治疗大疱性表皮松解症(EB)的进展:以干细胞疗法为重点。

IF 4.5 3区 医学 Q2 CELL & TISSUE ENGINEERING
Stem Cell Reviews and Reports Pub Date : 2024-07-01 Epub Date: 2024-03-02 DOI:10.1007/s12015-024-10697-4
Ramin Raoufinia, Hamid Reza Rahimi, Neda Keyhanvar, Meysam Moghbeli, Nima Abdyazdani, Mehdi Rostami, Karim Naghipoor, Fatemeh Forouzanfar, Sara Foroudi, Ehsan Saburi
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引用次数: 0

摘要

大疱性表皮松解症(EB)是一种罕见的遗传性皮肤病,以皮肤脆弱和水疱形成为特征。EB 具有广泛的表型谱和潜在的皮外表现,具有显著的发病率和死亡率风险。目前,EB 根据皮肤裂开的程度分为四大亚型,其病因是基因突变影响了对维持皮肤完整性至关重要的蛋白质。EB 的治疗主要侧重于预防并发症,并通过伤口护理、疼痛控制和其他支持性措施治疗症状。然而,干细胞疗法、组织工程和基因疗法领域的最新进展表明,这些疗法有望成为 EB 的潜在治疗方法。能够分化成皮肤细胞的干细胞通过促进伤口愈合和减少炎症,在临床前和早期临床试验中取得了积极成果。另一方面,基因疗法旨在通过引入突变基因的功能拷贝或修改现有基因来恢复蛋白质功能,从而纠正导致 EB 的潜在基因缺陷。特别是对于像隐性营养不良性表皮松解症(RDEB)这样的严重亚型,基因疗法具有巨大的潜力。本综述旨在评估新的治疗方法在 EB 治疗中的作用。综述包括对人类进行的研究结果。虽然早期研究和临床试验已显示出良好的效果,但仍有必要开展进一步的研究和试验,以确定这些治疗 EB 的创新方法的安全性和有效性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Advances in Treatments for Epidermolysis Bullosa (EB): Emphasis on Stem Cell-Based Therapy.

Advances in Treatments for Epidermolysis Bullosa (EB): Emphasis on Stem Cell-Based Therapy.

Epidermolysis bullosa (EB) is a rare genetic dermatosis characterized by skin fragility and blister formation. With a wide phenotypic spectrum and potential extracutaneous manifestations, EB poses significant morbidity and mortality risks. Currently classified into four main subtypes based on the level of skin cleavage, EB is caused by genetic mutations affecting proteins crucial for maintaining skin integrity. The management of EB primarily focuses on preventing complications and treating symptoms through wound care, pain management, and other supportive measures. However, recent advancements in the fields of stem cell therapy, tissue engineering, and gene therapy have shown promise as potential treatments for EB. Stem cells capable of differentiating into skin cells, have demonstrated positive outcomes in preclinical and early clinical trials by promoting wound healing and reducing inflammation. Gene therapy, on the other hand, aims to correct the underlying genetic defects responsible for EB by introducing functional copies of mutated genes or modifying existing genes to restore protein function. Particularly for severe subtypes like Recessive Dystrophic Epidermolysis Bullosa (RDEB), gene therapy holds significant potential. This review aims to evaluate the role of new therapeutic approaches in the treatment of EB. The review includes findings from studies conducted on humans. While early studies and clinical trials have shown promising results, further research and trials are necessary to establish the safety and efficacy of these innovative approaches for EB treatment.

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来源期刊
Stem Cell Reviews and Reports
Stem Cell Reviews and Reports 医学-细胞生物学
CiteScore
9.30
自引率
4.20%
发文量
0
审稿时长
3 months
期刊介绍: The purpose of Stem Cell Reviews and Reports is to cover contemporary and emerging areas in stem cell research and regenerative medicine. The journal will consider for publication: i) solicited or unsolicited reviews of topical areas of stem cell biology that highlight, critique and synthesize recent important findings in the field. ii) full length and short reports presenting original experimental work. iii) translational stem cell studies describing results of clinical trials using stem cells as therapeutics. iv) papers focused on diseases of stem cells. v) hypothesis and commentary articles as opinion-based pieces in which authors can propose a new theory, interpretation of a controversial area in stem cell biology, or a stem cell biology question or paradigm. These articles contain more speculation than reviews, but they should be based on solid rationale. vi) protocols as peer-reviewed procedures that provide step-by-step descriptions, outlined in sufficient detail, so that both experts and novices can apply them to their own research. vii) letters to the editor and correspondence. In order to facilitate this exchange of scientific information and exciting novel ideas, the journal has created five thematic sections, focusing on: i) the role of adult stem cells in tissue regeneration; ii) progress in research on induced pluripotent stem cells, embryonic stem cells and mechanism governing embryogenesis and tissue development; iii) the role of microenvironment and extracellular microvesicles in directing the fate of stem cells; iv) mechanisms of stem cell trafficking, stem cell mobilization and homing with special emphasis on hematopoiesis; v) the role of stem cells in aging processes and cancerogenesis.
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