Cardiac disease modeling using induced pluripotent stem cell-derived human cardiomyocytes.
AuthorsDell'Era P, Benzoni P, Crescini E, Valle M, Xia E, Consiglio A, Memo M,
Journal
PubMed ID25815118
Causative mutations and variants associated with cardiac diseases have been found in genes encoding cardiac ion channels, accessory proteins, cytoskeletal components, junctional proteins, and signaling molecules. In most cases the functional evaluation of the genetic alteration has been carried out by expressing the mutated proteins in in-vitro heterologous systems. While ... More
Generation of the human induced pluripotent stem cell (hiPSC) line PSMi004-A from a carrier of the KCNQ1-R594Q mutation.
AuthorsMura M, Lee YK, Pisano F, Ginevrino M, Boni M, Calabrò F, Crotti L, Valente EM, Schwartz PJ, Tse HF, Gnecchi M
JournalStem Cell Res
PubMed ID30974404
'We generated human induced pluripotent stem cells (hiPSCs) from dermal fibroblasts of a male carrier of the heterozygous mutation c.1781?G?>?A p.R594Q on the KCNQ1 gene. hiPSCs, generated using four retroviruses each encoding for OCT4, SOX2, KLF4 and cMYC, display pluripotent stem cell characteristics, and can be differentiated into spontaneously beating ... More
Reprogramming of Urine-Derived Renal Epithelial Cells into iPSCs Using srRNA and Consecutive Differentiation into Beating Cardiomyocytes.
AuthorsSteinle H, Weber M, Behring A, Mau-Holzmann U, von Ohle C, Popov AF, Schlensak C, Wendel HP, Avci-Adali M
JournalMol Ther Nucleic Acids
PubMed ID31476669
'The generation of induced pluripotent stem cells (iPSCs) from patient''s somatic cells and the subsequent differentiation into desired cell types opens up numerous possibilities in regenerative medicine and tissue engineering. Adult cardiomyocytes have limited self-renewal capacity; thus, the efficient, safe, and clinically applicable generation of autologous cardiomyocytes is of great ... More
Generation of the human induced pluripotent stem cell (hiPSC) line PSMi007-A from a Long QT Syndrome type 1 patient carrier of two common variants in the NOS1AP gene.
AuthorsMura M, Pisano F, Stefanello M, Ginevrino M, Boni M, Calabrò F, Crotti L, Valente EM, Schwartz PJ, Brink PA, Gnecchi M
JournalStem Cell Res
PubMed ID30878014
We generated human induced pluripotent stem cells (hiPSCs) from a symptomatic Long QT Syndrome (LQTS) type 1 patient, belonging to a South African (SA) founder population segregating the heterozygous mutation c.1022C?>?T p.A341V on the KCNQ1 gene. The patient is also homozygous for the two minor variants rs4657139 and rs16847548 on ... More
Development of a model of ischemic heart disease using cardiomyocytes differentiated from human induced pluripotent stem cells.
AuthorsWei H, Wang C, Guo R, Takahashi K, Naruse K
JournalBiochem Biophys Res Commun
PubMed ID31623826
Ischemic heart disease remains the largest cause of death worldwide. Accordingly, many researchers have sought curative options, often using laboratory animal models such as rodents. However, the physiology of the human heart differs significantly from that of the rodent heart. In this study, we developed a model of ischemic heart ... More
Development of a bio-MEMS device for electrical and mechanical conditioning and characterization of cell sheets for myocardial repair.
AuthorsRoberts EG, Kleptsyn VF, Roberts GD, Mossburg KJ, Feng B, Domian IJ, Emani SM, Wong JY
JournalBiotechnol Bioeng
PubMed ID31317531
Here we propose a bio-MEMS device designed to evaluate contractile force and conduction velocity of cell sheets in response to mechanical and electrical stimulation of the cell source as it grows to form a cellular sheet. Moreover, the design allows for the incorporation of patient-specific data and cell sources. An ... More
Generation of human iPS cell line CBTCi001-A from dermal fibroblasts obtained from a healthy donor.
AuthorsMartins GLS, Paredes BD, Sampaio GLA, Nonaka CKV, da Silva KN, Allahdadi KJ, França LSA, Soares MBP, Dos Santos RR, Souza BSF
JournalStem Cell Res
PubMed ID31706097
Human-induced pluripotent stem cell (hiPSC) CBTCi001-A line was generated from a healthy 30-year old male dermal fibroblasts using non-integrative reprogramming method using episomal-based plasmids expressing OCT4, SOX2, KLF4, and MYCL. Characterization of CBTCi001-A was confirmed by the expression of typical markers of pluripotency and differentiation potential in vitro. ... More
Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models.
AuthorsBarthélémy F, Wang RT, Hsu C, Douine ED, Marcantonio EE, Nelson SF, Miceli MC
JournalMol Ther Nucleic Acids
PubMed ID31678734
Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models. In humans with Duchenne muscular dystrophy treated with AOs, low levels of dystrophin have been induced, and modest slowing of disease progression has ... More
Generation of the human induced pluripotent stem cell (hiPSC) line PSMi005-A from a patient carrying the KCNQ1-R190W mutation.
AuthorsMura M, Lee YK, Pisano F, Ginevrino M, Boni M, Calabrò F, Crotti L, Valente EM, Schwartz PJ, Tse HF, Gnecchi M
JournalStem Cell Res
PubMed ID31009818
We generated human induced pluripotent stem cells (hiPSCs) from dermal fibroblasts of a woman carrier of the heterozygous mutation c.568C?>?T p.R190W on the KCNQ1 gene. hiPSCs, obtained using four retroviruses enconding the reprogramming factors OCT4, SOX2, cMYC and KLF4, display pluripotent stem cell characteristics, and can be differentiated into spontaneously ... More
RNA-Based Strategies for Cardiac Reprogramming of Human Mesenchymal Stromal Cells.
AuthorsMueller P, Wolfien M, Ekat K, Lang CI, Koczan D, Wolkenhauer O, Hahn O, Peters K, Lang H, David R, Lemcke H
JournalCells
PubMed ID32098400
Multipotent adult mesenchymal stromal cells (MSCs) could represent an elegant source for the generation of patient-specific cardiomyocytes needed for regenerative medicine, cardiovascular research, and pharmacological studies. However, the differentiation of adult MSC into a cardiac lineage is challenging compared to embryonic stem cells or induced pluripotent stem cells. Here we ... More