Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2021 Dec 20;22(24):13674.
doi: 10.3390/ijms222413674.

Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases

Giulia Paolini Sguazzi  1 , Valentina Muto  1 , Marco Tartaglia  1 , Enrico Bertini  1 , Claudia Compagnucci  1
Affiliations
Review

Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases

Giulia Paolini Sguazzi et al. Int J Mol Sci. .

Abstract

To date, gene therapy has employed viral vectors to deliver therapeutic genes. However, recent progress in molecular and cell biology has revolutionized the field of stem cells and gene therapy. A few years ago, clinical trials started using stem cell replacement therapy, and the induced pluripotent stem cells (iPSCs) technology combined with CRISPR-Cas9 gene editing has launched a new era in gene therapy for the treatment of neurological disorders. Here, we summarize the latest findings in this research field and discuss their clinical applications, emphasizing the relevance of recent studies in the development of innovative stem cell and gene editing therapeutic approaches. Even though tumorigenicity and immunogenicity are existing hurdles, we report how recent progress has tackled them, making engineered stem cell transplantation therapy a realistic option.

Keywords: CRISPR-Cas9 gene editing; gene therapy; iPSCs; neurodegeneration; non-viral vector; pediatric diseases; stem cells; viral vector.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Schematic drawing of the workflow allowing the use of genetically modified iPSCs for transplantation therapy. Starting from the patients, somatic cells can be reprogrammed into iPSCs that are then corrected or modified with CRISPR/Cas9 gene editing with the aim to modify the MHC complex to make them compatible for a wider population of patients. The engineered stem cells can be used for autologous and/or for allogeneic transplantation and used as therapy for monogenic disorders.
See this image and copyright information in PMC

References

    1. Takahashi K., Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006;126:663–676. doi: 10.1016/j.cell.2006.07.024. - DOI - PubMed
    1. Takahashi K., Tanabe K., Ohnuki M., Narita M., Ichisaka T., Tomoda K., Yamanaka S. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 2007;131:861–872. doi: 10.1016/j.cell.2007.11.019. - DOI - PubMed
    1. Compagnucci C., Bertini E. The Potential of iPSCs for the Treatment of Premature Aging Disorders. Int. J. Mol. Sci. 2017;18:2350. doi: 10.3390/ijms18112350. - DOI - PMC - PubMed
    1. Deverman B.E., Ravina B.M., Bankiewicz K.S., Paul S.M., Sah D.W.Y. Gene therapy for neurological disorders, progress and prospects. Nat. Rev. Drug Discov. 2018;17:641–659. doi: 10.1038/nrd.2018.110. - DOI - PubMed
    1. Karpe Y., Chen Z., Li X.J. Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases. Pharmaceuticals. 2021;14:565. doi: 10.3390/ph14060565. - DOI - PMC - PubMed

MeSH terms