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

Biocell

article

MOHAMED AGHYAD AL KABBANI¹  GILBERT WUNDERLICH³  CHRISTOPH KöHLER⁵  HANS ZEMPEL¹ 

[1] Faculty of Medicine and University Hospital Cologne, Institute of Human Genetics, University of Cologne;Faculty of Medicine and University Hospital Cologne, Center for Molecular Medicine Cologne ,(CMMC), University of Cologne;Faculty of Medicine and University Hospital Cologne, Department of Neurology, University of Cologne;Faculty of Medicine and University Hospital Cologne, Center for Rare Diseases, University of Cologne;Faculty of Medicine and University Hospital Cologne, Center Anatomy, Department of Molecular and Translational Neuroscience, University of Cologne

关键词: Tauopathies;    Neurogenetic diseases;    AAV-based gene therapy;    Neurodegeneration;    Alzheimer disease;    TAU;   

DOI  :  10.32604/biocell.2022.018144

学科分类：仪器

来源: Biocell

【 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.

【 授权许可】

CC BY