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A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells
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A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells

Published on: December 12, 2017

Novel cell-specific and pH-sensitive non-viral gene carrier system. Preface

Xuesi Chen1, Tae Gwan Park, Atsushi Maruyama

  • 1Changchun Institute of Applied Chemistry, Chinese Academy of Sciences, China. xschen@ciac.jl.cn

Journal of Controlled Release : Official Journal of the Controlled Release Society
|September 6, 2011
PubMed
Summary

No abstract available in PubMed .

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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