Researchers Develop Nasal Spray Gene Therapy To Target Lungs And Airways

US scientists have engineered a novel gene therapy vector, AAV.CPP.16, which can be delivered via a nasal spray to efficiently target the airway and lungs.

This breakthrough could improve treatment for respiratory diseases by enhancing the delivery of therapeutic molecules directly to the lungs.

Nasal Spray Gene Therapy With AAV.CPP.16

Gene therapy often uses adeno-associated viruses (AAV) to deliver therapeutic genes to specific body sites.

Researchers at Mass General Brigham modified the existing AAV to create AAV.CPP.16, which shows superior ability to reach lung and airway cells compared to earlier versions like AAV6 and AAV9.

“We initially designed AAV.CPP.16 to enter the central nervous system,” said senior author FengFeng Bei from Brigham and Women’s Hospital. “But we found it also targets lung cells very efficiently, prompting us to explore its use for intranasal delivery to the respiratory tract.”

Successful Testing In Multiple Models

The team tested AAV.CPP.16 in cell cultures, mice, and non-human primates. In all models, it outperformed previous vectors by effectively delivering genes to lung tissues. The researchers published these results in the journal Cell Reports Medicine.

Using AAV.CPP.16, researchers delivered gene therapy that prevented scarring in a mouse model of pulmonary fibrosis, a serious lung disease. They also used the vector to block SARS-CoV-2 replication in a COVID-19 mouse model, demonstrating its potential for treating viral infections.

Promising Future For Respiratory Gene Therapy

Bei concluded that their findings suggest intranasal AAV.CPP.16 has strong translational potential as a delivery tool for lung and airway gene therapies, although more research is needed.

This advancement could open new avenues for treating respiratory illnesses by enabling non-invasive, targeted gene delivery directly through the nose.

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