ArticlePloS one2014
Dual transgene expression in murine cerebellar Purkinje neurons by viral transduction in vivo.
Article in PloS one, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 17 citations in OpenAlex.
- Cas9 editing ofMolecular therapy. Nucleic acids · 2024Article
- Comparative Analysis of Six Adeno-Associated Viral Vector Serotypes in Mouse Inferior Colliculus and Cerebellum.eNeuro · 2024Article
- Comparative analysis of six adeno-associated viral vector serotypes in mouse inferior colliculus and cerebellum.bioRxiv : the preprint server for biology · 2024Article
- Selective transduction of cerebellar Purkinje and granule neurons using delivery of AAV-PHP.eB and AAVrh10 vectors at axonal terminal locations.Frontiers in molecular neuroscience · 2022Article
- Transfer ofMolecular therapy. Nucleic acids · 2021Article
- Rapid and Sparse Labeling of Neurons Based on the Mutant Virus-Like Particle of Semliki Forest Virus.Neuroscience bulletin · 2019Article
- Tailored transgene expression to specific cell types in the central nervous system after peripheral injection with AAV9.Molecular therapy. Methods & clinical development · 2016Article
- Improved Methods for Fluorescence Microscopy Detection of Macromolecules at the Axon Initial Segment.Frontiers in cellular neuroscience · 2016Article
- Progresses towards safe and efficient gene therapy vectors.Oncotarget · 2015Review
- Reversal of Aging-Related Neuronal Ca2+ Dysregulation and Cognitive Impairment by Delivery of a Transgene Encoding FK506-Binding Protein 12.6/1b to the Hippocampus.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2015Article
- Intracellular FGF14 (iFGF14) Is Required for Spontaneous and Evoked Firing in Cerebellar Purkinje Neurons and for Motor Coordination and Balance.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2015Article
- The production of viral vectors designed to express large and difficult to express transgenes within neurons.Molecular brain · 2015Article
- Internal ribosome entry site-based vectors for combined gene therapy.World journal of experimental medicine · 2015Review
- Copackaging of multiple adeno-associated viral vectors in a single production step.Human gene therapy methods · 2014Article
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Viral-vector mediated gene transfer to cerebellar Purkinje neurons in vivo is a promising avenue for gene therapy of cerebellar ataxias and for genetic manipulation in functional studies of animal models of cerebellar disease. Here, we report the results of experiments designed to identify efficient methods for viral transduction of adult murine Purkinje neurons in vivo. For these analyses, several lentiviral and an adeno-associated virus (AAV), serotype 1, vector with various promoter combinations were generated and compared for in situ transduction efficiency, assayed by fluorescent reporter protein expression in Purkinje neurons. Additional experiments were also conducted to identify the optimal experimental strategy for co-expression of two proteins in individual Purkinje neurons. Of the viruses tested, AAV1 with a CAG promoter exhibited the highest specificity for Purkinje neurons. To deliver two proteins to the same Purkinje neuron, several methods were tested, including: an internal ribosome entry site (IRES), a 2A sequence, a dual promoter vector, and co-injection of two viruses. Efficient expression of both proteins in the same Purkinje neuron was only achieved by co-injecting two AAV1-CAG viruses. We found that use of an AAV1-CAG virus outperformed similar lentivirus vectors and that co-injection of two AAV1-CAG viruses could be used to efficiently deliver two proteins to the same Purkinje neuron in adult mice. AAV1 with a CAG promoter is highly efficient and selective at transducing adult cerebellar Purkinje neurons and two AAV-CAG viruses can be used to efficiently express two proteins in the same neuron in vivo.
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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.