Cardiomyocytes that are derived from human-induced pluripotent stem cells (iPSC-CM) are an exciting tool to investigate cardiomyopathy disease mechanisms at the cellular level as well as to screen for potential side effects of novel drugs. However, currently their benefit is limited due to their fairly immature differentiation status under conventional culture conditions. This review is mainly aimed at researchers outside of the iPSC-CM field and will describe potential pitfalls and which features at the level of the myofibrils would be desired to make them a more representative model system. We will also discuss different strategies that may help to achieve these.
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http://dx.doi.org/10.1007/s12551-023-01099-w | DOI Listing |
Int J Mol Sci
January 2025
Catholic High-Performance Cell Therapy Center & Department of Medical Life Science, College of Medicine, The Catholic University of Korea, Seocho-gu, Seoul 06591, Republic of Korea.
The immune-modulatory effects of mesenchymal stromal cells (MSCs) are widely used to treat inflammatory disorders, with indoleamine 2,4-dioxygenase-1 (IDO-1) playing a pivotal role in suppressing stimulated T-cell proliferation. Taking that three-dimensional (3D) cultures enhance MSCs' anti-inflammatory properties compared with two-dimensional (2D) cultures, the differentially expressed miRNAs were examined. Thus, we identified hsa-miR-4662a-5p (miR-4662a) as a key inducer of IDO-1 via its suppression of bridging integrator-1 (BIN-1), a negative regulator of the IDO-1 gene.
View Article and Find Full Text PDFInt J Mol Sci
January 2025
Division of Molecular & Regenerative Prosthodontics, Tohoku University Graduate School of Dentistry, Sendai 980-8575, Japan.
Tooth/skeletal dysplasia, such as hypophosphatasia (HPP), has been extensively studied. However, there are few definitive treatments for these diseases owing to the lack of an in vitro disease model. Cells differentiated from patient-derived induced pluripotent stem cells (iPSCs) demonstrate a pathological phenotype.
View Article and Find Full Text PDFInt J Mol Sci
January 2025
Cell Engineering Laboratory, La Paz University Hospital Health Research Institute, IdiPAZ, 28046 Madrid, Spain.
The World Health Organization estimates that approximately 285 million people suffer from visual impairments, around 5% of which are caused by corneal pathologies. Currently, the most common clinical treatment consists of a corneal transplant (keratoplasty) from a human donor. However, worldwide demand for donor corneas amply exceeds the available supply.
View Article and Find Full Text PDFInt J Mol Sci
January 2025
Laboratory of Genome Editing, Research Centre for Medical Genetics, Moskvorechye, 1, 115522 Moscow, Russia.
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the gene. Currently, CFTR modulators are the most effective treatment for CF; however, they may not be suitable for all patients. A representative and convenient model is needed to screen therapeutic agents under development.
View Article and Find Full Text PDFGenes (Basel)
January 2025
Department of Ophthalmology, Boston Children's Hospital, Boston, MA 02115, USA.
Background/objectives: Strabismus is the most common ocular disorder of childhood. Three rare, recurrent genetic duplications have been associated with both esotropia and exotropia, but the mechanisms by which they contribute to strabismus are unknown. This work aims to investigate the mechanisms of the smallest of the three, a 23 kb duplication on chromosome 4 (hg38|4:25,554,985-25,578,843).
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