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Delivery Technologies for Stem Cells Based Gene Therapy

Call for Papers

Gene therapy (GT) is a relatively new approach, broadly accepted by scientist and clinicians, with a great potential to treat a genetic diseases. In general terms, the GT concept is based on the modification of the genome, either in vivo or in vitro, in order to restore a damaged essential gene or to overthrow the function of a disease gene. On the other hand, considering the regenerative ability of the human stem cells, the combination of both approaches, gene modification (Edition and Addition) and stem cells, shows several advantages over conventional cells therapy. Several types of stem cells were used in combination with gene modification approaches; for instance, the hematopoietic stem cell (HSC) based gene therapy holds promise for the cure of many inherited and acquired diseases. Other reports have identified the great potential of the neural stem cells gene modification (NSCs) and mesenchymal stem cells. Although a number of phase I/II gene therapy clinical trials have reported remarkable rate of efficacy and safety, the development of successful therapies will depend on better optimization and characterization of vector used for the gene transfer. Several technical limitations should be solved before a general clinical use of this approach. First, the therapeutic genetic material must overcome all the cellular and tissue barriers in order to reach the target cells and deliver the genetic material. Second, the manipulated stem cells should maintain their properties and express the transgene for the necessary time frame to achieve therapeutic benefit.

The principal aim of this special issue is to discuss the recent development of nonviral and viral gene delivery technology platforms, including the delivery of therapeutic genetic material and genome editing tools, but mainly focused in stem cells. In this respect, a number of requirements for gene transfer to stem cells determine the choice of the appropriate vector or gene transfer vehicle.

We encourage the submission of original or review articles focused on improving efficacy and safety of gene therapy delivery platforms for in vivo and in vitro applications using stem cells as target cells.

Potential topics include but are not limited to the following:

  • Genome-editing tools in stem Cells
  • Safety of stem cells gene editing
  • Ethical issue in stem cell genome editing
  • Genome editing for basic research in stem cells
  • Genome editing for modeling disease with stem cells
  • Stem cells gene editing for novel therapeutics

Authors can submit their manuscripts through the Manuscript Tracking System at

Submission DeadlineFriday, 24 August 2018
Publication DateJanuary 2019

Papers are published upon acceptance, regardless of the Special Issue publication date.

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