TY - JOUR
T1 - Induced pluripotent stem cells (Ipscs) and gene therapy
T2 - A new era for the treatment of neurological diseases
AU - Sguazzi, Giulia Paolini
AU - Muto, Valentina
AU - Tartaglia, Marco
AU - Bertini, Enrico
AU - Compagnucci, Claudia
N1 - Funding Information:
Funding: This research was funded by Fondazione Bambino Gesù (Vite coraggiose), Italian Ministry of Health (Ricerca Finalizzata Giovani Ricercatori 2019).
Publisher Copyright:
© 2021 by the authors. Licensee MDPI, Basel, Switzerland.
PY - 2021/12/1
Y1 - 2021/12/1
N2 - 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.
AB - 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.
KW - CRISPR‐Cas9 gene editing
KW - Gene therapy
KW - IPSCs
KW - Neurodegeneration
KW - Non‐viral vector
KW - Pediatric diseases
KW - Stem cells
KW - Viral vector
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U2 - 10.3390/ijms222413674
DO - 10.3390/ijms222413674
M3 - Review article
C2 - 34948465
AN - SCOPUS:85121359442
SN - 1661-6596
VL - 22
JO - International Journal of Molecular Sciences
JF - International Journal of Molecular Sciences
IS - 24
M1 - 13674
ER -