Astrocyte transduction is required for rescue of behavioral phenotypes in the YAC128 mouse model with AAV-RNAi mediated HTT lowering therapeutics
Open accessAbstract
Huntington's disease (HD) is a fatal autosomal dominant neurodegenerative disease caused by a CAG expansion, which translates into an elongated polyglutamine (polyQ) repeat near the amino-terminus of the huntingtin protein (HTT). This results in production of a toxic mutant huntingtin protein (mHTT) that leads to neuronal dysfunction and death. Currently, no disease-modifying treatments are available; however, numerous therapeutic strategies aimed at lowering HTT levels in the brain are under development. To date, studies have not closely examined the contribution of mHTT in neurons vs astrocytes to disease pathophysiology. To better understand the role of astrocytes in HD pathophysiology and the need for cell type specific targeting of HTT lowering therapeutic strategies, AAV capsids were employed that selectively transduce neurons, or both neurons and astrocytes. These vectors carrying miRNA sequences directed against HTT were injected into the YAC128 mouse model of HD to selectively lower HTT expression in neurons alone versus neurons and astrocytes. The results suggested that HTT lowering in neurons alone was not sufficient to rescue the motor phenotype in YAC128 mice. Furthermore, HTT lowering in both cell types was required to achieve maximal functional benefit. The study suggested that astrocyte dysfunction may play a critical role in HD pathogenesis, and thus astrocytes represent an important therapeutic target.
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In YAC128 Huntington's disease mice, motor deficits were rescued when HTT-suppressing miRNA was delivered bilaterally into the striatum to neurons plus astrocytes (AAV1), but not when delivered to neurons only (AAV2-HBKO).
“Furthermore, bilateral intrastriatal AAV-RNAi delivery of HTT-suppressing miRNA targeted to neurons and astrocytes (AAV1), but not neurons alone (AAV2-HBKO), rescued motor deficits in the YAC128 mouse model of Huntington's disease”
From Microglial depletion prevents extracellular matrix changes and striatal volume reduction in a model of Huntington's disease (2020) · cites this paper as Stanek et al. , 2019
Astrocyte mHTT directly alters reactivity in a way that worsens disease.
“suggesting that astrocyte mHTT directly alters reactivity in a manner detrimental to disease (Stanek et al. , 2019).”
From Microglial depletion prevents extracellular matrix changes and striatal volume reduction in a model of Huntington's disease (2020) · cites this paper as Stanek et al. , 2019
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- Authors
- Stanek, L. M., Bu, J., & Shihabuddin, L. S.
- Affiliations
- Sanofi (United States) (US)
- Corresponding author
- Stanek, Lisa M.
- ORCID
- Bu, Jie
- Venue
- Neurobiology of Disease
- Publisher
- Elsevier BV
- Published
- 2019-09
- Volume
- 129
- Pages
- 29-37
- ISSN
- 0969-9961 (ISSN-L)
- OpenAlex
- W2942386241
- Cited by
- 15
- References
- 60
- Citations by year
- 2026: 1 · 2025: 1 · 2024: 4 · 2023: 1 · 2022: 2 · 2021: 2 · 2020: 3 · 2019: 1
- FWCI
- 0.96
- Type
- journal article
- Language
- en
- Open access
- green
- doaj.org
- Licence
- Locations
- Neurobiology of Disease (publishedVersion)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- Sanofi Genzyme
- Topics
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Keywords
- Huntington's disease, astrocytes, RNA interference, adeno-associated virus vectors, YAC128 mouse model, microRNA, astrocyte dysfunction, polyglutamine repeat, neurodegeneration
- MeSH terms
- Huntingtin Protein, Dependovirus, Animals, Astrocytes, Brain, Disease Models, Animal, Genetic Vectors, Huntington Disease, Mice, Transgenic, Neurons, Phenotype, Transduction, Genetic, MicroRNAs, Mice
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