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 …
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
Strategies for non-viral vectors targeting organs beyond the liver
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 …
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 …
ventricular wall thickness not solely explained by abnormal loading conditions. It is often …
Familial hypertrophic cardiomyopathy: diagnosis and management
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 …
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 …
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 …
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 …
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 …
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 …
affecting approximately 1 in 500 individuals. Current therapeutic interventions comprise …
Applications of gene therapy in cardiomyopathies
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 …
existing genes and/or their regulatory portions via gene replacement and gene editing …