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Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa.系统性同种异体间充质基质细胞疗法治疗隐性营养不良型大疱性表皮松解症患儿的潜力
J Invest Dermatol. 2015 Sep;135(9):2319-2321. doi: 10.1038/jid.2015.158. Epub 2015 Apr 23.
2
Phase I/II open-label trial of intravenous allogeneic mesenchymal stromal cell therapy in adults with recessive dystrophic epidermolysis bullosa.I 期/II 期开放性标签试验：静脉输注同种异体间充质基质细胞疗法治疗隐性营养不良型大疱性表皮松解症成人患者。
J Am Acad Dermatol. 2020 Aug;83(2):447-454. doi: 10.1016/j.jaad.2019.11.038. Epub 2019 Nov 28.
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Toxic Epidermal Necrolysis in Recessive Dystrophic Epidermolysis Bullosa following Bone Marrow Transplantation.骨髓移植后隐性营养不良型大疱性表皮松解症中的中毒性表皮坏死松解症
J Pediatr. 2016 Jun;173:242-4. doi: 10.1016/j.jpeds.2016.02.037. Epub 2016 Mar 11.
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Reduced Toxicity Conditioning and Allogeneic Hematopoietic Progenitor Cell Transplantation for Recessive Dystrophic Epidermolysis Bullosa.低毒性预处理及异基因造血祖细胞移植治疗隐性营养不良性大疱性表皮松解症
J Pediatr. 2015 Sep;167(3):765-9.e1. doi: 10.1016/j.jpeds.2015.05.051. Epub 2015 Jul 3.
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Preconditioning of mesenchymal stem cells for improved transplantation efficacy in recessive dystrophic epidermolysis bullosa.对间充质干细胞进行预处理以提高其在隐性营养不良性大疱性表皮松解症中的移植效果。
Stem Cell Res Ther. 2014 Nov 6;5(6):121. doi: 10.1186/scrt511.
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A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa.一项用于隐性营养不良型大疱性表皮松解症的皮内同种异体成纤维细胞的 II 期随机对照试验。
J Am Acad Dermatol. 2013 Dec;69(6):898-908.e7. doi: 10.1016/j.jaad.2013.08.014. Epub 2013 Sep 24.
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Inpatient management of children with recessive dystrophic epidermolysis bullosa: A review.隐性营养不良型大疱性表皮松解症患儿的住院治疗：综述
Pediatr Dermatol. 2017 Nov;34(6):647-655. doi: 10.1111/pde.13276. Epub 2017 Sep 25.
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Japanese guidelines for diagnosis and treatment of junctional and dystrophic epidermolysis bullosa.日本交界性和营养不良性大疱性表皮松解症诊断与治疗指南。
Arch Dermatol Res. 2003 Apr;295 Suppl 1:S24-8. doi: 10.1007/s00403-002-0379-y. Epub 2003 Mar 11.
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Recessive Dystrophic Epidermolysis Bullosa: Advances in the Laboratory Leading to New Therapies.隐性营养不良性大疱性表皮松解症：实验室进展带来新疗法
J Invest Dermatol. 2015 Jul;135(7):1705-1707. doi: 10.1038/jid.2015.149.
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Dystrophic epidermolysis bullosa.营养不良性大疱性表皮松解症
J Perinatol. 2004 Jan;24(1):41-7. doi: 10.1038/sj.jp.7211019.

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Mesenchymal intravenous stromal cell infusions in children with recessive dystrophic epidermolysis bullosa: MissionEB protocol for a randomised, double-blinded, placebo-controlled, two-centre, crossover trial with an internal phase I dose de-escalation phase and open-label extension.间充质静脉基质细胞输注治疗隐性营养不良性大疱性表皮松解症患儿：MissionEB方案，一项随机、双盲、安慰剂对照、两中心、交叉试验，含内部I期剂量递减阶段和开放标签扩展阶段。
BMJ Open. 2025 May 21;15(5):e089857. doi: 10.1136/bmjopen-2024-089857.
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Evaluation of safety and efficacy of multiple intravenous and intraosseous doses of foetal liver-derived mesenchymal stem cells in children with severe osteogenesis imperfecta : the BOOST2B clinical trial protocol.多次静脉注射和骨内注射胎儿肝脏来源间充质干细胞治疗重度成骨不全患儿的安全性和有效性评估：BOOST2B临床试验方案
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Progress in skin gene therapy: From the inside and out.皮肤基因治疗的进展：由内而外
Mol Ther. 2025 May 7;33(5):2065-2081. doi: 10.1016/j.ymthe.2025.03.017. Epub 2025 Mar 12.
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Safety and tolerability of losartan to treat recessive dystrophic epidermolysis bullosa in children (REFLECT): an open-label, single-arm, phase 1/2 trial.氯沙坦治疗儿童隐性遗传性营养不良性大疱性表皮松解症的安全性和耐受性（REFLECT）：一项开放标签、单臂、1/2期试验。
EClinicalMedicine. 2024 Oct 30;77:102900. doi: 10.1016/j.eclinm.2024.102900. eCollection 2024 Nov.
5
Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa.隐性营养不良型大疱性表皮松解症的基因和细胞治疗用生物医学产品的现状。
Int J Mol Sci. 2024 Sep 24;25(19):10270. doi: 10.3390/ijms251910270.
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Revolutionizing dermatology: harnessing mesenchymal stem/stromal cells and exosomes in 3D platform for skin regeneration.颠覆皮肤科学：三维平台中利用间充质干细胞/基质细胞和外泌体进行皮肤再生。
Arch Dermatol Res. 2024 May 25;316(6):242. doi: 10.1007/s00403-024-03055-4.
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Twin Prime Editing Mediated Exon Skipping/Reinsertion for Restored Collagen VII Expression in Recessive Dystrophic Epidermolysis Bullosa.双同源 Prime 编辑介导的外显子跳跃/重新插入恢复隐性营养不良性大疱性表皮松解症中的 VII 型胶原表达。
J Invest Dermatol. 2024 Dec;144(12):2764-2777.e9. doi: 10.1016/j.jid.2024.04.013. Epub 2024 May 17.
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Donor Muse Cell Treatment Without HLA-Matching Tests and Immunosuppressant Treatment.供者骨髓间充质干细胞治疗，无需 HLA 配型检测及免疫抑制剂治疗。
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Gene-edited cells: novel allogeneic gene/cell therapy for epidermolysis bullosa.基因编辑细胞：用于大疱性表皮松解症的新型异基因基因/细胞疗法。
J Appl Genet. 2024 Dec;65(4):705-726. doi: 10.1007/s13353-024-00839-2. Epub 2024 Mar 9.
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Advances in Treatments for Epidermolysis Bullosa (EB): Emphasis on Stem Cell-Based Therapy.表皮松解症（EB）治疗的新进展：以干细胞为基础的治疗。
Stem Cell Rev Rep. 2024 Jul;20(5):1200-1212. doi: 10.1007/s12015-024-10697-4. Epub 2024 Mar 2.

