ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026
Lung-tropic dual AAV-SP-C and microRNA gene therapy attenuates lung injury in mutant Sftpc mice.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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4 citing papers in PubMed.
- Evaluation and Management of Genetic Respiratory Disorders Presenting as Hypoxemic Respiratory Failure in the Newborn Infant.Clinics in perinatology · 2026Review
- Advancing dual gene therapy for SFTPC deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Loss of Alkbh5 enhances AT2 cell differentiation and alveolar repair across diverse injury models via mInternational journal of biological sciences · 2026Article
- Lactylation-driven PDLIM1/PDAP1 axis remodels the inflammatory landscape of acute lung injury: mechanistic insights and precision intervention.Frontiers in immunology · 2026Article
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13 authors.
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Abstract
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. Here, we capitalized on our rationally designed lung-tropic adeno-associated virus (AAV)6.2FF vector to develop a combinatorial gene therapy approach for treating SP-C disorders. In I73T-knockin mice exhibiting decreased Sftpc expression and toxic prosurfactant protein C (proSP-C) accumulation resulting in focal airspace enlargement, gene replacement therapy via airway delivery of AAV6.2FF expressing SP-C restored wild-type (WT) Sftpc and mature SP-C protein expression while significantly improving lung function and focal airspace enlargement. Next, we developed a dual-function AAV6.2FF to express functional SP-C and suppress the toxic 173T SFTPC gene (AAV-SPC-miR). The dual-function AAV6.2FF-SP-C-miR decreased endogenous Sftpc expression, restored WT Sftpc, re-expressed the mature SP-C protein without significant proSP-C protein accumulation, and attenuated airspace enlargement. These findings suggest that combination gene therapy is feasible and represents a promising tool for treating SP-C deficiencies and SFTPC mutation-linked lung diseases in humans.
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