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

Authors :
Al Kabbani, Mohamed Aghyad
Wunderlich, Gilbert
Koeler, Christoph
Zempel, Hans
Al Kabbani, Mohamed Aghyad
Wunderlich, Gilbert
Koeler, Christoph
Zempel, Hans
Publication Year :
2022

Abstract

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.

Details

Database :
OAIster
Notes :
English
Publication Type :
Electronic Resource
Accession number :
edsoai.on1383741445
Document Type :
Electronic Resource