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Current Gene Therapy

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ISSN (Print): 1566-5232
ISSN (Online): 1875-5631

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Review Article

Vector-Mediated Delivery of Transgenes and RNA Interference-Based Gene Silencing Sequences to Astrocytes for Disease Management: Advances and Prospectives

Author(s): Deepika Yadav and Rishabha Malviya*

Volume 24, Issue 2, 2024

Published on: 25 October, 2023

Page: [110 - 121] Pages: 12

DOI: 10.2174/0115665232264527231013072728

Price: $65

Abstract

Astrocytes are a type of important glial cell in the brain that serve crucial functions in regulating neuronal activity, facilitating communication between neurons, and keeping everything in balance. In this abstract, we explore current methods and future approaches for using vectors to precisely target astrocytes in the fight against various illnesses. In order to deliver therapeutic cargo selectively to astrocytes, researchers have made tremendous progress by using viral vectors such as adeno-associated viruses (AAVs) and lentiviruses. It has been established that engineered viral vectors are capable of either crossing the blood-brain barrier (BBB) or being delivered intranasally, which facilitates their entrance into the brain parenchyma. These vectors are able to contain transgenes that code for neuroprotective factors, synaptic modulators, or anti-inflammatory medicines, which pave the way for multiple approaches to disease intervention. Strategies based on RNA interference (RNAi) make vector-mediated astrocyte targeting much more likely to work. Small interfering RNAs (siRNAs) and short hairpin RNAs (shRNAs) are two types of RNA that can be made to silence disease-related genes in astrocytes. Vector-mediated delivery in conjunction with RNAi techniques provides a powerful toolkit for investigating the complex biological pathways that contribute to disease development. However, there are still a number of obstacles to overcome in order to perfect the specificity, safety, and duration of vector-mediated astrocyte targeting. In order to successfully translate research findings into clinical practise, it is essential to minimise off-target effects and the risk of immunogenicity. To demonstrate the therapeutic effectiveness of these strategies, rigorous preclinical investigation and validation are required.

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Graphical Abstract

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