Life-threatening arrhythmias are a major consequence of reduced cardiac sodium current with limited treatment options available. A recent study published in the European Heart Journal by researchers from Amsterdam University Medical Centers and collaborators explored a novel gene therapy approach to enhance the cardiac sodium current and prevent arrhythmias.
Raythera Inc. has identified TNF-α modulators reported to be useful for the treatment of inflammatory disorders, autoimmune disease, neurological disorders, pain, cardiovascular disorders, cancer, metabolic disorders and eye disorders.
Epigenetic editing is a promising method for gene regulation in vitro and in vivo, allowing precise control of gene expression without altering the DNA sequence, thereby minimizing genotoxic risks.
The map of cystic fibrosis (CF) research is being redrawn in the U.K. as improvements in treatment, and in particular the introduction of CF modulator drugs, mean people with the rare inherited disease are living much longer.
Pannexin 1 (Panx1) is a high-conductance, voltage-sensitive ion channels that exhibit flexible gating behavior upon activation, enabling the passage of ions such as Ca2+, Na⁺, K⁺, and ATP. Panx1 is expressed in cardiac tissue, but its role in ATP release and electrophysiological processes affecting cardiac function is not fully understood. Researchers from Vanderbilt University Medical Center have found that in isolated ventricular cardiomyocytes, Panx1 activation during spontaneous sarco/endoplasmic reticulum Ca2+ release amplified the NCX-driven transient inward current.
Pulmonary hypertension, despite advances in treatment, still has a poor prognosis, which is often associated with right heart failure, and for which pulmonary arterial hypertension (PAH) is a severe form. There is an urgent need to unravel the molecular mechanisms behind PAH and identify new targets, especially those involved in the remodeling process. The proliferation and resistance to apoptosis of pulmonary artery smooth muscle cells are key contributors to pulmonary vascular remodeling in PAH.
Researchers from Tikkunlev Therapeutics Inc. and the University of Utah have presented preclinical data on TLT-101, a gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector encoding cardiac bridging integrator 1 (cBIN1) designed for the treatment of heart failure.
The lung and thrombosis may play a key role in cancer and metastasis progression, according to a collaborative study led by Cornell University scientists. In the nonmetastatic lung microenvironment of several cancer types, the development of a prothrombosis niche promotes metastasis formation through the release of small extracellular vesicles loaded with an integrin protein.
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