CRISPR-Cas tools and their application in genetic engineering of human stem cells and organoids

D Hendriks, H Clevers, B Artegiani - Cell stem cell, 2020 - cell.com
CRISPR-Cas technology has revolutionized biological research and holds great therapeutic
potential. Here, we review CRISPR-Cas systems and their latest developments with an …

Recent advances in CRISPR-based genome editing technology and its applications in cardiovascular research

ZH Li, J Wang, JP Xu, J Wang, X Yang - Military Medical Research, 2023 - Springer
The rapid development of genome editing technology has brought major breakthroughs in
the fields of life science and medicine. In recent years, the clustered regularly interspaced …

Serine biosynthesis as a novel therapeutic target for dilated cardiomyopathy

I Perea-Gil, T Seeger, AAN Bruyneel… - European Heart …, 2022 - academic.oup.com
Aims Genetic dilated cardiomyopathy (DCM) is a leading cause of heart failure. Despite
significant progress in understanding the genetic aetiologies of DCM, the molecular …

Unfolded protein response as a compensatory mechanism and potential therapeutic target in PLN R14del cardiomyopathy

DAM Feyen, I Perea-Gil, RGC Maas, M Harakalova… - Circulation, 2021 - Am Heart Assoc
Background: Phospholamban (PLN) is a critical regulator of calcium cycling and contractility
in the heart. The loss of arginine at position 14 in PLN (R14del) is associated with dilated …

KLF13 loss‐of‐function mutations underlying familial dilated cardiomyopathy

YH Guo, J Wang, XJ Guo, RF Gao… - Journal of the …, 2022 - Am Heart Assoc
Background Dilated cardiomyopathy (DCM), characterized by progressive left ventricular
enlargement and systolic dysfunction, is the most common type of cardiomyopathy and a …

CRISPRi/a screens in human iPSC-cardiomyocytes identify glycolytic activation as a druggable target for doxorubicin-induced cardiotoxicity

C Liu, M Shen, Y Liu, A Manhas, SR Zhao, M Zhang… - Cell Stem Cell, 2024 - cell.com
Doxorubicin is limited in its therapeutic utility due to its life-threatening cardiovascular side
effects. Here, we present an integrated drug discovery pipeline combining human induced …

CRISPR/CAS9: a promising approach for the research and treatment of cardiovascular diseases

M Dong, J Liu, C Liu, H Wang, W Sun, B Liu - Pharmacological Research, 2022 - Elsevier
The development of gene-editing technology has been one of the biggest advances in
biomedicine over the past two decades. Not only can it be used as a research tool to build a …

Characterization of a novel zebrafish model of SPEG-related centronuclear myopathy

KG Espinosa, S Geissah, L Groom… - Disease Models & …, 2022 - journals.biologists.com
Centronuclear myopathy (CNM) is a congenital neuromuscular disorder caused by
pathogenic variation in genes associated with membrane trafficking and excitation …

Homozygous SPEG Mutation Is Associated With Isolated Dilated Cardiomyopathy

M Almannai, S Luo, E Faqeih, F Al Mutairi… - Circulation: Genomic …, 2021 - Am Heart Assoc
SPEG (Striated Preferentially Expressed Pro-tein Kinase), a member of the myosin light
chain kinase family, is critical for cardiac and skeletal muscle function involved in excitation …

Genetic cardiomyopathies: the lesson learned from hiPSCs

I My, E Di Pasquale - Journal of Clinical Medicine, 2021 - mdpi.com
Genetic cardiomyopathies represent a wide spectrum of inherited diseases and constitute
an important cause of morbidity and mortality among young people, which can manifest with …