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The ABCA3 (ATP-binding cassette transporter A3) protein transports phospholipids from the cytoplasm into lamellar bodies in alveolar type II cells (AEC2s) and is critical for pulmonary surfactant synthesis and function.… Click to show full abstract
The ABCA3 (ATP-binding cassette transporter A3) protein transports phospholipids from the cytoplasm into lamellar bodies in alveolar type II cells (AEC2s) and is critical for pulmonary surfactant synthesis and function. Biallelic pathogenic ABCA3 variants cause diverse pulmonary phenotypes, including lethal neonatal respiratory distress syndrome, childhood interstitial lung disease, and idiopathic pulmonary fi brosis Current therapies, including steroids, fi c ef fi cacy transplantation fi
Keywords:
first steps;
steps toward;
therapies abca3;
toward personalized;
personalized therapies;
abca3 deficiency
Journal Title: American Journal of Respiratory Cell and Molecular Biology
Year Published: 2022
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Journal Title: American Journal of Respiratory Cell and Molecular Biology
Year Published: 2022
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!