Demonstration of Safety in Wild Type Mice of npFOXF1, a Novel Nanoparticle-Based Gene Therapy for Alveolar Capillary Dysplasia with Misaligned Pulmonary Veins.
Fatemeh KohramZicheng DengYufang ZhangAbid A RezaEnhong LiOlena A KolesnichenkoSamriddhi ShuklaVladimir UstiyanJose Gomez-ArroyoAnusha AcharyaDonglu ShiVladimir V KalinichenkoAlan P KennyPublished in: Biologics : targets & therapy (2023)
In conclusion, npFOXF1 has a very good safety profile and combined with preceding studies showing therapeutic efficacy, npFOXF1 can be considered as a good candidate therapy for ACDMPV in human neonates.