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Crispr/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases As Therapeutic Delivery; Current Applications and Challenges Publisher Pubmed



Saberi F1 ; Yousefinajafabadi Z3, 4 ; Shams F1, 5 ; Dehghan Z6 ; Ahmadi S1 ; Pilehchi T1 ; Noori E7 ; Esmaeelzadeh Z1 ; Bazgiri M8 ; Mohammadi R1 ; Khani F9 ; Sameni M1 ; Moradbeigi P10 ; Kardar GA3, 4 Show All Authors
Authors
  1. Saberi F1
  2. Yousefinajafabadi Z3, 4
  3. Shams F1, 5
  4. Dehghan Z6
  5. Ahmadi S1
  6. Pilehchi T1
  7. Noori E7
  8. Esmaeelzadeh Z1
  9. Bazgiri M8
  10. Mohammadi R1
  11. Khani F9
  12. Sameni M1
  13. Moradbeigi P10
  14. Kardar GA3, 4
  15. Salehi M1
  16. Teng Y2
  17. Jajarmi V1
Show Affiliations
Authors Affiliations
  1. 1. Department of Medical Biotechnology, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, Tehran, 1968917313, Iran
  2. 2. Department of Hematology and Medical Oncology, Winship Cancer Institute, Emory University, Atlanta, 30322, GA, United States
  3. 3. Department of Medical Biotechnology, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences, Tehran, 5469775141, Iran
  4. 4. Immunology, Asthma & Allergy Research Institute (IAARI), Tehran University of Medical Sciences, Tehran, Iran
  5. 5. Medical Nanotechnology and Tissue Engineering Research Center, Shahid Beheshti University of Medical Sciences, Tehran, 1968917313, Iran
  6. 6. Department of Comparative Biomedical Sciences, School of Advanced Medical Sciences and Technologies, Shiraz University of Medical Sciences, Shiraz, 7134874478, Iran
  7. 7. Department of Biotechnology, Faculty of Medicine, Shahed University, Tehran, 3319118651, Iran
  8. 8. Department of Animal Science, Agriculture and Natural Resources University of Khuzestan, Ahvaz, 6341773637, Iran
  9. 9. Department of Stem Cell and Regenerative Medicine, Institute of Medical Biotechnology, National Institute of Genetic Engineering & Biotechnology (NIGEB), Tehran, 14965161, Iran
  10. 10. Department of Clinical Science, School of Veterinary Medicine, Shiraz University, Shiraz, 7144169155, Iran

Source: Current Gene Therapy Published:2025


Abstract

The 5,000 to 8,000 monogenic diseases are inherited disorders leading to mutations in a single gene. These diseases usually appear in childhood and sometimes lead to morbidity or premature death. Although treatments for such diseases exist, gene therapy is considered an effective and targeted method and has been used in clinics for monogenic diseases since 1989. Monogenic diseases are good candidates for novel therapeutic technologies like gene editing approaches to repair gene mutations. Clustered regularly interspaced short palindromic repeats (CRISPR)-based systems, the pioneer and effective gene editing tool, are utilized for ex vivo and in vivo treatment of monogenic diseases. The current review provides an overview of recent therapeutic applications of CRISPR-based gene editing in monogenic diseases in in vivo and ex vivo models. Furthermore, this review consolidates strategies aimed at providing new treatment options with gene therapy, thereby serving as a valuable reference for advancing the treatment landscape for patients with monogenic disorders. © 2025 Bentham Science Publishers.
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