The mechanisms and current uses of gene upregulation strategies are reviewed and their advantages and limitations are critically evaluated as therapies for inherited cardiomyopathies.
Chronic lung diseases arise from complex interactions between environmental exposures and host genetic factors. However, these interactions are not fully understood, and despite decades of study, effective curative therapies remain unavailable for many patients. Recent breakthroughs in therapeutic gene-editing have bec...
A. Duffy, Anja Schweikert, Sze-Ying Tan et al.· Biomedicines· 0 citations
A multidimensional framework is proposed in which gene therapy platforms are evaluated according to three intrinsic properties—genetic precision, temporal control, and dosage tunability—while delivery, clinical maturity, and disease context act as major translational constraints.
Jared Wieland, Peyton Jackson, William Penrod et al.· Cells· 0 citations
A generalizable framework to modulate gene dosage by alternative splicing via genome editing is presented, allowing high-fidelity animal models for human diseases caused by haploinsufficiency and leaving endogenous gene expression regulatory logic intact.
Eric D. Smith, Joshua K. Meisner, Abbey Bullard et al.· bioRxiv· 0 citations
The potential for gene editing and epigenetic therapy for the management of dyslipidemia appears to be well tolerated, and a number of agents with the potential for robust and durable changes to atherosclerotic lipid and lipoprotein risk factors are developed.
S. Nicholls· Current Opinion in Lipidolog...· 0 citations
It is demonstrated that adeno-associated virus-mediated delivery of full-length human ALPK3 prevents cardiomyopathy in neonatal mouse models and reverses established disease in adult mice, restoring cardiac structure, function and proteostasis, as well as contractile function in human cardiac organoids carrying ALPK3-...
James W. McNamara, Ellen B. Keen, Rebecca Sutton et al.· Nature Cardiovascular Resear...· 2 citations
Cell-specific epigenetic editing holds very high therapeutic value for atherosclerosis, cardiomyopathy, and fibrosis, provided that delivery, specificity, and safety challenges are also addressed.
Majed Alsulami, M. Rasool, Ahmed Masoud et al.· The Cardiology· 0 citations
