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用于治疗杜氏肌营养不良症的含细胞外基质肽聚合物颗粒的科技展望

Scientific and Technological Prospecting on Polymeric Particles Containing Extracellular Matrix Peptides for the Treatment of Duchenne Muscular Dystrophy.

作者信息

Filgueiras Livia Alves, Bandeira Silva Francisco Thiago, das Chagas Alves Lima Francisco, Mendes Anderson Nogueira

机构信息

Department of Biophysics and Physiology, Laboratory of Innovation in Science and Technology - LACITEC, Federal University of Piauí, Teresina, Piauí, PI, Brazil.

Postgraduate Program in Biotechnology (RENORBIO), Federal University of Piauí, 64049-550, Teresina, Piauí, Brazil.

出版信息

Recent Adv Drug Deliv Formul. 2025;19(2):105-126. doi: 10.2174/0126673878329404250106065202.

DOI:10.2174/0126673878329404250106065202
PMID:39886796
Abstract

Duchenne muscular dystrophy is a neuromuscular disease with an overall incidence of between 1 in 5,000 newborn males. Carriers may manifest progressive muscle weakness, resulting from the progressive degeneration of skeletal muscles, generating cardiac and respiratory disorders. Considering the lack of effective treatments, different therapeutic approaches have been developed, such as protein synthesis and extracellular matrix derivatives that can be used to improve muscle regeneration, maintenance, or repair. At the same time, the use of other anti-inflammatory drugs or biological agents to replace corticosteroids conjugated to these extracellular matrix derivatives may act more effectively in controlling the progression of Duchenne muscular dystrophy. Extracellular matrix-derived peptides (e.g. laminin-111 derivatives) and the use of essential oils with antiinflammatory activity in polymeric particles for application in the treatment of Duchenne muscular dystrophy are discussed. For this purpose, the literature of patents and scientific articles from 2012- 2024 on LM-111 peptides and Duchenne muscular dystrophy was reviewed. Many patents focus on palliative technologies that seek to prolong the progressive effects of the disease, considering the control of the inflammatory process. The technological and scientific prospecting suggests the need for continuous research on systems that can serve as a treatment for Dystrophy.

摘要

杜氏肌营养不良症是一种神经肌肉疾病,在新生男婴中的总体发病率约为五千分之一。携带者可能会出现进行性肌肉无力,这是由骨骼肌的进行性退化引起的,进而导致心脏和呼吸系统疾病。鉴于缺乏有效的治疗方法,人们开发了不同的治疗方法,例如可用于改善肌肉再生、维持或修复的蛋白质合成和细胞外基质衍生物。同时,使用其他抗炎药物或生物制剂来替代与这些细胞外基质衍生物结合的皮质类固醇,可能在控制杜氏肌营养不良症的进展方面更有效。本文讨论了细胞外基质衍生肽(如层粘连蛋白-111衍生物)以及在聚合物颗粒中使用具有抗炎活性的精油用于治疗杜氏肌营养不良症。为此,回顾了2012年至2024年关于LM-111肽和杜氏肌营养不良症的专利文献和科学文章。许多专利侧重于姑息技术,即考虑到炎症过程的控制,试图延长疾病的进展性影响。技术和科学展望表明,需要持续研究可作为肌营养不良症治疗方法的系统。

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本文引用的文献

1
Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients.对接受腺相关病毒基因治疗的杜氏肌营养不良症患者的免疫细胞进行单细胞和TCR分析。
Mol Ther Methods Clin Dev. 2024 Sep 30;32(4):101349. doi: 10.1016/j.omtm.2024.101349. eCollection 2024 Dec 12.
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Peptide-based therapeutics targeting genetic disorders.针对遗传性疾病的肽类疗法。
Drug Discov Today. 2024 Dec;29(12):104209. doi: 10.1016/j.drudis.2024.104209. Epub 2024 Oct 16.
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Advancements in Drug Delivery Systems for the Treatment of Sarcopenia: An Updated Overview.
药物输送系统治疗肌肉减少症的研究进展:最新综述。
Int J Mol Sci. 2024 Oct 7;25(19):10766. doi: 10.3390/ijms251910766.
4
Oral administration of plumbagin is beneficial in in vivo models of Duchenne muscular dystrophy through control of redox signaling.通过控制氧化还原信号,口服白花丹醌对杜氏肌营养不良症的体内模型有益。
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Upregulation of utrophin improves the phenotype of Duchenne muscular dystrophy hiPSC-derived CMs.上调肌动蛋白可改善杜氏肌营养不良症人诱导多能干细胞衍生心肌细胞的表型。
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Comprehensive review of adverse reactions and toxicology in ASO-based therapies for Duchenne Muscular Dystrophy: From FDA-approved drugs to peptide-conjugated ASO.杜氏肌营养不良症基于反义寡核苷酸疗法的不良反应与毒理学综合综述:从美国食品药品监督管理局批准的药物到肽缀合反义寡核苷酸
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Synergistic Polymer Blending Informs Efficient Terpolymer Design and Machine Learning Discerns Performance Trends for pDNA Delivery.协同聚合物共混物为高效三元共聚物设计提供信息,机器学习可识别 pDNA 递送的性能趋势。
Bioconjug Chem. 2024 Jul 17;35(7):897-911. doi: 10.1021/acs.bioconjchem.4c00028. Epub 2024 Jun 26.
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Diapocynin treatment induces functional and structural improvements in an advanced disease state in the mdx mice.地波辛尼治疗可改善 mdx 小鼠的晚期疾病状态的功能和结构。
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Polymeric Nanoparticles for Drug Delivery.高分子纳米粒药物递送系统
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The extracellular matrix differentially directs myoblast motility and differentiation in distinct forms of muscular dystrophy: Dystrophic matrices alter myoblast motility.细胞外基质在不同形式的肌肉萎缩症中差异指导成肌细胞的迁移和分化:萎缩性基质改变成肌细胞的迁移。
Matrix Biol. 2024 May;129:44-58. doi: 10.1016/j.matbio.2024.04.001. Epub 2024 Apr 4.