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JoVE Journal Genetics
Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
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Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Article DOI: 10.3791/59241-v • 13:47 min • March 29th, 2019
March 29th, 2019 •
1,2, 3, 3, 1, 1,2, 4, 4, 1,2, 3
1Department of Neurology, Duke University Medical Center, 2Center for Genomic and Computational Biology, Duke University Medical Center, 3Viral Vector Core, Duke University Medical Center, 4Division of Gynecologic Oncology, Department of Obstetrics and Gynecology, Duke University Medical Center

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Summary
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Targeted DNA epigenome editing represents a powerful therapeutic approach. This protocol describes the production, purification, and concentration of all-in-one lentiviral vectors harboring the CRISPR-dCas9-DNMT3A transgene for epigenome-editing applications in human induced pluripotent stem cell (hiPSC)-derived neurons.

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Lentiviral Vector Epigenome-editing Tools Human Induced Pluripotent Stem Cells Disease Models CRISPR/Cas Tools Alpha-synuclein Expression Gene Expression Guide RNA Screening Validation Animal Models Rodent Nonhuman Primate Clinical Studies Molecular Cloning Procedure Lentiviral Vector Plasmids Lentiviral Vector Production
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