MINI REVIEW article
Front. Med.
Sec. Gene and Cell Therapy
This article is part of the Research TopicNext-Generation Gene and Cell Therapies: Targeting Diseases at Their OriginView all 3 articles
Viral and non-viral cellular therapies for neurodegeneration
Provisionally accepted
Jyotsana Srivastav
Sachin Sharma*- Graphic Era Deemed to be University, Dehradun, India
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Neurodegenerative diseases such as Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), and amyotrophic lateral sclerosis (ALS) are characterized by progressive loss of neurons and still lack curative treatment options. In this review, we describe current and developing therapeutic strategies that include viral vector-based gene delivery, antisense oligonucleotide (ASO) and RNA interference methods, stem cell transplantation, and genome editing technologies. Adeno-associated viruses (AAVs) and lentiviruses have been used for gene delivery in preclinical and clinical studies, while ASOs are under development to reduce expression of pathogenic proteins such as tau, α-synuclein, and mutant huntingtin. Cellular therapies, including mesenchymal stem cell (MSC)-based paracrine support and transplantation of neurons derived from induced pluripotent stem cells (iPSCs), are being evaluated, particularly in PD and AD. We also discuss important gene targets such as APOE4, GBA1, SCNA, and MAPT, and how treatment strategies may differ between monogenic and polygenic forms of these disorders. Lastly, we highlight recent efforts focused on genes like TREM2, PINK1, and progranulin, and examine their role in the future development of gene-and cell-based interventions.
Keywords: neurodegeneration, Gene Therapy, Stem Cell Therapy, Antisense oligonucleotides (ASOs), adeno-associated virus (AAV)
Received: 04 Oct 2025; Accepted: 10 Dec 2025.
Copyright: © 2025 Srivastav and Sharma. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Sachin Sharma
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