ArticleMolecular and cellular neurosciences2011
Cellular fusion for gene delivery to SCA1 affected Purkinje neurons.
Article in Molecular and cellular neurosciences, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 31 citations in OpenAlex.
- Translational Relevance of SCA1 Models for the Development of Therapies for Spinocerebellar Ataxia Type 1.Biomedicines · 2025Review
- Consensus Paper: Strengths and Weaknesses of Animal Models of Spinocerebellar Ataxias and Their Clinical Implications.Cerebellum (London, England) · 2022Article
- Optimization study of plasmonic cell fusion.Scientific reports · 2022Article
- Binucleated and Multinucleated Neurons are Formed by Fusion.Bulletin of experimental biology and medicine · 2021Article
- Task Force Paper On Cerebellar Transplantation: Are We Ready to Treat Cerebellar Disorders with Cell Therapy?Cerebellum (London, England) · 2019Review
- Spinocerebellar ataxia: an update.Journal of neurology · 2019Review
- Aberrant cerebellar Purkinje cell function repaired in vivo by fusion with infiltrating bone marrow-derived cells.Acta neuropathologica · 2018Article
- Bone marrow transplantation stimulates neural repair in Friedreich's ataxia mice.Annals of neurology · 2018Article
- Purkinje cell injury, structural plasticity and fusion in patients with Friedreich's ataxia.Acta neuropathologica communications · 2016Article
- Fusion of Human Fetal Mesenchymal Stem Cells with "Degenerating" Cerebellar Neurons in Spinocerebellar Ataxia Type 1 Model Mice.PloS one · 2016Article
- Microglia-specific targeting by novel capsid-modified AAV6 vectors.Molecular therapy. Methods & clinical development · 2016Article
- Gene, Stem Cell, and Alternative Therapies for SCA 1.Frontiers in molecular neuroscience · 2016Review
- Stem cell models of polyglutamine diseases and their use in cell-based therapies.Frontiers in neuroscience · 2015Article
- Cell fusion in the brain: two cells forward, one cell back.Acta neuropathologica · 2014Review
- Autosomal dominant cerebellar ataxia type III: a review of the phenotypic and genotypic characteristics.Orphanet journal of rare diseases · 2013Review
- Mouse models of polyglutamine diseases in therapeutic approaches: review and data table. Part II.Molecular neurobiology · 2012Review
- Stem cell pathologies and neurological disease.Modern pathology : an official journal of the United States and Canadian Academy of Pathology, Inc · 2012Review
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
Cerebellar Purkinje neurons (PNs) possess a well characterized propensity to fuse with bone marrow-derived cells (BMDCs), producing heterokaryons with Purkinje cell identities. This offers the potential to rescue/repair at risk or degenerating PNs in the inherited ataxias, including Spinocerebellar Ataxia 1 (SCA1), by introducing therapeutic factors through BMDCs to potentially halt or reverse disease progression. In this study, we combined gene therapy and a stem cell-based treatment to attempt repair of at-risk PNs through cell-cell fusion in a Sca1(154Q/2Q) knock-in mouse model. BMDCs enriched for the hematopoietic stem cell (HSC) population were genetically modified using adeno-associated viral vector 7 (AAV7) to carry SCA1 modifier genes and transplanted into irradiated Sca1(154Q/2Q) mice. Binucleated Purkinje heterokaryons with sex-mismatched donor Y chromosomes were detected and successfully expressed the modifier genes in vivo. Potential effects of the new genome within Purkinje heterokaryons were evaluated using nuclear inclusions (NIs) as a biological marker to reflect possible modifications of the SCA1 disease process. An overall decrease in number of NIs and an increase in the number of surviving PNs were observed in treated Sca1(154Q/2Q). Furthermore, Bergmann glia were found to have fusogenic potential with the donor population and reveal another potential route of therapeutic entry into at-risk cells of the SCA1 cerebellum. This study presents a first step towards a proof-of-principle that combines somatic cellular fusion events with a neuroprotective gene therapy approach for providing potential neuronal protection/repair in a variety of neurodegenerative disorders.
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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.