ArticleMolecular therapy. Advances2026
Efficient and safe lung gene delivery using AAV6.2FF in neonatal pigs demonstrates pediatric translational potential.
Article in Molecular therapy. Advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
While adeno-associated virus (AAV) vectors have demonstrated efficacy in multiple organ systems, gene delivery to the lung remains an unmet therapeutic target. To treat a monogenic lung disease like surfactant protein B deficiency, a gene delivery system must overcome pulmonary barriers and be validated in models that closely resemble the human respiratory tract. Here, we optimized the endotracheal delivery of AAV6.2FF, an engineered capsid with enhanced pulmonary tropism, in neonatal piglets with lung features comparable to those of human newborns. We optimized delivery parameters that improved pulmonary distribution and applied them to administer AAV6.2FF expressing secreted alkaline phosphatase (SEAP) at two different doses: 5 × 10
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