Genetic engineering of stem cells for enhanced therapy

Adam Nowakowski, Anna Andrzejewska, Miroslaw Janowski, Piotr Walczak, Barbara Lukomska

Research output: Contribution to journalReview articlepeer-review

Abstract

Stem cell therapy is a promising strategy for overcoming the limitations of current treatment methods. The modification of stem cell properties may be necessary to fully exploit their potential. Genetic engineering, with an abundance of methodology to induce gene expression in a precise and well-controllable manner, is particularly attractive for this purpose. There are virus-based and non-viral methods of genetic manipulation. Genome-integrating viral vectors are usually characterized by highly efficient and long-term transgene expression, at a cost of safety. Non-integrating viruses are also highly efficient in transduction, and, while safer, offer only a limited duration of transgene expression. There is a great diversity of transfectable forms of nucleic acids; however, for efficient shuttling across cell membranes, additional manipulation is required. Both physical and chemical methods have been employed for this purpose. Stem cell engineering for clinical applications is still in its infancy and requires further research. There are two main strategies for inducing transgene expression in therapeutic cells: transient and permanent expression. In many cases, including stem cell trafficking and using cell therapy for the treatment of rapid-onset disease with a short healing process, transient transgene expression may be a sufficient and optimal approach. For that purpose, mRNA-based methods seem ideally suited, as they are characterized by a rapid, highly efficient transfection, with outstanding safety. Permanent transgene expression is primarily based on the application of viral vectors, and, due to safety concerns, these methods are more challenging. There is active, ongoing research toward the development of non-viral methods that would induce permanent expression, such as transposons and mammalian artificial chromosomes.

Original languageEnglish (US)
Pages (from-to)1-18
Number of pages18
JournalActa Neurobiologiae Experimentalis
Volume73
Issue number1
StatePublished - Jun 5 2013

Keywords

  • Gene therapy
  • Physical- and chemical-based methods
  • Stem cells
  • Transfection
  • Viral methods

ASJC Scopus subject areas

  • Neuroscience(all)

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