化学遗传学治疗痉挛的未来。

IF 4.4 3区 医学 Q1 GENETICS & HEREDITY
Molecular Diagnosis & Therapy Pub Date : 2025-09-01 Epub Date: 2025-07-30 DOI:10.1007/s40291-025-00798-2
Kelly Poth, Roy Raheb Khelo, Anthony Donsante, Nicholas M Boulis
{"title":"化学遗传学治疗痉挛的未来。","authors":"Kelly Poth, Roy Raheb Khelo, Anthony Donsante, Nicholas M Boulis","doi":"10.1007/s40291-025-00798-2","DOIUrl":null,"url":null,"abstract":"<p><p>The management of spasticity poses a significant challenge for both physicians and patients. This condition, often characterized by increased muscle tone, clonus, and muscle spasms, can be painful, disrupt daily activities, and increases the risk of injuries. First-line treatment for spasticity includes oral medications, such as baclofen, but these drugs can have significant side effects due to inhibitory effects on neural circuits, such as drowsiness and dizziness. Surgical approaches can be applied to patients for whom oral medications fail. However, these treatments can have life-threatening side effects or are irreversible. An emerging technology, chemogenetics, has the potential to provide targeted relief of spasticity, combining a non-destructive surgical approach with the convenience of an oral medication. This Current Opinion describes the current treatment approaches for spasticity, the pathophysiology of this condition, the current state of chemogenetics, and how this technology may be applied to patients with poorly controlled spasticity. Although the use of this approach is at an early developmental stage, we believe that it shows great promise.</p>","PeriodicalId":49797,"journal":{"name":"Molecular Diagnosis & Therapy","volume":" ","pages":"563-569"},"PeriodicalIF":4.4000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"The Future of Chemogenetics for the Treatment of Spasticity.\",\"authors\":\"Kelly Poth, Roy Raheb Khelo, Anthony Donsante, Nicholas M Boulis\",\"doi\":\"10.1007/s40291-025-00798-2\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>The management of spasticity poses a significant challenge for both physicians and patients. This condition, often characterized by increased muscle tone, clonus, and muscle spasms, can be painful, disrupt daily activities, and increases the risk of injuries. First-line treatment for spasticity includes oral medications, such as baclofen, but these drugs can have significant side effects due to inhibitory effects on neural circuits, such as drowsiness and dizziness. Surgical approaches can be applied to patients for whom oral medications fail. However, these treatments can have life-threatening side effects or are irreversible. An emerging technology, chemogenetics, has the potential to provide targeted relief of spasticity, combining a non-destructive surgical approach with the convenience of an oral medication. This Current Opinion describes the current treatment approaches for spasticity, the pathophysiology of this condition, the current state of chemogenetics, and how this technology may be applied to patients with poorly controlled spasticity. Although the use of this approach is at an early developmental stage, we believe that it shows great promise.</p>\",\"PeriodicalId\":49797,\"journal\":{\"name\":\"Molecular Diagnosis & Therapy\",\"volume\":\" \",\"pages\":\"563-569\"},\"PeriodicalIF\":4.4000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Diagnosis & Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s40291-025-00798-2\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/7/30 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q1\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Diagnosis & Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40291-025-00798-2","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/7/30 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0

摘要

痉挛的管理对医生和患者都是一个重大的挑战。这种情况通常以肌肉紧张度增加、阵挛和肌肉痉挛为特征,可能会引起疼痛,扰乱日常活动,并增加受伤的风险。痉挛的一线治疗包括口服药物,如巴氯芬,但由于这些药物对神经回路的抑制作用,可能有明显的副作用,如嗜睡和头晕。口服药物治疗无效的患者可采用手术治疗。然而,这些治疗方法可能会产生危及生命的副作用,或者是不可逆的。一项新兴的技术,化学遗传学,结合了非破坏性的手术方法和口服药物的便利,有可能提供有针对性的痉挛缓解。本《当前意见》描述了目前痉挛的治疗方法、这种疾病的病理生理学、化学遗传学的现状,以及该技术如何应用于控制不佳的痉挛患者。虽然这种方法的使用还处于早期发展阶段,但我们相信它显示出巨大的希望。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The Future of Chemogenetics for the Treatment of Spasticity.

The management of spasticity poses a significant challenge for both physicians and patients. This condition, often characterized by increased muscle tone, clonus, and muscle spasms, can be painful, disrupt daily activities, and increases the risk of injuries. First-line treatment for spasticity includes oral medications, such as baclofen, but these drugs can have significant side effects due to inhibitory effects on neural circuits, such as drowsiness and dizziness. Surgical approaches can be applied to patients for whom oral medications fail. However, these treatments can have life-threatening side effects or are irreversible. An emerging technology, chemogenetics, has the potential to provide targeted relief of spasticity, combining a non-destructive surgical approach with the convenience of an oral medication. This Current Opinion describes the current treatment approaches for spasticity, the pathophysiology of this condition, the current state of chemogenetics, and how this technology may be applied to patients with poorly controlled spasticity. Although the use of this approach is at an early developmental stage, we believe that it shows great promise.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
7.80
自引率
2.50%
发文量
53
审稿时长
>12 weeks
期刊介绍: Molecular Diagnosis & Therapy welcomes current opinion articles on emerging or contentious issues, comprehensive narrative reviews, systematic reviews (as outlined by the PRISMA statement), original research articles (including short communications) and letters to the editor. All manuscripts are subject to peer review by international experts.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信