Aug 2026
Durational Study of Persistent Transduction of Rhesus Macaque Lung and Other Organs Following Single Dosing with AAV1-CFTR
The findings indicate sustained gene transfer and transduction for at least 180 days following a single AAV1 administration to the primate lung, with minimal immunopathology, which supports the potential feasibility of extended dosing intervals and informs the development of repeatable AAV-based gene therapies for CF.
Prajita Paul, Tanvi Sharma, M. Afshani et al.
· Human Gene Therapy · 0 citations