SLN501
/ Silence Therap, Mallinckrodt
- LARVOL DELTA
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February 19, 2024
Efficacy of GalNAc C3 siRNAs in factor H-deficient mice with C3 glomerulopathy.
(PubMed, Mol Immunol)
- "SLN501 effectively prevented liver C3 synthesis, thus limiting AP dysregulation, glomerular C3d deposits and the development of ultrastructural alterations. These data provide firm evidence of the use of siRNA-mediated liver C3 gene silencing as a potential therapy for treating C3G patients with either partial or complete FH loss of function."
Journal • Preclinical • Complement-mediated Rare Disorders • Glomerulonephritis • Nephrology • Renal Disease
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