CRISPR-Edited Mice Help Reveal Potential Nanoparticle Treatment for ACDMPV

Research By Vladimir Kalinichenko, MD, PhD

Post Date: August 12, 2019 | Publish Date: June 14, 2019

This confocal microscopic image shows FOXF1 deficient fetal lung cells from mice. FOXF1 deficiency inhibits the normal development and proliferation of lung cells and helps cause a fatal disease in infants called Alveolar Capillary Dysplasia with Misalignment of Pulmonary Veins.

This confocal microscopic image shows FOXF1 deficient fetal lung cells from mice. FOXF1 deficiency inhibits the normal development and proliferation of lung cells and helps cause a fatal disease in infants called Alveolar Capillary Dysplasia with Misalignment of Pulmonary Veins.

Alveolar Capillary Dysplasia with Misalignment of Pulmonary Veins (ACDMPV) is a very rare congenital respiratory disorder that usually proves fatal to infants within a month of birth.

But new findings from researchers at Cincinnati Children’s suggest a potential step forward toward longer survival.

Scientists here used CRISPR/Cas9 to generate mice that faithfully mimic the disorder. The new mouse model allowed researchers to pinpoint the disorder’s cause and identify a potential nanoparticle-based treatment.

Findings appear in the  American Journal of Respiratory and Critical Care Medicine.

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Publication Information

Original Title:The S52F FOXF1 Mutation Inhibits STAT3 Signaling and Causes Alveolar Capillary Dysplasia
Published in:American Journal of Respiratory and Critical Care Medicine
Publish date:June 14, 2019

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