ArticleMolecular therapy. Methods & clinical development2014
Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy.
Article in Molecular therapy. Methods & clinical development, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 34 citations in OpenAlex.
- Intrathecal delivery of AAVrh10-mHexa combined with anti-inflammatory treatment reduces neuropathological markers and extends the lifespan of mice with early-onset Tay-Sachs disease.Metabolic brain disease · 2026Article
- A dose escalation and safety study of AAVrh10-mediated Schwann cell-targeted gene therapy for CMT1X.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025Article
- AnMolecular therapy. Methods & clinical development · 2024Article
- IGF-1 as a Potential Therapy for Spinocerebellar Ataxia Type 3.Biomedicines · 2022Article
- Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery.Scientific reports · 2021Article
- AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy.Gene therapy · 2021Article
- Biodistribution of Adeno-Associated Virus Serotype 5 Viral Vectors Following Intrathecal Injection.Molecular pharmaceutics · 2021Article
- GRP78 Overexpression Triggers PINK1-IPBiomedicines · 2021Article
- Gene Therapy Overexpressing Neuregulin 1 Type I in Combination With Neuregulin 1 Type III Promotes Functional Improvement in the SOD1Frontiers in neurology · 2021Article
- Insulin-Like Growth Factor-1: A Promising Therapeutic Target for Peripheral Nerve Injury.Frontiers in bioengineering and biotechnology · 2021Review
- Therapeutic Role of Neuregulin 1 Type III in SOD1-Linked Amyotrophic Lateral Sclerosis.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2020Article
- Machine intelligence for nerve conduit design and production.Journal of biological engineering · 2020Review
- Article
- Intravascular injections of adenoassociated viral vector serotypes rh10 and PHP.B transduce murine sciatic nerve axons.Neuroscience letters · 2019Article
- On the development of optical peripheral nerve interfaces.Neural regeneration research · 2019Review
- Insulin-like growth factor-1 activates AMPK to augment mitochondrial function and correct neuronal metabolism in sensory neurons in type 1 diabetes.Molecular metabolism · 2019Article
- An automated method for the quantification of transgene expression in motor axons of the peripheral nerve.Journal of neuroscience methods · 2018Article
- ATG5 overexpression is neuroprotective and attenuates cytoskeletal and vesicle-trafficking alterations in axotomized motoneurons.Cell death & disease · 2018Article
- Neuroprotective Drug for Nerve Trauma Revealed Using Artificial Intelligence.Scientific reports · 2018Article
- Adeno-associated virus serotype rh10 is a useful gene transfer vector for sensory nerves that innervate bone in immunodeficient mice.Scientific reports · 2017Article
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Different adeno-associated virus (AAV) serotypes efficiently transduce neurons from central and peripheral nervous systems through various administration routes. Direct administration of the vectors to the cerebrospinal fluid (CSF) could be an efficient and safe strategy. Here, we show that lumbar puncture of a nonhuman AAV leads to wide and stable distribution of the vector along the spinal cord in adult mice. AAVrh10 efficiently and specifically infects neurons, both in dorsal root ganglia (60% total sensory neurons) and in the spinal cord (up to one-third of α-motor neurons). As a proof of concept, we demonstrate the efficacy of AAVrh10 in a mouse model of diabetic neuropathy, in which intrathecal delivery of the vector coding for insulin-like growth factor (IGF-I) favored the release of the therapeutic protein into the CSF through its expression by sensory and motor neurons. IGF-I-treated diabetic animals showed increased vascular endothelial growth factor expression, activation of Akt/PI3K pathway, and stimulated nerve regeneration and myelination in injured limbs. Moreover, we achieved restoration of nerve conduction velocities in both sensory and motor nerves by AAVrh10, whereas we reached only sensory nerve improvement with AAV1. Our results indicate that intrathecal injection of AAVrh10 is a promising tool to design gene therapy approaches for sensorimotor diseases.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.