Dual Transgene Expression in Murine Cerebellar Purkinje Neurons by Viral Transduction In Vivo
Dual Transgene Expression in Murine Cerebellar Purkinje Neurons by Viral Transduction In Vivo
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.
- University of Mary United States
- WASHINGTON UNIVERSITY
- Washington University in St. Louis United States
- Washington University in St. Louis School of Medicine United States
- Washington University in St. Louis United States
Science, Genetic Vectors, Gene Expression, Mice, Purkinje Cells, Cricetulus, Transduction, Genetic, Cerebellum, Cricetinae, Animals, Humans, Transgenes, Cells, Cultured, Mice, Knockout, Q, Lentivirus, R, Genetic Therapy, Dependovirus, Mice, Inbred C57BL, HEK293 Cells, Medicine, Research Article
Science, Genetic Vectors, Gene Expression, Mice, Purkinje Cells, Cricetulus, Transduction, Genetic, Cerebellum, Cricetinae, Animals, Humans, Transgenes, Cells, Cultured, Mice, Knockout, Q, Lentivirus, R, Genetic Therapy, Dependovirus, Mice, Inbred C57BL, HEK293 Cells, Medicine, Research Article
4 Research products, page 1 of 1
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
- 2017IsRelatedTo
citations This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).16 popularity This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.Top 10% influence This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).Average impulse This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.Top 10%
