Neural Regeneration Research ›› 2024, Vol. 19 ›› Issue (5): 1020-1026.doi: 10.4103/1673-5374.385288

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New insights into the therapeutic approaches for the treatment of tauopathies

Himanshi Singh1, 2, 3, Asmita Das2, Mohammad Moshahid Khan3, 4, 5, *, Tayebeh Pourmotabbed1, *   

  1. 1Department of Microbiology, Immunology and Biochemistry, University of Tennessee Health Science Center, Memphis, TN, USA; 2Department of Biotechnology, Delhi Technological University, Shahbad Daulatpur, Delhi, India; 3Department of Neurology, College of Medicine, University of Tennessee Health Science Center, Memphis, TN, USA; 4Neuroscience Institute, University of Tennessee Health Science Center, Memphis, TN, USA; 5Center for Muscle, Metabolism and Neuropathology, Division of Regenerative and Rehabilitation Sciences and Department of Physical Therapy, College of Health Professions, University of Tennessee Health Science Center, Memphis, TN, USA
  • Online:2024-05-15 Published:2023-10-31
  • Contact: Mohammad Moshahid Khan, PhD, mkhan26@uthsc.edu; Tayebeh Pourmotabbed, PhD, tpourmot@uthsc.edu.
  • Supported by:
    This work was supported by National Institute of Health grant number R03AG075597 (to MMK and TP) and Department of Defense Award Number HT9425-23-1-0043 (to MMK).

Abstract: Tauopathies are a group of neurological disorders, including Alzheimer’s disease and frontotemporal dementia, which involve progressive neurodegeneration, cognitive deficits, and aberrant tau protein accumulation. The development of tauopathies cannot currently be stopped or slowed down by treatment measures. Given the significant contribution of tau burden in primary tauopathies and the strong association between pathogenic tau accumulation and cognitive deficits, there has been a lot of interest in creating therapies that can alleviate tau pathology and render neuroprotective effects. Recently, small molecules, immunotherapies, and gene therapy have been used to reduce the pathological tau burden and prevent neurodegeneration in animal models of tauopathies. However, the major pitfall of the current therapeutic approach is the difficulty of drugs and gene-targeting modalities to cross the blood-brain barrier and their unintended side effects. In this review, the current therapeutic strategies used for tauopathies including the use of oligonucleotide-based gene therapy approaches that have shown a promising result for the treatment of tauopathies and Alzheimer’s disease in preclinical animal models, have been discussed.

Key words: dementia, gene therapies, immunotherapy, neurodegeneration, oligonucleotides, tau, tauopathies, therapies