Highly Efficient In Vivo Hematopoietic Stem Cell Transduction Using an Optimized Self-Complementary Adeno-Associated Virus

Investigators used adeno-associated virus serotype 6 (AAV6) vectors and Cre recombination to systematically optimize the parameters for effective in vivo HSC transduction. They establishing an protocol that achieved functional recombination in over two-thirds of immunophenotypic HSCs.