New Gene Therapy To Target Airway And Lungs Via Nasal Spray

New Delhi: 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.