Nature Methods · 20 authors, 10 centres
This summary was generated by AI from a single paper. It has not been reviewed by a clinician and is not clinical advice. Verify against the source before acting on it.
The study developed the SELECTIV mouse system, which combines engineered AAV receptor (AAVR) expression with Cre recombinase to achieve efficient, tissue-specific transgene delivery using adeno-associated virus (AAV) vectors. The central result is that this system enables near-complete loss of transduction in non-target tissues while allowing efficient expression in target cells.
The researchers created a versatile platform named SELECTIV for targeted in vivo transgene delivery in mice. This design restricts efficient AAV transduction to cells where Cre recombinase is active. The authors validated the system using five different Cre mouse lines targeting various tissues. They demonstrated that AAVR overexpression significantly enhanced transduction in target tissues, such as the heart (9.7-fold increase), while reducing or eliminating transduction in non-target organs like the liver. The system also improved transduction in cell types normally refractory to AAV, such as muscle stem cells. A key limitation noted is that the system requires extra breeding to establish the necessary genetic crosses. The study establishes SELECTIV as a flexible tool for biomedical research, enabling precise genetic manipulation in specific cell types for applications like gene function studies and disease modeling.
