Volume 29, Issue 3 (8-2026)
J Arak Uni Med Sci 2026, 29(3): 0-0 |
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Soltani M, Karami H, Satarzadeh M. Evaluating the Effectiveness of Adeno-Associated Virus (AAV) Vectors in Gene Therapy of Animal Models of Amyotrophic Lateral Sclerosis: A Systematic Review. J Arak Uni Med Sci 2026; 29 (3)
URL: http://jams.arakmu.ac.ir/article-1-8188-en.html
URL: http://jams.arakmu.ac.ir/article-1-8188-en.html
1- General Practitioner, Student Research Committee, Arak University of Medical Sciences, Arak, Markazi Province, Iran
2- Associate Professor, Department of Molecular Medicine and Biotechnology, School of Medicine, Arak University of Medical Sciences, Arak, Iran
3- Bachelor of Nursing, Student Research Committee, Hormozgan University of Medical Sciences, Bandar Abbas, Hormozgan Province, Iran ,m.satarzadeh78@gmail.com
2- Associate Professor, Department of Molecular Medicine and Biotechnology, School of Medicine, Arak University of Medical Sciences, Arak, Iran
3- Bachelor of Nursing, Student Research Committee, Hormozgan University of Medical Sciences, Bandar Abbas, Hormozgan Province, Iran ,
Abstract: (57 Views)
Introduction: Amyotrophic lateral sclerosis (ALS) is a degenerative immune system disease that results from inflammation of neurons, primarily affecting motor neurons. Mutations in the SOD1 gene are implicated in ALS and disrupt many proteins and factors. Gene therapy is being pursued as a treatment for ALS in animal models, and this study sought to investigate the role of adeno-associated virus (AAV) in this type of therapy.
Methods: We reviewed studies in 5 years from 2020 to 2025 in PubMed, Scopus, and Web of Science databases. We extracted our keywords from the MeSH database and wrote our study based on PRISMA. We used the SYRCLE’s risk of bias tool to assess the risk of bias.
Results: Twenty-one studies were included in this systematic review, all of which were animal studies, and used the AAV family as vectors. The majority were AAV serotype 9, which was injected intravenously, intrathecally, and in most cases intracerebrovascularly. These strains showed the ability to carry plasmids encoding brain factors and regulatory proteins, which ultimately improved motor function, neuromuscular junctions (NMJs) balance, and ultimately reduced the accumulation of mutant SOD1 in affected mice.
Conclusions: Our results showed that AAVs have the potential to treat ALS as vectors; this group has the ability to cross the blood-brain barrier; however, the current studies are limited to animal populations. We recommend that clinical studies based on the AAV family as gene therapy for ALS patients challenge our findings.
Methods: We reviewed studies in 5 years from 2020 to 2025 in PubMed, Scopus, and Web of Science databases. We extracted our keywords from the MeSH database and wrote our study based on PRISMA. We used the SYRCLE’s risk of bias tool to assess the risk of bias.
Results: Twenty-one studies were included in this systematic review, all of which were animal studies, and used the AAV family as vectors. The majority were AAV serotype 9, which was injected intravenously, intrathecally, and in most cases intracerebrovascularly. These strains showed the ability to carry plasmids encoding brain factors and regulatory proteins, which ultimately improved motor function, neuromuscular junctions (NMJs) balance, and ultimately reduced the accumulation of mutant SOD1 in affected mice.
Conclusions: Our results showed that AAVs have the potential to treat ALS as vectors; this group has the ability to cross the blood-brain barrier; however, the current studies are limited to animal populations. We recommend that clinical studies based on the AAV family as gene therapy for ALS patients challenge our findings.
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