Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
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Gene editing approaches introduce additional concerns, including irreversible genomic modification, chronic expression, or immune activation, which are especially problematic in aged or diseased brains.
“Gene editing approaches introduce additional concerns, including irreversible genomic modification, chronic expression, or immune activation, which are especially problematic in aged or diseased brains.”
From Reverse the progression of Alzheimer’s disease through Nano-ERASER-based adult neuroregeneration (2026) · cites this paper as [19]
The indexed copy contains a correction notice (Neuron, vol. 102, p. 263) about an AAV vector dose, rather than discussion of gene-editing risks. Searches for irreversible genomic modification, chronic expression, immune activation, and aged or diseased brains found no passage addressing the claim. (LLM, conf=0.75)
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“However, while AAVs are promising therapeutic gene delivery vehicles, 42 particularly for many neurological disorders, 43 – 45 they possess a limited carrying capacity that restricts their ability to deliver a full-length base editor.”
BackgroundTherapeutic repair for spinal cord injury: combinatory approaches to address a multifaceted problem
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“However, with this route of delivery, high vector doses resulting in widespread systemic exposure are required to achieve clinically relevant levels of transgene expression in the CNS, which may result in more pronounced immune responses(23) and the potential for other end-organ injury such as hepatotoxicity.(24) Deli…”
BackgroundChoice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate
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A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4 Mice
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“Several routes have been exploited in order to bypass the BBB and deliver AAVs to the brain in both preclinical models and humans.(16) Local administration has been used to deliver therapeutic genes to peripheral sensory organs, while the intrathecal route can target specific areas of the brain parenchyma.”
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“Viral gene therapy offers the unique opportunity to obtain longlived therapeutic effects, particularly for neurological diseases where the treatment needs to reach restricted tissues such as the brain and achieve high levels of transduction (27).”
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“Therefore, there is a clear need to identify other therapeutic approaches for TSC. Adeno-associated virus (AAV) vectors have been used widely in clinical trials for many hereditary diseases with little-to-no toxicity, long-term action in nondividing cells, and improvement in symptoms ( 9 – 11 ).”
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“disorders, as well as in preclinical disease models.(11,12) We previously obtained proof of concept for this approach in a MPS IIIA mouse model, where unilateral intracranial injection of an AAVrh.”
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Blocking cholesterol storage to treat Alzheimer’s disease
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“Efficient transgene expression either in a broad or cell type-specific fashion requires binding and action of cell-derived TFs to the promoter region.6,47 Changes in the neuronal environment such as aging or differentiation can also alter the recruitment of cell-specific regulatory proteins and therefore gene expressi…”
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“Adeno-associated virus (AAV) has long attracted attention as a safe and ef fi cient vector for gene delivery (47, 48).”
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“Other intraoperative surgeries in humans and/or macaques could benefit from accurate dMRI data on white matter organization and connectivity, including the current push for adeno-associated virus-mediated gene therapies (Hudry and Vandenberghe, 2019; Weiss et al., 2022).”
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“Our current study used only intrastriatal injection (which generates localized delivery) for both AAV and crRNA / P.O. Intracerebroventricular (ICV) injection for both AAV and crRNA / P.O. could be tested and may enhance editing and distribution in CNS tissues ( 106 , 113 , 114 , 136–140 ).”
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“38,39 Currently, AAV-based delivery is dominating this space.”
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“The usual administration is via intrathecal administration using adeno-associated viruses (AAVs), but novel capsids are under evaluation for neurodegenerative diseases [67].”
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“Gene therapy produces an alternate treatment for agents that fail to penetrate numerous physiologic barriers [2–4].”
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“2,3,8 Its innate ability to deliver gene expression locally and to a defined cell population adds powerful dimensions to AAV-tailoring for disease treatment.”
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Dere, V. V., Patil, S. S., Kale, A. Y., & Otari, K. V. (2026) · Biosciences Biotechnology Research Asia · doi:10.13005/bbra/3515
- Authors
- Hudry, E., & Vandenberghe, L. H.
- Corresponding author
- Vandenberghe, Luk H.
- ORCID
- Vandenberghe, Luk H.
- Venue
- Neuron
- Publisher
- Elsevier BV
- Published
- 2019-04
- Volume
- 102
- Issue
- 1
- Pages
- 263
- ISSN
- 0896-6273 (ISSN-L)
- OpenAlex
- W4252656301
- Cited by
- 77
- References
- 0
- Citations by year
- 2026: 10 · 2025: 13 · 2024: 12 · 2023: 6 · 2022: 11 · 2021: 10 · 2020: 8 · 2019: 7
- FWCI
- 5.36
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Neuron (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- adeno-associated virus vectors, gene therapy, neurological disorders, lysosomal storage disease, MPS IIIA, vector dose, clinical trials
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