Article
Lung-tropic dual AAV-SP-C and microRNA gene therapy attenuates lung injury in mutant Sftpc mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2026
Bardin Pauline, Xu Liqun, Pei Yanlong, Vineeth Adithya Achuthan, Nangle Kennedy, Zhong Shumei, Cyr-Depauw Chanèle, Vadivel Arul, Martin Emily, Weaver Timothy E, Sitaraman Sneha, Wootton Sarah K, Thébaud Bernard
Abstract excerpt
Mutations in the surfactant protein C (SP-C) gene (SFTPC) impair surfactant homeostasis, leading to respiratory distress in newborns or progressive interstitial lung disease. The most frequent mutation, I73T, results in the expression of a toxic dominant-negative form of SP-C, insinuating that for gene replacement therapy to be successful, suppression of the toxic form of the SP-C protein may also be necessary....
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