Aug 2026
Durational Study of Persistent Transduction of Rhesus Macaque Lung and Other Organs Following Single Dosing with AAV1-CFTR.
Adeno-associated virus (AAV)-mediated gene transfer remains a promising strategy for cystic fibrosis (CF), but durability of expression and optimal dosing intervals are unresolved challenges. Here, we evaluated long-term gene transfer, transduction, and immunological responses following a single pulmonary administratio...
Prajita Paul, Tanvi Sharma, Masoud Afshani et al.
· Human Gene Therapy · 0 citations