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Barbara L. Mui
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Journal Articles

Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy

Maxwell E. Chappell, Laura Breda, Lucas Tricoli, Amaliris Guerra, Danuta Jarocha, Carlo Castruccio Castracani, Tyler E. Papp, Naoto Tanaka, Nolan Hamilton, Michael P. Triebwasser, Valentina Ghiaccio, Megan T. Fedorky, Kandace L. Gollomp, Veronica Bochenek, Aoife M. Roche, John K. Everett, Emma J. Cook, Frederic D. Bushman, Nattiya Teawtrakul, Stavros Glentis, Antonis Kattamis, Barbara L. Mui, Ying K. Tam, Drew Weissman, Osheiza Abdulmalik, Hamideh Parhiz, Stefano Rivella
Journal: Blood
Blood (2024) 144 (15): 1633–1645.
https://doi.org/10.1182/blood.2023023349
Published: 2024
Includes: Supplemental data