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lenti-ACE2 is an experimental gene therapy consisting of a lentiviral vector encoding the Angiotensin-Converting Enzyme 2 (ACE2) gene. It is designed to increase the expression of ACE2 in target tissues, particularly the lungs. ACE2 is a critical negative regulator of the renin-angiotensin system (RAS); it cleaves Angiotensin II (a potent vasoconstrictor and pro-fibrotic peptide) into Angiotensin (1-7), which possesses vasodilatory, anti-proliferative, and anti-inflammatory properties. By shifting the RAS balance away from the ACE/Ang II/AT1 receptor axis and toward the ACE2/Ang(1-7)/Mas receptor axis, lenti-ACE2 aims to treat conditions characterized by RAS overactivation, such as pulmonary arterial hypertension (PAH). Preclinical studies have demonstrated that local delivery of lenti-ACE2 to the lungs can prevent and reverse pulmonary vascular remodeling, reduce right ventricular hypertrophy, and lower pulmonary artery pressure.
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