Key Features in the Design and Function of Nanocarriers for Intranasal Administration of Gene Therapy in Huntington Disease.
Key Features in the Design and Function of Nanocarriers for Intranasal Administration of Gene Therapy in Huntington Disease.
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DOI：
10.33696/nanotechnol.4.043
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发表时间：
2023
期刊：
影响因子：
--
通讯作者：
Sanchez-Ramos, Juan
中科院分区：
文献类型：
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作者：
Fihurka, Oksana;Aradi, Stephen;Sava, Vasyl;Sanchez-Ramos, Juan
实现遗传性脑部疾病（如亨廷顿病（HD））基因疗法的治疗承诺的主要障碍是对病毒载体和/或侵入性递送系统的要求（立体定向注射到大脑或注入鞘内空间）。HD是一种常染色体显性神经退行性疾病，一些临床试验已经证明鞘内给药后基因降低效果。这些技术上的限制推动了脑部疾病基因治疗的非侵入性递送系统的发展。本综述的总体目标是讨论用于HD基因治疗的鼻内给药纳米载体设计的关键特征，主要关注我们在基因治疗中使用纳米载体的一系列已发表的工作。基因降低药物包装的纳米载体的设计和开发代表了HD和其他遗传性脑部疾病的无创鼻到脑基因治疗的重大进展。
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.