Abstract
Induced pluripotent stem cells (iPSCs) are defined as reprogrammed somatic cells exhibiting embryonic stem cell characteristics. Since their discovery in 2006, efforts have been made to utilize iPSCs in clinical settings. One of the promising fields of medicine, in which genetically patient-specific stem cells may prove themselves useful, is gene therapy. iPSCs technology holds potential in both creating models of genetic diseases and delivering therapeutic agents into the organism via auto-transplants, which reduces the risk of rejection compared to allotransplants. However, in order to safely administer genetically corrected stem cells into patients’ tissues, efforts must be made to establish stably pluripotent stem cells and reduce the risk of insertional tumorigenesis. In order to achieve this, optimal reprogramming factors and vectors must be considered. Therefore, in this review, the molecular bases of reprogramming safe iPSCs for clinical applications and recent attempts to translate iPSCs technology into the clinical setting are discussed.
Highlights
Since the advent of the term ‘stem cells’ in the 19th century [1], the idea of singlecell capacity to differentiate into any type of tissue has been appealing to both biologists and clinicians
Some SCID-causing mutations affect non-homologous end-joining (NHEJ), a DNA repair process, which is essential in T- and B-cell receptor recombination [65], providing TCR and BCR diversity needed for correct lymphocyte function
To improve the therapeutic effects of osteogenesis imperfecta (OI) and other genetic diseases of connective tissue caused by mutations in collagen, further investigations are needed on experimental approaches, in particular based on stem cell transplantation and genetic engineering, which at present constitute an interesting and well-developed niche of studies [34,75,76]
Summary
Since the advent of the term ‘stem cells’ in the 19th century [1], the idea of singlecell capacity to differentiate into any type of tissue has been appealing to both biologists and clinicians. Biomolecules 2021, 11, 699 work on reprogramming mouse embryonic fibroblasts into a new type of cell, called induced pluripotent stem (iPSCs) cells. The iPSCs exhibited morphology, growth properties, and cell marker gene expression of embryonic stem cells [7]. This procedure makes it properties, cellgenetically marker gene expression ofstem embryonic stem cellstypes [7] This procedure possible toand gather patient-specific cells from many of obtainmakes it possible gather genetically patient-specific cells from types of able somatic cellsto[8]. Review, the molecular bases of cellespecially reprogramming and precise medical techniques In this the molecular bases of cell reprogramming possible applications of iPSCs in review, this developing branch of medicine, namely gene and therpossible apy, areapplications discussed. In this the molecular bases of cell reprogramming possible applications of iPSCs in review, this developing branch of medicine, namely gene and therpossible apy, areapplications discussed. of iPSCs in this developing branch of medicine, namely gene therapy, are discussed
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