Graphical summary. Credit score: Cell Stories Drugs (2025). DOI: 10.1016/j.xcrm.2025.102144
For gene remedy to work effectively, therapeutic molecules should be effectively delivered to the proper areas within the physique—a job generally given to adeno-associated viruses (AAV).
To enhance the AAV’s skill to ship therapeutics particularly to the lungs and airway, Mass Common Brigham researchers developed and utilized a brand new model, referred to as AAV.CPP.16, that may be administered with a nasal spray. In preclinical fashions, the modern device outperformed earlier variations by extra successfully focusing on the airway and lungs and exhibiting promise for respiratory and lung gene remedy. Outcomes are revealed in Cell Stories Drugs.
“We noticed that AAV.CPP.16, which we initially engineered to enter the central nervous system, also efficiently targeted lung cells,” stated senior writer FengFeng Bei, Ph.D., of the Division of Neurosurgery at Brigham and Girls’s Hospital. “This prompted us to further investigate AAV.CPP.16 for intranasal gene delivery to the respiratory airways.”
The researchers discovered AAV.CPP.16 outperformed earlier variations (AAV6 and AAV9) in cell tradition, mouse fashions, and non-human primate fashions. They then used the extra environment friendly device to ship scar-preventing gene remedy for pulmonary fibrosis, utilizing a mouse mannequin of the respiratory illness. Additionally they used the device to ship gene remedy for a viral an infection, the place the remedy prevented the replication of the SARS-CoV-2 virus in a mouse mannequin of COVID-19.
“Although further research is needed, our findings suggest that intranasal AAV.CPP.16 has strong translational potential as a promising delivery tool for targeting the airway and lung,” stated Bei.
Extra data:
Zhi Yang et al, Cross-species tropism of AAV.CPP.16 within the respiratory tract and its gene therapies in opposition to pulmonary fibrosis and viral an infection, Cell Stories Drugs (2025). DOI: 10.1016/j.xcrm.2025.102144
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Researchers develop gene remedy that may goal airway and lungs by way of nasal spray (2025, Might 22)
retrieved 23 Might 2025
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