Gene therapy: a battle against biological barriers
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Updated: Aug 18, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
1Division of Gene Therapy Science, Graduate School of Medicine, Osaka University, Suita, Japan. kaneday@gts.med.osaka-u.ac.jp
Abstract:
One factor critical to successful human gene therapy is development of efficient gene delivery systems. Although numerous vector systems for gene transfer have been developed, a perfect vector system has not yet been constructed. Difficulties of in vivo gene transfer appear to result from resistance of living cells to invasion by foreign materials and from interference of cellular functions. We should analyze what barriers in tissues affect in vivo gene transfection and how to solve these problems for gene therapy. In this review article, the biological barriers to in vivo gene transfection are discussed and possible solutions to each barrier are discussed with respect to construction of a perfect gene therapy vector system.
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