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    VIEWPOINT

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

    MOHAMED AGHYAD AL KABBANI1,2, GILBERT WUNDERLICH3,4, CHRISTOPH KöHLER5, HANS ZEMPEL1,2,*

    BIOCELL, Vol.46, No.4, pp. 847-853, 2022, DOI:10.32604/biocell.2022.018144

    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… More >

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