Abstract
Neurological diseases (NDs) are one of the leading causes of disability and the second leading cause of death globally. Among these stroke, Alzheimer's disease (AD), and Parkinson's disease (PD) are the most common NDs. A rise in the absolute number of individuals affected with these diseases indicates that the current treatment strategies in management and prevention of these debilitating diseases are not effective sufficiently. Therefore, novel treatment strategies are being explored to cure these diseases by addressing the causative mechanisms at the molecular level. Advanced therapies like gene therapy (gene editing and gene silencing) and stem cell therapies aim to cure diseases by gene editing, gene silencing and tissue regeneration, respectively. Gene editing results in the deletion of the aberrant gene or insertion of the corrected gene which can be executed using the CRISPR/Cas gene editing tool a promising treatment strategy being explored for many other prevalent diseases. Gene silencing using siRNA silences the gene by inhibiting protein translation, thereby silencing its expression. Stem cell therapy aims to regenerate damaged cells or tissues because of their ability to divide into any type of cell in the human body. Among these approaches, gene editing and gene silencing have currently been applied in vitro and to animal models, while stem cell therapy has reached the clinical trial stage for the treatment of NDs. The current status of these strategies suggests a promising outcome in their clinical translation.
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Acknowledgements
Financial assistance from DST-FIST, Indian Council of Medical Research (ICMR), New Delhi, and Council for Scientific and Industrial Research (CSIR), India, is highly acknowledged.
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Financial support from ICMR, New Delhi (RFC No. (p-64) NCD/Adhoc/139/2020–21), DST-FIST (SR/FST/LS-I/2017/49) and SRF awarded to Mr. Abhilash Ludhiadch (Award No-09/ 1051(0029)/2 019-EMR-1) from the Council for Scientific and Industrial Research (CSIR) India is acknowledged with thanks.
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Katta, M., Mathew, B.A., Chaturvedi, P. et al. Advanced molecular therapies for neurological diseases: focus on stroke, alzheimer's disease, and parkinson's disease. Neurol Sci 44, 19–36 (2023). https://doi.org/10.1007/s10072-022-06356-6
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DOI: https://doi.org/10.1007/s10072-022-06356-6