本文引用的文献

1
Inherited epidermolysis bullosa: updated recommendations on diagnosis and classification.遗传性大疱性表皮松解症：诊断和分类的更新建议。
J Am Acad Dermatol. 2014 Jun;70(6):1103-26. doi: 10.1016/j.jaad.2014.01.903. Epub 2014 Mar 29.
2
Evaluation of wound care options in patients with recessive dystrophic epidermolysis bullosa: a costly necessity.隐性营养不良型大疱性表皮松解症患者伤口护理选择的评估：一项代价高昂但必要的工作。
Pediatr Dermatol. 2014 Jan-Feb;31(1):33-7. doi: 10.1111/pde.12243. Epub 2013 Nov 14.
3
A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa.一项用于隐性营养不良型大疱性表皮松解症的皮内同种异体成纤维细胞的 II 期随机对照试验。
J Am Acad Dermatol. 2013 Dec;69(6):898-908.e7. doi: 10.1016/j.jaad.2013.08.014. Epub 2013 Sep 24.
4
Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds: results of a randomized, vehicle-controlled trial.成纤维细胞疗法增强隐性营养不良型大疱性表皮松解症创面的初始愈合：一项随机、对照载体试验的结果。
Br J Dermatol. 2013 Nov;169(5):1025-33. doi: 10.1111/bjd.12599.
5
Bone marrow transplantation for recessive dystrophic epidermolysis bullosa.骨髓移植治疗隐性营养不良型大疱性表皮松解症。
N Engl J Med. 2010 Aug 12;363(7):629-39. doi: 10.1056/NEJMoa0910501.
6
Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa.两名隐性营养不良型大疱性表皮松解症患者接受同种异体间充质基质细胞皮内注射后，VII 型胶原得到补充，慢性溃疡皮肤得以再上皮化。
Cytotherapy. 2010 May;12(3):429-31. doi: 10.3109/14653241003587637.
7
Stem cell paracrine actions and tissue regeneration.干细胞旁分泌作用与组织再生。
Regen Med. 2010 Jan;5(1):121-43. doi: 10.2217/rme.09.74.
8
Extracutaneous manifestations and complications of inherited epidermolysis bullosa: part II. Other organs.遗传性大疱性表皮松解症的皮肤外表现及并发症：第二部分。其他器官。
J Am Acad Dermatol. 2009 Sep;61(3):387-402; quiz 403-4. doi: 10.1016/j.jaad.2009.03.053.
9
Paracrine factors of mesenchymal stem cells recruit macrophages and endothelial lineage cells and enhance wound healing.间充质干细胞的旁分泌因子可募集巨噬细胞和内皮谱系细胞，并促进伤口愈合。
PLoS One. 2008 Apr 2;3(4):e1886. doi: 10.1371/journal.pone.0001886.
10
Concise review: mesenchymal stem/multipotent stromal cells: the state of transdifferentiation and modes of tissue repair--current views.简要综述：间充质干/多能基质细胞：转分化状态与组织修复模式——当前观点
Stem Cells. 2007 Nov;25(11):2896-902. doi: 10.1634/stemcells.2007-0637. Epub 2007 Sep 27.

Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa.

作者信息

Petrof Gabriela, Lwin Su M, Martinez-Queipo Magdalena, Abdul-Wahab Alya, Tso Simon, Mellerio Jemima E, Slaper-Cortenbach Ineke, Boelens Jaap J, Tolar Jakub, Veys Paul, Ofuya Mercy, Peacock Janet L, Martinez Anna E, McGrath John A

机构信息

St John's Institute of Dermatology, King's College London (Guy's Campus), London, UK.

St John's Institute of Dermatology, King's College London (Guy's Campus), London, UK.

出版信息

J Invest Dermatol. 2015 Sep;135(9):2319-2321. doi: 10.1038/jid.2015.158. Epub 2015 Apr 23.

DOI:10.1038/jid.2015.158
PMID:25905587
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5696540/
Abstract
摘要