AAV-based gene therapy approaches for genetic forms of tauopathies and related neurogenetic disorders

被引：3

作者：

Al Kabbani, Mohamed Aghyad ^{[1
,2
,3
]}

Wunderlich, Gilbert ^{[1
,4
,5
]}

Koeler, Christoph ^{[1
,6
]}

Zempel, Hans ^{[1
,2
,3
]}

机构：

[1] Univ Cologne, Fac Med, D-50931 Cologne, Germany

[2] Univ Cologne, Univ Hosp Cologne, Inst Human Genet, D-50931 Cologne, Germany

[3] Univ Cologne, Univ Hosp Cologne, Ctr Mol Med Cologne CMMC, D-50931 Cologne, Germany

[4] Univ Cologne, Univ Hosp Cologne, Dept Neurol, D-50937 Cologne, Germany

[5] Univ Cologne, Univ Hosp Cologne, Ctr Rare Dis, D-50937 Cologne, Germany

[6] Univ Cologne, Univ Hosp Cologne, Ctr Anat, Dept Mol & Translat Neurosci, D-50931 Cologne, Germany

来源：

BIOCELL | 2022年 / 46卷 / 04期

关键词：

Tauopathies; Neurogenetic diseases; AAV-based gene therapy; Neurodegeneration; Alzheimer disease; TAU; ADENOASSOCIATED VIRUS; EFFICIENT TRANSDUCTION; VIRAL VECTORS; MOUSE MODEL; DELIVERY; TAU; EXPRESSION; TOXICITY; PRIMATES;

D O I：

10.32604/biocell.2022.018144

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Tauopathies comprise a spectrum of genetic and sporadic neurodegenerative diseases mainly characterized by the presence of hyperphosphorylated TAU protein aggregations in neurons or glia. Gene therapy, in particular adeno-associated virus (AAV)-based, is an effective medical approach for difficult-to-treat genetic diseases for which there are no convincing traditional therapies, such as tauopathies. Employing AAV-based gene therapy to treat, in particular, genetic tauopathies has many potential therapeutic benefits, but also drawbacks which need to be addressed in order to successfully and efficiently adapt this still unconventional therapy for the various types of tauopathies. In this Viewpoint, we briefly introduce some potentially treatable tauopathies, classify them according to their etiology, and discuss the potential advantages and possible problems of AAV-based gene therapy. Finally, we outline a future vision for the application of this promising therapeutic approach for genetic and sporadic tauopathies.

引用

页码：847 / 853

页数：7

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