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Journal Articles

In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers

Free
Yaser Heshmati, Giulia Schiroli, Lorenzo Tozzi, Michael Monte, Nouhaila Beytour, Apiwat Wangweerawong, James Rottman, William E. Salomon, Rahul Palchaudhuri, Z. Jane Wang, Anne Bothmer, Kim Vanuytsel, John Manis, Lev Gorfinkel, Francesca Alvarez Calderon, Ulrike Gerdemann, Leslie Kean, Hari Pujar, Cecilia Cotta-Ramusino, Raja Prince-Eladnani, Michael C Holmes
Journal: Blood
Blood (2025) 146 (Supplement 1): 2536.
https://doi.org/10.1182/blood-2025-2536
Published: 2025
Journal Articles

In Vivo RNA delivery by targeted lipid nanoparticles enable gene editing in hematopoietic stem cells and T cells

Free
Rahul Palchaudhuri, Michael Monte, Mohit Gupta, Giulia Schiroli, Lorenzo Tozzi, Lei Liu, Han Gu, Rebecca Goldman, Apiwat Wangweerawong, Aaron Zannini, Yaser Heshmati, Christina Ferren, Alyssa Sanfilippo, Tiffany Morales, Jessica Desmond, Kartika Venugopal, Jason Rodriguez, Albert De Iaco, Cecilia Cotta-Ramusino, William E. Salomon, James Rottman, William Querbes, Hari Pujar, Michael C Holmes, Z. Jane Wang
Journal: Blood
Blood (2025) 146 (Supplement 1): 4318.
https://doi.org/10.1182/blood-2025-4318
Published: 2025
Journal Articles

Targeted lnp delivery of an RNA gene writer In Vivo enables generation of functional CAR-T cells

Free
Cecilia Cotta-Ramusino, Albert De Iaco, James Rottman, Jason Rodriguez, Donghui Li, Kartika Venugopal, Jessica Desmond, Christina Ferren, Mohit Gupta, Rebecca Levy, Rahul Palchaudhuri, Z. Jane Wang, William Querbes, Hari Pujar, Michael C Holmes
Journal: Blood
Blood (2025) 146 (Supplement 1): 6097.
https://doi.org/10.1182/blood-2025-6097
Published: 2025
Journal Articles

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

Free
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 2197.
https://doi.org/10.1182/blood-2024-208968
Published: 2024
Journal Articles

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

Free
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 515.
https://doi.org/10.1182/blood-2024-208626
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

Free
Michael T. Monte, Han Gu, Laura Saunders, Mohit Gupta, Lei Liu, Apiwat Wangweerawong, Thomas V. Colace, Aaron Zannini, Lorenzo Tozzi, Yu Cao, Giulia Schiroli, William E. Salomon, James B. Rottman, Rahul Palchaudhuri, Ulrike Gerdemann, Lev Gorfinkel, Francesca Alvarez Calderon, Leslie Kean, Hari Pujar, Z. Jane Wang
Journal: Blood
Blood (2023) 142 (Supplement 1): 4818.
https://doi.org/10.1182/blood-2023-187271
Published: 2023
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

Free
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