Inhaled siRNA nanoparticles targeting <i>IL11</i> inhibit lung fibrosis and improve pulmonary function post-bleomycin challenge.

Bai, Xin; Zhao, Guolin; Chen, Qijing; Li, Zhongyu; Gao, Mingzhu; Ho, William; Xu, Xiaoyang; Zhang, Xue-Qing · Sci Adv · 2022

basic_science · Level V

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Abstract

Interleukin-11 (IL-11) is a profibrotic cytokine essential for the differentiation of fibroblasts into collagen-secreting, actin alpha 2, smooth muscle-positive (ACTA2<sup>+</sup>) myofibroblasts, driving processes underlying the pathogenesis of idiopathic pulmonary fibrosis (IPF). Here, we developed an inhalable and mucus-penetrative nanoparticle (NP) system incorporating siRNA against <i>IL11</i> (si<i>IL11</i>@PPGC NPs) and investigated therapeutic potential for the treatment of IPF. NPs are formulated through self-assembly of a biodegradable PLGA-PEG diblock copolymer and a self-created cationic lipid-like molecule G0-C14 to enable efficient transmucosal delivery of si<i>IL11</i>. Noninvasive aerosol inhalation hindered fibroblast differentiation and reduced ECM deposition via inhibition of ERK and SMAD2. Furthermore, si<i>IL11</i>@PPGC NPs significantly diminished fibrosis development and improved pulmonary function in a mouse model of bleomycin-induced pulmonary fibrosis without inducing systemic toxicity. This work presents a versatile NP platform for the locally inhaled delivery of siRNA therapeutics and exhibits promising clinical potential in the treatment of numerous respiratory diseases, including IPF.

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