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间充质干细胞(MSCs)的基因改造:扩大其在危重症中原始应用的新策略。

Genetic modification of mesenchymal stem cells (MSCs): novel strategy to expand their naïve applications in critical illness.

作者信息

Nasiri Hajar, Manoochehrabadi Tahereh, Eskandari Fatemeh, Majidi Jila, Gholipourmalekabadi Mazaher

机构信息

Department of Tissue Engineering and Applied Cell Sciences, School of Paramedicine, Guilan University of Medical Sciences, Rasht, Iran.

Department of Tissue Engineering & Regenerative Medicine, Faculty of Advanced Technologies in Medicine, Iran University of Medical Sciences, Tehran, Iran.

出版信息

Mol Biol Rep. 2025 May 24;52(1):501. doi: 10.1007/s11033-025-10570-8.

DOI:10.1007/s11033-025-10570-8
PMID:40411639
Abstract

Mesenchymal stem cells (MSCs) have emerged as a promising option for gene and cell therapy due to their unique biological properties. MSC-based cell therapies have garnered significant attention for various clinical applications; however, repeated administrations are often necessary to achieve sustained therapeutic effects. Genetic modification techniques have enhanced MSCs' intrinsic capabilities, improving their therapeutic efficacy in both experimental and clinical settings. Key functional properties anti-inflammatory, anti-fibrotic, survival, and migratory capacities have become central targets for genetic enhancement. Numerous studies have explored the genetic modification of MSCs to address overcoming the transient nature of their therapeutic effects. Notably, the safety of genetically engineered MSCs remains a critical concern in preclinical and clinical investigations.In this review, we summarize current strategies for the genetic modification of MSCs and discuss recent findings on their application in animal disease models.

摘要

间充质干细胞(MSCs)因其独特的生物学特性,已成为基因治疗和细胞治疗的一个有前景的选择。基于MSCs的细胞疗法在各种临床应用中已引起广泛关注;然而,通常需要重复给药才能实现持续的治疗效果。基因改造技术增强了MSCs的内在能力,提高了它们在实验和临床环境中的治疗效果。关键的功能特性,如抗炎、抗纤维化、存活和迁移能力,已成为基因增强的核心靶点。许多研究探索了对MSCs进行基因改造,以解决其治疗效果的短暂性问题。值得注意的是,基因工程化MSCs的安全性在临床前和临床研究中仍然是一个关键问题。在这篇综述中,我们总结了目前MSCs基因改造的策略,并讨论了它们在动物疾病模型中应用的最新研究结果。

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本文引用的文献

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Mesenchymal stem cell exosome therapy: current research status in the treatment of neurodegenerative diseases and the possibility of reversing normal brain aging.间充质干细胞外泌体疗法:治疗神经退行性疾病的当前研究现状及逆转正常脑衰老的可能性
Stem Cell Res Ther. 2025 Feb 21;16(1):76. doi: 10.1186/s13287-025-04160-5.
2
Enhanced therapeutic effects of hypoxia-preconditioned mesenchymal stromal cell-derived extracellular vesicles in renal ischemic injury.缺氧预处理的间充质基质细胞衍生的细胞外囊泡对肾缺血损伤的治疗作用增强。
Stem Cell Res Ther. 2025 Feb 4;16(1):39. doi: 10.1186/s13287-025-04166-z.
3
Migrasomes derived from human umbilical cord mesenchymal stem cells: a new therapeutic agent for ovalbumin-induced asthma in mice.
源自人脐带间充质干细胞的迁移小体:一种治疗小鼠卵清蛋白诱导哮喘的新型治疗剂。
Stem Cell Res Ther. 2025 Jan 26;16(1):26. doi: 10.1186/s13287-025-04145-4.
4
miRTarBase 2025: updates to the collection of experimentally validated microRNA-target interactions.miRTarBase 2025:经实验验证的微小RNA-靶标相互作用集合的更新
Nucleic Acids Res. 2025 Jan 6;53(D1):D147-D156. doi: 10.1093/nar/gkae1072.
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Roles of extracellular vesicles from mesenchymal stem cells in regeneration.间充质干细胞来源的细胞外囊泡在再生中的作用。
Mol Cells. 2024 Dec;47(12):100151. doi: 10.1016/j.mocell.2024.100151. Epub 2024 Nov 13.
6
Optimization of lipofection protocols for CRISPR/Cas9 delivery in porcine zona pellucida intact oocytes: A study of coincubation duration and reagent efficacy.优化 CRISPR/Cas9 递送至猪卵透明带完整卵母细胞的脂质体转染方案:共孵育时间和试剂效力的研究。
Theriogenology. 2024 Dec;230:121-129. doi: 10.1016/j.theriogenology.2024.09.014. Epub 2024 Sep 14.
7
Decellularized Placental Sponge Seeded with Human Mesenchymal Stem Cells Improves Deep Skin Wound Healing in the Animal Model.接种人骨髓间充质干细胞的脱细胞胎盘海绵促进动物模型中深度皮肤伤口愈合
ACS Appl Bio Mater. 2024 Apr 15;7(4):2140-2152. doi: 10.1021/acsabm.3c00747. Epub 2024 Mar 12.
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Highly efficient genome editing via CRISPR-Cas9 ribonucleoprotein (RNP) delivery in mesenchymal stem cells.通过 CRISPR-Cas9 核糖核蛋白 (RNP) 递送来实现间充质干细胞中的高效基因组编辑。
BMB Rep. 2024 Jan;57(1):60-65. doi: 10.5483/BMBRep.2023-0113.
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Stem Cell Res Ther. 2023 May 24;14(1):138. doi: 10.1186/s13287-023-03364-x.
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Mol Biol Rep. 2023 Mar;50(3):2293-2304. doi: 10.1007/s11033-022-08111-8. Epub 2022 Dec 28.