- 2019/05/09 06:00 [pubmed]
PHST- 2019/08/28 06:00 [medline]
PST - epublish
SO  - Iran J Allergy Asthma Immunol. 2019 Apr 1;18(2):131-142.