<p><strong><em>Background: </em></strong><em>Current researches on Stem Cell Interventions (SCI) are mostly at various stages of clinical translation, and not ripe for immediate marketing. However, there is a global rise in patients opting for unproven SCI from dubious stem cell clinics.</em></p><p><strong><em>Objectives:</em></strong><em> To describes the usual stages of SCI’s clinical translation, factors fuelling the increase in unproven SCI and possible strategies for tackling the situation that have been mentioned in research articles and patient information sources. </em></p><strong><em>Conclusion: </em></strong><em>Stem cell tourism is on the rise. Direct to customer marketing of SCI through websites and social media using emotional narratives, patient testimonials and false claims of benefits, and unrealistically positive portrayal on social media are contributing factors. The situation may be addressed by treating stem cells as medical innovation and speeding up their clinical translation using modifications like expedited, conditional marketing approval. Medical societies can generate meta-analyses and updates for keeping clinicians informed about the latest therapies so they can guide patients. These along with organisations like ALSuntangled, can generate patient information booklets to help patients make informed decisions. Also, Patient Information Campaigns on social media and media’s coverage of increasing SCI related lawsuits can possibly help raise public awareness about unproven SCI. </em>
Background The most common muscular dystrophy, Duchenne muscular dystrophy (DMD), is a lethal, X‐linked disorder with no widespread cure. Worldwide, in vitro studies involving new, mutation‐specific cures and regenerative therapies are employing disease‐specific patient‐specific cells. However, these may not be completely relevant for Pakistani children because of the human genome diversities and geographic variation in mutation type and frequency. Therefore, this study aimed to generate DMD induced pluripotent stem cells (iPSCs) from the urine of Pakistani children with DMD, to serve as a precious source of differentiated cells, such as Pakistani DMD‐cardiomyocytes, for future disease‐modelling, drug testing, and gene therapy. Methods Urine‐derived cells (UDCs) isolated from mid‐stream urine underwent molecular characterization and cellular reprogramming towards iPSCs using the episomal vector system followed by molecular profiling of the iPSCs. Results Colonies of elongated and spindle‐shaped or rounded rice‐grain like UDCs were spotted 4–7 days after plating and expanded rapidly with a second passage at 2–3 weeks. Multicolor flow cytometry confirmed the expression of mesenchymal stem‐cell markers. The reprogramed iPSCs consisted of colonies of round, tightly‐packed cells with large nuclei that were positively fluorescent for the pluripotency markers octamer binding transcription factor‐4 (OCT‐4), tumour resistance antigen 1–60 (TRA‐1‐60), and stage specific embryonic 4 antigen (SSEA‐4), but not for the negative pluripotency marker SSEA‐1. To the best of our knowledge, this was the first time DMD‐iPSCs have been generated for Pakistani children. Conclusion This integration‐free, feeder‐free, efficient, and reproducible reprogramming method employed UDCs. Urine is a low‐cost, non‐invasive, painless, and repeatable source of rapidly expandable cells from children and morbid individuals for obtaining autologous cells for drug‐assays and disease‐modelling, suitable for DMD and other debilitating diseases.
Objective: This study aimed at the in vitro generation of DMD-cardiomyocytes from patient-specific induced pluripotent stem cells derived from a Pakistani patient for future work on DMD in vitro disease modeling and drug testing for efficacy and toxicity. Methods: This in vitro experimental study was carried out from December 2018 to January 2019 at Stem Cells and Regenerative Medicine Lab (SCRML) at Dow Research Institute of Biotechnology and Biomedical Sciences (DRIBBS), Dow University of Health Sciences (DUHS) Urine derived DMD-iPSCs were used which had been generated previously from a Pakistani DMD patient who had been selected through non-random purposive sampling. These were differentiated towards cardiomyocytes using Cardiomyocytes Differentiation media having specified growth factors and then the molecular characterization of the differentiated cells was done using immunofluorescence. Results: Pakistani patient’s DMD-Cardiomyocytes were generated and their identity was confirmed by positive immunofluorescence for the expression of cardiac markers NKX2-5 and TNNT-2. Conclusion: This study aimed for in vitro generation of DMD cardiomyocytes for future application in disease modeling, new drug testing for efficacy and toxicity, as well as for drug-testing for tailored personalized therapy. To the best of our knowledge, this was the first time DMD-Cardiomyocytes were generated from Pakistani DMD patients using their own induced pluripotent stem cells. doi: https://doi.org/10.12669/pjms.37.5.3104 How to cite this:Ghori FF, Wahid M. Induced pluripotent stem cells derived cardiomyocytes from Duchenne Muscular Dystrophy patients in vitro. Pak J Med Sci. 2021;37(5):---------. doi: https://doi.org/10.12669/pjms.37.5.3104 This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/3.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
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