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肺靶向性肌浆网Ca2+-ATP酶2a基因治疗:从发现到在博来霉素诱导的肺纤维化中的治疗应用

Lung-targeted SERCA2a Gene Therapy: From Discovery to Therapeutic Application in Bleomycin-Induced Pulmonary Fibrosis.

作者信息

Bisserier Malik, Hadri Lahouaria

机构信息

Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.

出版信息

J Cell Immunol. 2020;2(4):149-156. Epub 2020 May 6.

Abstract

Idiopathic pulmonary fibrosis (IPF) is an interstitial lung disease characterized by an accumulation of scar tissue within the lungs and the common presence of usual interstitial pneumonia. Unfortunately, only a few FDA-approved therapeutic options are currently available for the treatment of IPF and IPF remains associated with poor prognosis. Therefore, the identification of new pharmacological targets and strategies are critical for the treatment of IPF. This commentary aims to further discuss the role of sarcoplasmic reticulum Ca-ATPase 2a and its downstream signaling in IPF. Finally, this commentary offers new insights and perspectives regarding the therapeutic potential of AAV-mediated SERCA2A gene therapy as an emerging therapy for respiratory diseases.

摘要

特发性肺纤维化(IPF)是一种间质性肺疾病,其特征是肺部瘢痕组织积聚以及普通型间质性肺炎普遍存在。不幸的是,目前FDA批准的用于治疗IPF的治疗选择很少,且IPF的预后仍然很差。因此,确定新的药理学靶点和策略对于IPF的治疗至关重要。本评论旨在进一步探讨肌浆网Ca-ATP酶2a及其下游信号在IPF中的作用。最后,本评论就腺相关病毒介导的SERCA2A基因治疗作为一种新兴的呼吸系统疾病治疗方法的治疗潜力提供了新的见解和观点。

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