Title:Vector-Mediated Delivery of Transgenes and RNA Interference-Based
Gene Silencing Sequences to Astrocytes for Disease Management:
Advances and Prospectives
Volume: 24
Issue: 2
Author(s): Deepika Yadav and Rishabha Malviya*
Affiliation:
- Department of Pharmacy, School of Medical and Allied Sciences, Galgotias University, Greater Noida, Uttar
Pradesh, India
Keywords:
Gene silencing, astrocyte, miRNA, lentiviral vector, targeted therapy, genomics, gene therapy.
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.
