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Naoto Tanaka
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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
Journal Articles

Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6 -ASO Is Superior for Correcting β-Thalassemia

Amaliris Guerra, Perry Demsko, Ariel Rivera, Carlo Castruccio Castracani, Laura Breda, Naoto Tanaka, Shuling Guo, Stefano Rivella
Journal: Blood
Blood (2022) 140 (Supplement 1): 1944–1945.
https://doi.org/10.1182/blood-2022-166801
Published: 2022
Journal Articles

In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA

Laura Breda, Tyler E Papp, Michael Triebwasser, Amir Yadegari, Megan T Fedorky, Naoto Tanaka, Houping Ni, Drew Weissman, Stefano Rivella, Hamideh Parhiz
Journal: Blood
Blood (2022) 140 (Supplement 1): 305–306.
https://doi.org/10.1182/blood-2022-165089
Published: 2022
Journal Articles

Improved Gene Therapy for Metachromatic Leukodystrophy

Lucas Tricoli, Adeline Vanderver, Laura Adang, Maxwell Chappell, Laura Breda, Naoto Tanaka, Michael Triebwasser, David Wenger, Danuta Jadwiga Jarocha, Amaliris Guerra, Lars Schlotawa, Karthikeyan Radhakrishnan, Rebecca Ahrens-Nicklas, Peter Kurre, Stephanie Hurwitz, Stefano Rivella
Journal: Blood
Blood (2021) 138 (Supplement 1): 3979.
https://doi.org/10.1182/blood-2021-150986
Published: 2021
Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2242.
https://doi.org/10.1182/blood-2019-124043
Published: 2019