Genetically engineered hypoimmunogenic cell therapy

A Hotta, S Schrepfer, A Nagy - Nature Reviews Bioengineering, 2024 - nature.com
Immune rejection is an important obstacle to the use of allogeneic 'off-the-shelf'cells for
transplantation into immunocompetent patients. Genetic modification has emerged as a …

Engineering considerations of iPSC-based personalized medicine

S Park, Y Gwon, SA Khan, KJ Jang, J Kim - Biomaterials Research, 2023 - spj.science.org
Personalized medicine aims to provide tailored medical treatment that considers the clinical,
genetic, and environmental characteristics of patients. iPSCs have attracted considerable …

A reference human induced pluripotent stem cell line for large-scale collaborative studies

CB Pantazis, A Yang, E Lara, JA McDonough… - Cell stem cell, 2022 - cell.com
Human induced pluripotent stem cell (iPSC) lines are a powerful tool for studying
development and disease, but the considerable phenotypic variation between lines makes it …

Flexible 3D printed microwires and 3D microelectrodes for heart-on-a-chip engineering

Q Wu, P Zhang, G O'Leary, Y Zhao, Y Xu… - …, 2023 - iopscience.iop.org
We developed a heart-on-a-chip platform that integrates highly flexible, vertical, 3D
micropillar electrodes for electrophysiological recording and elastic microwires for the …

[HTML][HTML] Progress of organoid platform in cardiovascular research

X Du, H Jia, Y Chang, Y Zhao, J Song - Bioactive Materials, 2024 - Elsevier
Cardiovascular disease is a significant cause of death in humans. Various models are
necessary for the study of cardiovascular diseases, but once cellular and animal models …

Whole genome sequencing delineates regulatory, copy number, and cryptic splice variants in early onset cardiomyopathy

R Lesurf, A Said, O Akinrinade, J Breckpot… - NPJ genomic …, 2022 - nature.com
Cardiomyopathy (CMP) is a heritable disorder. Over 50% of cases are gene-elusive on
clinical gene panel testing. The contribution of variants in non-coding DNA elements that …

A Fixable Fluorescence‐Quenched Substrate for Quantitation of Lysosomal Glucocerebrosidase Activity in Both Live and Fixed Cells

S Zhu, MC Deen, Y Zhu, PA Gilormini… - Angewandte …, 2023 - Wiley Online Library
Fluorogenic substrates are emerging tools that enable studying enzymatic processes within
their native cellular environments. However, fluorogenic substrates that function within live …

A versatile fluorescence-quenched substrate for quantitative measurement of glucocerebrosidase activity within live cells

MC Deen, Y Zhu, C Gros, N Na, PA Gilormini… - Proceedings of the …, 2022 - pnas.org
Loss of activity of the lysosomal glycosidase β-glucocerebrosidase (GCase) causes the
lysosomal storage disease Gaucher disease (GD) and has emerged as the greatest genetic …

Machine learning identifies clinical and genetic factors associated with anthracycline cardiotoxicity in pediatric cancer survivors

MA Chaix, N Parmar, C Kinnear, M Lafreniere-Roula… - Cardio Oncology, 2020 - jacc.org
Background Despite known clinical risk factors, predicting anthracycline cardiotoxicity
remains challenging. Objectives This study sought to develop a clinical and genetic risk …

Myosin inhibitor reverses hypertrophic cardiomyopathy in genotypically diverse pediatric iPSC-cardiomyocytes to mirror variant correction

C Kinnear, A Said, G Meng, Y Zhao, EY Wang… - Cell Reports …, 2024 - cell.com
Pathogenic variants in MYH7 and MYBPC3 account for the majority of hypertrophic
cardiomyopathy (HCM). Targeted drugs like myosin ATPase inhibitors have not been …