Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy
| Autor: | Chappell, Maxwell E., Breda, Laura, Tricoli, Lucas, Guerra, Amaliris, Jarocha, Danuta, Castruccio Castracani, Carlo, Papp, Tyler E., Tanaka, Naoto, Hamilton, Nolan, Triebwasser, Michael P., Ghiaccio, Valentina, Fedorky, Megan T., Gollomp, Kandace L., Bochenek, Veronica, Roche, Aoife M., Everett, John K., Cook, Emma J., Bushman, Frederic D., Teawtrakul, Nattiya, Glentis, Stavros, Kattamis, Antonis, Mui, Barbara L., Tam, Ying K., Weissman, Drew, Abdulmalik, Osheiza, Parhiz, Hamideh, Rivella, Stefano |
|---|---|
| Zdroj: | In Blood 10 October 2024 144(15):1633-1645 |
| Databáze: | ScienceDirect |
| Externí odkaz: |
|