With the fresh wave of Covid-19 cases rising globally, a new study has found promising potential in tackling lung diseases through nasal spray gene therapy. Researchers at Mass General Brigham have developed a new viral delivery system designed efficiently to target the lungs and respiratory airways including pulmonary fibrosis.
AAV.CPP.16, the new adeno-associated virus variant--which is a non-enveloped virus that can be engineered to deliver DNA to target cells--can be administered through a nasal spray and has shown remarkable efficacy during the preclinical trials.
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Gene therapy itself has proved its promising role in various treatments including cancer by delivering therapeutic genes to specific tissues in the body.
Though AAV vectors struggled to target the lungs effectively as it was designed to penetrate the central nervous system, the recent development of a new viral variant to enter the lung cells was an unexpected turn for the researchers.
"We initially designed it to enter the CNS, but later we noticed that AAV. CPP. 16 efficiently targeted lung cells," said Dr FengFeng Bei, Department of Neurosurgery at Brigham and Women's Hospital.
With further research, scientists investigated the viral variant for intranasal gene delivery to the respiratory airways.
According to researchers, the new gene therapy not only helps in targeting pulmonary fibrosis but also could be effective for Covid-19 treatment as well.
"The gene therapy was administered in two key disease models. First, they targeted pulmonary fibrosis, a chronic lung condition characterised by scar tissue formation, by delivering genes that prevent scarring. Second, a model of Covid-19," said researchers.
“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,” said Bei.