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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.

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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