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Journal Articles

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Lorenzo Tozzi, Giulia Schiroli, Yaser Heshmati, Yu Cao, Michael T. Monte, Apiwat Wangweerawong, James B. Rottman, Rahul Palchaudhuri, Jean-Antoine Ribeil, John Manis, Hari Pujar, William Querbes, Anne Bothmer, Cecilia Cotta-Ramusino, Z. Jane Wang, William E. Salomon, Michael C Holmes

Blood (2024) 144 (Supplement 1): 515.

https://doi.org/10.1182/blood-2024-208626

Published: 2024

Journal Articles

Delivery of Gene Writers In Vivo to Hematopoietic Stem Cells and T Cells Using Targeted and Untargeted Lipid Nanoparticles (LNP)

Michael T. Monte, Lorenzo Tozzi, Rahul Palchaudhuri, Apiwat Wangweerawong, Yaser Heshmati, Giulia Schiroli, Han Gu, Yu Cao, William E. Salomon, Anne Bothmer, Jason Rodriguez, Mohit Gupta, James B. Rottman, William Querbes, Cecilia Cotta-Ramusino, Michael C Holmes, Hari Pujar, Z. Jane Wang

Blood (2024) 144 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2024-208968

Published: 2024

Journal Articles

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

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

Blood (2024) 144 (Supplement 1): 4799.

https://doi.org/10.1182/blood-2024-208176

Published: 2024

Journal Articles

In Vivo Delivery of RNA By Lipid Nanoparticles (LNP) to Hematopoietic Stem Cells and T Cells in Humanized Mice and Non-Human Primates

Blood (2023) 142 (Supplement 1): 4818.

https://doi.org/10.1182/blood-2023-187271

Published: 2023