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Raji, Sevil, Soltaninejad, Hossein, Hosseinkhani, Saman, Hamidieh, Amir Ali. (1405). CRISPR/Cas9-Mediated Targeting of the Integrin Beta-2 Gene as a Gene-Editing Strategy for Leukocyte Adhesion Deficiency Type I. سامانه مدیریت نشریات علمی, (), -. doi: 10.30476/ijms.2026.109631.4499
Sevil Raji; Hossein Soltaninejad; Saman Hosseinkhani; Amir Ali Hamidieh. "CRISPR/Cas9-Mediated Targeting of the Integrin Beta-2 Gene as a Gene-Editing Strategy for Leukocyte Adhesion Deficiency Type I". سامانه مدیریت نشریات علمی, , , 1405, -. doi: 10.30476/ijms.2026.109631.4499
Raji, Sevil, Soltaninejad, Hossein, Hosseinkhani, Saman, Hamidieh, Amir Ali. (1405). 'CRISPR/Cas9-Mediated Targeting of the Integrin Beta-2 Gene as a Gene-Editing Strategy for Leukocyte Adhesion Deficiency Type I', سامانه مدیریت نشریات علمی, (), pp. -. doi: 10.30476/ijms.2026.109631.4499
Raji, Sevil, Soltaninejad, Hossein, Hosseinkhani, Saman, Hamidieh, Amir Ali. CRISPR/Cas9-Mediated Targeting of the Integrin Beta-2 Gene as a Gene-Editing Strategy for Leukocyte Adhesion Deficiency Type I. سامانه مدیریت نشریات علمی, 1405; (): -. doi: 10.30476/ijms.2026.109631.4499

CRISPR/Cas9-Mediated Targeting of the Integrin Beta-2 Gene as a Gene-Editing Strategy for Leukocyte Adhesion Deficiency Type I

Iranian Journal of Medical Sciences
مقالات آماده انتشار، اصلاح شده برای چاپ، انتشار آنلاین از تاریخ 07 مهر 1405    اصل مقاله (704.71 K)
نوع مقاله: Original Article(s)
شناسه دیجیتال (DOI): 10.30476/ijms.2026.109631.4499
نویسندگان
Sevil Raji1؛ Hossein Soltaninejad* 1؛ Saman Hosseinkhani2؛ Amir Ali Hamidieh3
1Department of Stem Cells Technology and Tissue Regeneration, Faculty of Interdisciplinary Science and Technologies, Tarbiat Modares University, Tehran, Iran
2Department of Biochemistry, Faculty of Biological Sciences, Tarbiat Modares University, Tehran, Iran
3Pediatric Cell and Gene Therapy Research Center, Gene, Cell, and Tissue Research Institute, Tehran University of Medical Sciences, Tehran, Iran
چکیده
Background: Leukocyte adhesion deficiency (LAD) is a genetic immunodeficiency that severely impairs the leukocyte adhesion cascade and migration to the sites of infection and inflammation.  As a result, the main consequence is recurrent and severe infections with bacteria and fungi, which can lead to death. The most common variant, LAD-I, is due to mutations in the integrin beta 2 (ITGB2) gene responsible for the β2 integrin (CD18) subunit. Corrective strategies based on gene therapy and genome editing are the most promising methods for treating genetic defects.
Methods: In the present experimental in vitro study conducted at Tarbiat Modares University, Tehran, Iran, in 2024, we designed a single-guide RNA (sgRNA) for the CRISPR/Cas9 system to target exon 6 of ITGB2 and performed validation experiments. We also investigated peptide nanocarriers as a potential strategy for delivering the CRISPR components. The PX458 plasmid encoding Cas9 and the designed sgRNA was delivered into HEK293T cells, followed by genome-editing analysis.
Results: Genome-editing analysis confirmed a measurable level of indel formation at the target locus. Furthermore, the results of MPG nanopeptide production and purification demonstrate the potential of peptide nanocarriers for genome-editing systems.
Conclusion: Overall, this work establishes a foundation for subsequent knock-in and gene-correction studies in clinically relevant immune cell models, paving the way for potential LAD-I gene therapy applications.

تازه های تحقیق

Sevil Raji (Google Scholar)
Hossein Soltaninejad (Google Scholar) 

کلیدواژه‌ها
Leukocyte adhesion deficiency Type I؛ ITGB2 protein؛ CRISPR-Cas systems؛ Gene editing؛ Cell-penetrating peptide
مراجع
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