New Delhi, May 23
US researchers have engineered a novel gene therapy to target the airway and lungs via a nasal spray.
For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body. It is commonly done by using adeno-associated viruses (AAV) gene therapy.
To improve the AAV's ability to deliver therapeutics specifically to the lungs and airway, researchers at the Mass General Brigham engineered a new version, called AAV.CPP.16, which can be administered with a nasal spray.
In preclinical models, AAV.CPP.16 outperformed previous versions by more effectively targeting the airway and lungs and showing promise for respiratory and lung gene therapy, said the researchers in the paper published in the journal Cell Reports Medicine.
"We noticed that AAV.CPP.16, which we initially engineered to enter the central nervous system, also efficiently targeted lung cells," said senior author FengFeng Bei, from the Department of Neurosurgery at Brigham and Women's Hospital.
"This prompted us to further investigate AAV.CPP.16 for intranasal gene delivery to the respiratory airways," Bei added.
In the study, AAV.CPP.16 outperformed previous versions (AAV6 and AAV9) in cell culture, mouse models, and non-human primate models.
“Our findings highlight AAV.CPP.16 as a promising vector for respiratory and lung gene therapy,†the team said.
They then used the more efficient tool to deliver scar-preventing gene therapy for pulmonary fibrosis, using a mouse model of the respiratory disease.
They also used the tool to deliver gene therapy for a viral infection, where the therapy prevented the replication of the SARS-CoV-2 virus in a mouse model 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," said Bei.
— IANS
Reader Comments
This is revolutionary! India has such high rates of respiratory diseases due to pollution. If this therapy can help with pulmonary fibrosis and even COVID, it could save millions of lives here. Hope Indian researchers collaborate on this soon. ðŸ™
Nasal spray delivery is brilliant - so much easier than injections. But I wonder about the cost? Most Indians can't afford expensive gene therapies. The government should invest in making this accessible if it proves effective.
Interesting research but we should be cautious. Gene therapy is still new and modifying viruses to deliver treatment sounds risky. What if there are unintended consequences? More safety studies needed before this comes to India.
As someone with asthma in Delhi's terrible air, this gives me hope! But will it work for chronic conditions or just acute infections? Also curious if Ayurveda principles could be combined with this modern approach for better results.
Good to see progress, but Indian scientists should lead such innovations too. We have brilliant minds in IITs and AIIMS - they need more funding and support to develop homegrown solutions for our specific health challenges.
The COVID application is most exciting! If this can prevent virus replication, it could be a game-changer for future pandemics. Though I worry about equitable distribution - rich countries always get such treatments first. 😔
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