Gene Therapy Strategies for Alzheimer's Disease: An Overview
Abstract
Key neuropathological hallmarks of Alzheimer's disease (AD) are extracellular amyloid plaques and intracellular accumulation of hyperphosphorylated Tau protein. The mechanisms underlying these neuropathological changes remain unclear. So far, research on AD therapy has had limited success in terms of symptomatic treatments although it has also had several failures for disease-modifying drugs. Gene transfer strategies to the brain have contributed to evaluate in animal models many interesting tracks, some of which should deserve clinical applications in AD patients in the future.
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One strategy to prevent Alzheimer's disease in APOE4 carriers is to modify the central nervous system genetically to express APOE2, providing the CNS with the protective APOE2 to balance the Alzheimer's disease risk associated with expression of APOE4.
“Based on these observations, one strategy to prevent AD in APOE4 carriers is to modify the central nervous system (CNS) genetically to express APOE2, providing the CNS with the protective APOE2 to balance the AD risk associated with expression of APOE4.22–26”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [22]
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References 61 · Cited by 45
- Authors
- Alves, S., Fol, R., & Cartier, N.
- Affiliations
- Université Paris-Sud (FR)
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2016-02
- Volume
- 27
- Issue
- 2
- Pages
- 100-107
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2281605478
- Cited by
- 45
- References
- 61
- Citations by year
- 2026: 1 · 2024: 11 · 2023: 4 · 2022: 3 · 2021: 5 · 2020: 7 · 2019: 6 · 2018: 5 · 2017: 2 · 2016: 1
- FWCI
- 0.55
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Human Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Agence Nationale de la Recherche (ANR-10-MALZ-0103)
- Fondation pour la Recherche Médicale
- Topics
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Psychiatry and Mental health › Dementia and Cognitive Impairment Research
- Keywords
- Alzheimer's disease, gene therapy, amyloid plaques, hyperphosphorylated tau, disease-modifying drugs, symptomatic treatment, animal models of Alzheimer's disease, clinical translation
- MeSH terms
- Alzheimer Disease, Animals, Apolipoproteins E, Brain, Clinical Trials as Topic, Disease Models, Animal, Humans, Somatomedins, Genetic Therapy, Lentivirus, Aspartic Acid Endopeptidases, Disease Progression, Brain-Derived Neurotrophic Factor, RNA, Small Interfering, NF-E2-Related Factor 2, Amyloid Precursor Protein Secretases, Antibodies, Neutralizing, Plaque, Amyloid
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