Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

Strategies for non-viral vectors targeting organs beyond the liver

J Kim, Y Eygeris, RC Ryals, A Jozić, G Sahay - Nature nanotechnology, 2024 - nature.com
In recent years, nanoparticles have evolved to a clinical modality to deliver diverse nucleic
acids. Rising interest in nanomedicines comes from proven safety and efficacy profiles …

Targeted therapies in pediatric and adult patients with hypertrophic heart disease: from molecular pathophysiology to personalized medicine

E Monda, A Bakalakos, M Rubino, F Verrillo… - Circulation: Heart …, 2023 - Am Heart Assoc
Hypertrophic cardiomyopathy is a myocardial disease defined by an increased left
ventricular wall thickness not solely explained by abnormal loading conditions. It is often …

Familial hypertrophic cardiomyopathy: diagnosis and management

MJ Litt, A Ali, N Reza - Vascular health and risk management, 2023 - Taylor & Francis
Hypertrophic cardiomyopathy (HCM) is widely recognized as one of the most common
inheritable cardiac disorders. Since its initial description over 60 years ago, advances in …

[HTML][HTML] Current therapies for osteoarthritis and prospects of CRISPR-based genome, epigenome, and RNA editing in osteoarthritis treatment

Y Chen, X Luo, R Kang, K Cui, J Ou, X Zhang… - Journal of Genetics and …, 2024 - Elsevier
Osteoarthritis (OA) is one of the most common degenerative joint diseases worldwide,
causing pain, disability, and decreased quality of life. The balance between regeneration …

Human engineered cardiac tissue model of hypertrophic cardiomyopathy recapitulates key hallmarks of the disease and the effect of chronic mavacamten treatment

K Wang, BJ Schriver, R Aschar-Sobbi, AY Yi… - … in Bioengineering and …, 2023 - frontiersin.org
Introduction: The development of patient-specific induced pluripotent stem cell-derived
cardiomyocytes (iPSC-CMs) offers an opportunity to study genotype-phenotype correlation …

Gene editing innovations and their applications in cardiomyopathy research

E Kyriakopoulou, T Monnikhof… - Disease Models & …, 2023 - journals.biologists.com
Cardiomyopathies are among the major triggers of heart failure, but their clinical and genetic
complexity have hampered our understanding of these disorders and delayed the …

CRISPR-Cas9 base editing of pathogenic CaMKIIδ improves cardiac function in a humanized mouse model

S Lebek, XM Caravia, LG Straub… - The Journal of …, 2024 - Am Soc Clin Investig
Cardiovascular diseases are the most common cause of worldwide morbidity and mortality,
highlighting the necessity for advanced therapeutic strategies. Ca2+/calmodulin-dependent …

[HTML][HTML] Gene therapy in cardiology: is a cure for hypertrophic cardiomyopathy on the horizon?

ED Paratz, J Mundisugih, SJ Rowe, E Kizana… - Canadian Journal of …, 2023 - Elsevier
Hypertrophic cardiomyopathy (HCM) is the commonest genetic cardiomyopathy world-wide,
affecting approximately 1 in 500 individuals. Current therapeutic interventions comprise …

Applications of gene therapy in cardiomyopathies

A Argiro, Q Bui, KN Hong, E Ammirati, I Olivotto, E Adler - Heart Failure, 2024 - jacc.org
Gene therapy is defined by the introduction of new genes or the genetic modification of
existing genes and/or their regulatory portions via gene replacement and gene editing …