Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy
Maxwell Chappell(Children's Hospital of Philadelphia), Stefano Rivella(Children's Hospital of Philadelphia), J.K. Everett(University of Pennsylvania), Barbara L. Mui(Arbutus Biopharma (Canada)), Frederic D. Bushman(University of Pennsylvania), Lucas Tricoli(Children's Hospital of Philadelphia), Danuta Jarocha(Jagiellonian University), Carlo Castruccio Castracani(Children's Hospital of Philadelphia), Amaliris Guerra(Children's Hospital of Philadelphia), Stavros Glentis, Valentina Ghiaccio(Children's Hospital of Philadelphia), Antonis Kattamis, Michael Triebwasser(University of Michigan), Naoto Tanaka(Temple College), Osheiza Abdulmalik(Children's Hospital of Philadelphia), Ying K. Tam(Arbutus Biopharma (Canada)), Drew Weissman(California University of Pennsylvania), Laura Breda(Children's Hospital of Philadelphia), Aoife M. Roche(California University of Pennsylvania), Hamideh Parhiz(Translational Therapeutics (United States)), Veronica Bochenek(Children's Hospital of Philadelphia), Kandace Gollomp(Children's Hospital of Philadelphia), Nattiya Teawtrakul(Khon Kaen University), Emma J. Cook(University of Pennsylvania), Megan Fedorky(Children's Hospital of Philadelphia), Nolan Hamilton(Children's Hospital of Philadelphia), Tyler E. Papp(Translational Therapeutics (United States))
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