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用于亨廷顿病基因治疗鼻内给药的纳米载体的设计与功能关键特征

Key Features in the Design and Function of Nanocarriers for Intranasal Administration of Gene Therapy in Huntington Disease.

作者信息

Fihurka Oksana, Aradi Stephen, Sava Vasyl, Sanchez-Ramos Juan

机构信息

Department of Neurology, University of South Florida, USA.

出版信息

J Nanotechnol Nanomater. 2023;4(2):55-69. doi: 10.33696/nanotechnol.4.043.

DOI:10.33696/nanotechnol.4.043
PMID:37744989
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10514752/
Abstract

A major obstacle to fulfilling the therapeutic promise of gene therapies for hereditary brain diseases, such as Huntington' Disease (HD), is the requirement for viral vectors and/or an invasive delivery system (stereotaxic injection into brain or infusion into the intrathecal space). HD is an autosomal dominant neurodegenerative disease for which several clinical trials have demonstrated gene-lowering effects following intrathecal administration. These technical limitations have given impetus to the development of alternative non-invasive delivery systems for gene therapy of brain diseases. The overall objective of this review is to discuss the key features in the design of nanocarriers for intranasal administration of gene-therapy for HD, focusing primarily on our series of published work on the use of nanocarriers for gene therapy. Design and development of nanocarriers packaged with gene-lowering agents represents a significant advance towards non-invasive nose-to-brain delivery of gene therapy for HD and other hereditary brain disorders.

摘要

对于亨廷顿舞蹈症（HD）等遗传性脑部疾病而言，基因疗法要实现其治疗前景，一个主要障碍是需要病毒载体和/或侵入性给药系统（立体定向脑内注射或鞘内注射）。HD是一种常染色体显性神经退行性疾病，多项临床试验已证明鞘内给药后具有基因降低效应。这些技术限制推动了用于脑部疾病基因治疗的替代性非侵入性给药系统的开发。本综述的总体目标是讨论用于HD基因治疗鼻内给药的纳米载体设计的关键特征，主要聚焦于我们一系列关于使用纳米载体进行基因治疗的已发表研究。包装有基因降低剂的纳米载体的设计与开发代表了HD和其他遗传性脑部疾病基因治疗非侵入性鼻脑给药方面的重大进展。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9616/10514752/2544073fa476/nihms-1930121-f0013.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9616/10514752/27450d677c23/nihms-1930121-f0007.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9616/10514752/2bbafe72e162/nihms-1930121-f0008.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9616/10514752/cdf1be4e0f16/nihms-1930121-f0009.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9616/10514752/07b50f32a99f/nihms-1930121-f0010.jpg
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https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9616/10514752/2544073fa476/nihms-1930121-f0013.jpg

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