AAV Provides an Alternative for Gene Therapy of the Peripheral Sensory Nervous System
Open accessAbstract
Recombinant adeno-associated virus (rAAV) is a promising vector for applications in the central nervous system (CNS). Clinical phase I/II studies have demonstrated safety of rAAV-expressing therapeutic transgenes for a variety of benign (i.e., noncancer) CNS disorders, namely, Canavan disease,1 Parkinson's disease,2,3,4 and Batten disease.5 Human studies were also recently reported targeting the retina.6,7,8,9,9 From the early stages of preclinical development, rAAV was appealing because it was found to be neurotrophic and capable of long-term activity without immune interference or toxicity when injected into the CNS.
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Use of intrathecal delivery for gene therapy vectors has been limited to studies involving spinal cord and peripheral nerve disease.
“Use of intrathecal delivery for gene therapy vectors has been limited to studies involving spinal cord and peripheral nerve disease.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [91]
The passage discussing PNS gene therapy (indexed under References; page unavailable) identifies intrathecal delivery of rAAV vectors among techniques studied for peripheral sensory conditions, including neuropathic pain. It does not say that intrathecal vector delivery has been limited to spinal cord and peripheral nerve disease studies; that restriction is unsupported. (LLM, conf=0.82)
Experimental — accuracy not yet measuredAfter intrathecal delivery, vectors stay predominantly in the lower spine.
“The vectors stay predominantly in the lower spine in intrathecal delivery administrations.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [91]
The discussion of intrathecal delivery (section labeled References; page unavailable) says that rAAV serotypes have been administered by that route, but does not describe where the vectors remain afterward. Searches for lower-spine localization and lumbar distribution found no passage addressing the claim. (LLM, conf=0.74)
Experimental — accuracy not yet measured
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References 50 · Cited by 19
- Authors
- Beutler, A. S.
- Affiliations
- Mayo Clinic (US)
- Corresponding author
- Beutler, Andreas S
- ORCID
- Beutler, Andreas S
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2010-04
- Volume
- 18
- Issue
- 4
- Pages
- 670-673
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC2862524
- OpenAlex
- W2022351373
- Cited by
- 19
- References
- 50
- Citations by year
- 2024: 1 · 2021: 2 · 2019: 3 · 2018: 1 · 2017: 2 · 2016: 2 · 2015: 1 · 2014: 2 · 2013: 2 · 2012: 2
- FWCI
- 0.90
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Mayo Clinic
- Richard M. Schulze Family Foundation
- National Institute of Neurological Disorders and Stroke (R21NS062271, R01NS063022)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Keywords
- recombinant adeno-associated virus, gene therapy vectors, neurotrophic effects, long-term transgene expression, immune tolerance, retinal gene therapy, Parkinson's disease, Batten disease, Canavan disease
- MeSH terms
- Dependovirus, Animals, Genetic Vectors, Humans, Peripheral Nervous System Diseases, Genetic Therapy, Reassortant Viruses, Mice
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