shAAV-CXCL1

shAAV-CXCL1 is a preclinical gene therapy candidate consisting of an adeno-associated virus serotype 8 (AAV8) vector that delivers a short hairpin RNA (shRNA) targeting C-X-C motif chemokine ligand 1 (CXCL1). Developed by researchers at Yonsei University, it is designed for liver-specific knockdown of CXCL1 to treat cardiac dysfunction associated with heart failure. The therapy leverages the liver-to-heart communication axis, where hepatic CXCL1 expression is upregulated during heart failure and contributes to myocardial fibrosis and hypertrophy. By reducing hepatic CXCL1 levels via intravenous administration, shAAV-CXCL1 aims to attenuate cardiac remodeling and improve heart function without systemic toxicity.