Stem Cells: The Game Changers of Human Cardiac Disease Modelling and Regenerative Medicine

Abstract

A comprehensive understanding of the molecular basis and mechanisms underlying cardiac diseases is mandatory for the development of new and effective therapeutic strategies. The lack of appropriate in vitro cell models that faithfully mirror the human disease phenotypes has hampered the understanding of molecular insights responsible of heart injury and disease development. Over the past decade, important scientific advances have revolutionized the field of stem cell biology through the remarkable discovery of reprogramming somatic cells into induced pluripotent stem cells (iPSCs). These advances allowed to achieve the long-standing ambition of modelling human disease in a dish and, more interestingly, paved the way for unprecedented opportunities to translate bench discoveries into new therapies and to come closer to a real and effective stem cell-based medicine. The possibility to generate patient-specific iPSCs, together with the new advances in stem cell differentiation procedures and the availability of novel gene editing approaches and tissue engineering, has proven to be a powerful combination for the generation of phenotypically complex, pluripotent stem cell-based cellular disease models with potential use for early diagnosis, drug screening, and personalized therapy. This review will focus on recent progress and future outcome of iPSCs technology toward a customized medicine and new therapeutic options.

Insights into the Genetic Profile of Two Siblings Affected by Unverricht-Lundborg Disease Using Patient-Derived hiPSCs

“…In this study, to characterize the genetic mutation of the disease, we reprogrammed fibroblasts and T lymphocytes from two Italian siblings suffering from Unverricht-Lundborg disease, ULD1 and ULD2, to generate induced pluripotent stem cells (iPSCs). Patient-specific iPSCs provide an unlimited source of disease-relevant cells and represent a robust cellular platform for a plethora of biological applications, including in vitro model disease, drug screening, and testing, as well as holding the potential for clinical applications [ 55 , 56 , 57 , 58 , 59 ]. The vast majority of genetic disorders have different effects in different people and this holds true even when comparing the siblings examined in this study as they show different degrees of disease severity: ULD1 has a severe phenotype, while ULD2 has a mild form.…”

Uncovering the Metabolic and Stress Responses of Human Embryonic Stem Cells to FTH1 Gene Silencing

“…In addition to these properties, PSCs also have the ability to give rise to almost any cell type, both in vivo and in vitro. On the basis of these key features, PSCs technology holds enormous potential in many fields of biomedical research, including drug screening, development and repurposing [62][63][64][65], disease modelling [66][67][68][69][70], cell therapy [71], aging [72], and regenerative medicine [73]. Although significant progress has been made, many hurdles still need to be overcome to push PSCs technology towards the road to clinical practice.…”

A Comprehensive Review of the Therapeutic Value of Urine-Derived Stem Cells

“…Reprogrammed iPSCs from USCs are affected by genomic instability and epigenetic memory associated with the reprogramming process, resulting in aberrant differentiation in vitro and even influencing disease modelling and clinical applications ( Ji et al, 2017b ). Teratoma formation is a major side effect of iPSC transplantation ( Parrotta et al, 2019 ).…”