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Updated: Mar 29, 2026

Stable and Efficient Genetic Modification of Cells in the Adult Mouse V-SVZ for the Analysis of Neural Stem Cell Autonomous and Non-autonomous Effects

Stable and Efficient Genetic Modification of Cells in the Adult Mouse V-SVZ for the Analysis of Neural Stem Cell Autonomous and Non-autonomous Effects

Published on: February 17, 2016

A novel intranuclear RNA vector system for long-term stem cell modification.

Y Ikeda¹, A Makino^2,3, W E Matchett¹

¹Department of Molecular Medicine, Mayo Clinic, Rochester, MN, USA.

|December 4, 2015

Summary

This summary is machine-generated.

Researchers developed a novel RNA-based vector using Borna Disease Virus (BDV) for safe, long-term gene expression in stem cells. This genomic modification-free system overcomes biosafety concerns for regenerative medicine applications.

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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

Area of Science:

Regenerative Medicine
Gene Therapy
Virology

Background:

Genetically modified stem cells offer therapeutic potential for genetic and degenerative diseases.
Biosafety concerns, particularly insertional mutagenesis from viral vectors, hinder clinical application.
Existing gene delivery methods often lack long-term efficacy or pose safety risks.

Purpose of the Study:

To develop a novel, safe, and effective gene delivery system for sustained transgene expression in stem cells.
To utilize an RNA-based episomal vector for genomic modification-free gene transfer.
To assess the long-term efficacy and biosafety of Borna Disease Virus (BDV)-based vectors in stem cells.

Main Methods:

Development of an RNA-based episomal vector system using the intranuclear RNA virus, Borna Disease Virus (BDV).
Transduction of mesenchymal stem cells (MSCs) and human induced pluripotent stem cells (hiPSCs) with BDV-based vectors.
Evaluation of transgene expression duration, cellular morphology, stem cell marker expression (CD105), adipogenicity, and differentiation potential.

Main Results:

BDV-based vectors achieved long-term transgene expression in MSCs without altering cell morphology, CD105 expression, or adipogenic potential.
Replication-defective BDV vectors enabled sustained transduction of hiPSCs.
hiPSCs transduced with BDV vectors retained their ability to differentiate into the three embryonic germ layers.
The BDV system demonstrated genomic modification-free, episomal RNA delivery for stem cell transduction.

Conclusions:

Borna Disease Virus (BDV)-based vectors provide a promising platform for sustained stem cell transduction.
This RNA-based episomal vector system offers a safer alternative to integrating viral vectors, addressing key biosafety concerns.
The BDV system holds potential for advancing regenerative medicine by enabling reliable and safe genetic modification of stem cells.