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Journal Articles

Targeted Lipid Nanoparticle Delivery of an RNA Gene Writer In Vivo Enables Generation of CAR-T Cells in a Humanized Mouse Model

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Jason Rodriguez, Alberto De Iaco, James B. Rottman, Donghui Liu, Kartika Venugopal, Xin Qiao, Yu Cao, Rebecca Levy, Mohit Gupta, Rahul Palchaudhuri, Bill Querbes, Cecilia Cotta-Ramusino, Hari Pujar, Madhusudan V Peshwa, Michael C Holmes
Journal: Blood
Blood (2024) 144 (Supplement 1): 4799.
https://doi.org/10.1182/blood-2024-208176
Published: 2024
Journal Articles

Deploying an RNA-Based Gene Writer System and Lipid Nanoparticle (LNP) Delivery to Generate Functional Chimeric Antigen Receptor (CAR) T Cells with in Vitro and In Vivo Anti-Tumor Activity

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Michael S Magee, Alberto De Iaco, Jason Rodriguez, James B. Rottman, Donghui Liu, Giulia Schiroli, Pooja Sabhachandani, Yu Cao, Rebecca Levy, Mohit Gupta, Michael T. Monte, Jane Wang, Bill Querbes, Cecilia Cotta-Ramusino, Madhusudan V Peshwa, Michael C Holmes
Journal: Blood
Blood (2023) 142 (Supplement 1): 2073.
https://doi.org/10.1182/blood-2023-184755
Published: 2023
Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

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Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022