Transforming the Promise of Pluripotent Stem Cell-Derived Cardiomyocytes to a Therapy: Challenges and Solutions for Clinical Trials

Prowse, Andrew; Timmins, Nicholas; Yau, Terrence M.; Li, Ren‐Ke; Weisel, Richard D.; Keller, Gordon; Zandstra, Peter W.

doi:10.1016/j.cjca.2014.08.005

Cited by 27 publications

(16 citation statements)

References 115 publications

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“…Despite the still incomplete understanding of all mechanisms, several groups have independently demonstrated that stem cells can exert a beneficial effect on cardiac function (53,17,16,56,23). However, it is increasingly evident that the role of adult stem cells in cardiac tissue repair is related, at least in part, to the release of a variety of paracrine factors (22,67,14).…”

Section: Figure 1 Biogenesis Of Mirnas and Their Sorting Into Exosomesmentioning

confidence: 99%

Exosomal miRNAs in Heart Disease

et al. 2016

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Micro-RNAs (miRNAs) are small noncoding RNAs involved in the posttranscriptional regulation of gene expression. Exosomes have recently emerged as novel elements of intercellular communication in the cardiovascular system. Exosomal miRNAs could be key players in intercellular cross-talk, particularly during different diseases such as myocardial infarction (MI) and heart failure (HF). This review addresses the functional role played by exosomal miRNAs in heart disease and their potential use as new biomarkers.

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Section: Figure 1 Biogenesis Of Mirnas and Their Sorting Into Exosomesmentioning

confidence: 99%

Exosomal miRNAs in Heart Disease

et al. 2016

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“…The use of 'deleteriousness'-weighted mutational counts can improve the power of burden-of-mutation studies, further highlighting the importance of better technology for identifying deleterious variants. Application of clustered regularly interspaced short palindromic repeat (CRISPR) technology has enabled (for better or for worse) (Baltimore et al 2015;Bosley et al 2015) germline editing as well as regenerative medicine based on ex vivo engineering of induced pluripotent stem cells (iPSCs) and reintroduction of differentiated cells; however, the functionality of off-target and other passenger mutations still needs to be rigorously examined for safety (Prowse et al 2014;Baltimore et al 2015).…”

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confidence: 99%

An extended set of yeast-based functional assays accurately identifies human disease mutations

et al. 2016

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We can now routinely identify coding variants within individual human genomes. A pressing challenge is to determine which variants disrupt the function of disease-associated genes. Both experimental and computational methods exist to predict pathogenicity of human genetic variation. However, a systematic performance comparison between them has been lacking. Therefore, we developed and exploited a panel of 26 yeast-based functional complementation assays to measure the impact of 179 variants (101 disease-and 78 non-disease-associated variants) from 22 human disease genes. Using the resulting reference standard, we show that experimental functional assays in a 1-billion-year diverged model organism can identify pathogenic alleles with significantly higher precision and specificity than current computational methods.

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“…In a recent proof-of-principle clinical trial, single-cell, hESC-derived CPCs were embedded in a fibrin gel and implanted onto the epicardium of patients with severe heart failure by positioning the cell-fibrin mixture beneath the pericardial flap [56]. There are also ongoing efforts to gain approval for hPSC-CM clinical trials [16, 57]. …”

Section: Cell Injectionmentioning

confidence: 99%