PMID- 35741383
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220716
IS  - 2079-7737 (Print)
IS  - 2079-7737 (Electronic)
IS  - 2079-7737 (Linking)
VI  - 11
IP  - 6
DP  - 2022 Jun 4
TI  - Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment.
LID - 10.3390/biology11060862 [doi]
LID - 862
AB  - Beta (beta)-thalassemia is a group of human inherited abnormalities caused by 
      various molecular defects, which involves a decrease or cessation in the balanced 
      synthesis of the beta-globin chains in hemoglobin structure. Traditional treatment 
      for beta-thalassemia major is allogeneic bone marrow transplantation (BMT) from a 
      completely matched donor. The limited number of human leukocyte antigen 
      (HLA)-matched donors, long-term use of immunosuppressive regimen and higher risk 
      of immunological complications have limited the application of this therapeutic 
      approach. Furthermore, despite improvements in transfusion practices and 
      chelation treatment, many lingering challenges have encouraged researchers to 
      develop newer therapeutic strategies such as nanomedicine and gene editing. One 
      of the most powerful arms of genetic manipulation is gene editing tools, 
      including transcription activator-like effector nucleases, zinc-finger nucleases, 
      and clustered regularly interspaced short palindromic repeat-Cas-associated 
      nucleases. These tools have concentrated on gamma- or beta-globin addition, regulating 
      the transcription factors involved in expression of endogenous gamma-globin such as 
      KLF1, silencing of gamma-globin inhibitors including BCL11A, SOX6, and LRF/ZBTB7A, 
      and gene repair strategies. In this review article, we present a systematic 
      overview of the appliances of gene editing tools for beta-thalassemia treatment and 
      paving the way for patients' therapy.
FAU - Rahimmanesh, Ilnaz
AU  - Rahimmanesh I
AD  - Applied Physiology Research Center, Cardiovascular Research Institute, Isfahan 
      University of Medical Sciences, Isfahan 73461-81746, Iran.
FAU - Boshtam, Maryam
AU  - Boshtam M
AD  - Isfahan Cardiovascular Research Center, Cardiovascular Research Institute, 
      Isfahan University of Medical Sciences, Isfahan 81583-88994, Iran.
FAU - Kouhpayeh, Shirin
AU  - Kouhpayeh S
AD  - Erythron Genetics and Pathobiology Laboratory, Department of Immunology, Isfahan 
      76351-81647, Iran.
FAU - Khanahmad, Hossein
AU  - Khanahmad H
AD  - Department of Genetics and Molecular Biology, School of Medicine, Isfahan 
      University of Medical Sciences, Isfahan 73461-81746, Iran.
FAU - Dabiri, Arezou
AU  - Dabiri A
AUID- ORCID: 0000-0002-9696-5135
AD  - Applied Physiology Research Center, Cardiovascular Research Institute, Isfahan 
      University of Medical Sciences, Isfahan 73461-81746, Iran.
FAU - Ahangarzadeh, Shahrzad
AU  - Ahangarzadeh S
AUID- ORCID: 0000-0002-2377-3319
AD  - Infectious Diseases and Tropical Medicine Research Center, Isfahan University of 
      Medical Sciences, Isfahan 73461-81746, Iran.
FAU - Esmaeili, Yasaman
AU  - Esmaeili Y
AD  - Biosensor Research Center, School of Advanced Technologies in Medicine, Isfahan 
      University of Medical Sciences, Isfahan 73461-81746, Iran.
FAU - Bidram, Elham
AU  - Bidram E
AUID- ORCID: 0000-0002-6319-3204
AD  - Biosensor Research Center, School of Advanced Technologies in Medicine, Isfahan 
      University of Medical Sciences, Isfahan 73461-81746, Iran.
AD  - Department of Biomaterials, Nanotechnology and Tissue Engineering, School of 
      Advanced Technologies in Medicine, Isfahan University of Medical Sciences, 
      Isfahan 73461-81746, Iran.
FAU - Vaseghi, Golnaz
AU  - Vaseghi G
AD  - Isfahan Cardiovascular Research Center, Cardiovascular Research Institute, 
      Isfahan University of Medical Sciences, Isfahan 81583-88994, Iran.
FAU - Haghjooy Javanmard, Shaghayegh
AU  - Haghjooy Javanmard S
AD  - Applied Physiology Research Center, Cardiovascular Research Institute, Isfahan 
      University of Medical Sciences, Isfahan 73461-81746, Iran.
FAU - Shariati, Laleh
AU  - Shariati L
AD  - Department of Biomaterials, Nanotechnology and Tissue Engineering, School of 
      Advanced Technologies in Medicine, Isfahan University of Medical Sciences, 
      Isfahan 73461-81746, Iran.
AD  - Cancer Prevention Research, Isfahan University of Medical Sciences, Isfahan 
      73461-81746, Iran.
FAU - Zarrabi, Ali
AU  - Zarrabi A
AUID- ORCID: 0000-0003-0391-1769
AD  - Department of Biomedical Engineering, Faculty of Engineering and Natural 
      Sciences, Istinye University, Sariyer, Istanbul 34396, Turkey.
FAU - Varma, Rajender S
AU  - Varma RS
AUID- ORCID: 0000-0001-9731-6228
AD  - Regional Centre of Advanced Technologies and Materials, Department of Physical 
      Chemistry, Faculty of Science, Palacky University, Slechtitelu 27, 783 71 
      Olomouc, Czech Republic.
LA  - eng
GR  - 199306/Isfahan University of Medical Sciences/
PT  - Journal Article
PT  - Review
DEP - 20220604
PL  - Switzerland
TA  - Biology (Basel)
JT  - Biology
JID - 101587988
PMC - PMC9219845
OTO - NOTNLM
OT  - CRISPR
OT  - TALEN
OT  - ZFN
OT  - beta-thalassemia
OT  - gene therapy
COIS- The authors declare no conflict of interest.
EDAT- 2022/06/25 06:00
MHDA- 2022/06/25 06:01
PMCR- 2022/06/04
CRDT- 2022/06/24 01:11
PHST- 2022/04/10 00:00 [received]
PHST- 2022/05/19 00:00 [revised]
PHST- 2022/05/31 00:00 [accepted]
PHST- 2022/06/24 01:11 [entrez]
PHST- 2022/06/25 06:00 [pubmed]
PHST- 2022/06/25 06:01 [medline]
PHST- 2022/06/04 00:00 [pmc-release]
AID - biology11060862 [pii]
AID - biology-11-00862 [pii]
AID - 10.3390/biology11060862 [doi]
PST - epublish
SO  - Biology (Basel). 2022 Jun 4;11(6):862. doi: 10.3390/biology11060862.