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Matthew Heeney
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Journal Articles

Robust HbF induction and improvement of anemia and hemolysis with base editing in sickle cell disease: Safety and efficacy findings from the ongoing BEACON study

Free
Ashish Gupta, Akshay Sharma, Haydar Frangoul, Julie Kanter, Markus Mapara, Jignesh Dalal, Asif Alavi, Jennifer Jaroscak, Ernesto Ayala, John DiPersio, Edward D. Ziga, Mary Eapen, Stacey Rifkin-Zenenberg, Srikanth Ambati, Yinzhong Chen, Sarah Chesler, Marcelyne Joseney-Antoine, Leanne Ianniello, Bahru Habtemariam, Priya Chockalingam, Ling Lin, Sunita Goyal, Amy Simon, Alexis Thompson, Matthew Heeney
Journal: Blood
Blood (2025) 146 (Supplement 1): 2532.
https://doi.org/10.1182/blood-2025-2532
Published: 2025
Journal Articles

Transcriptomic analysis uncovers hidden intronic and synonymous pathogenic variants in red cell membrane disorders

Free
Wenying Zhang, Yasmin Elgammal, Ammar Husami, Aikaterini Voulgaridou, Haripriya Sakthivel, Marcus Cannon, Leah Bonavita, Michael Grimley, Matthew Heeney, Athina Ntoumaziou, Mary Risinger, Elizabeth Yang, Theodosia Kalfa
Journal: Blood
Blood (2025) 146 (Supplement 1): 282.
https://doi.org/10.1182/blood-2025-282
Published: 2025
Journal Articles

Apheresis blood processing with heparin improves stem cell collection outcomes for sickle cell gene therapy

Free
David Justus, Carly Howard, Ellen Proeung, Aimee Sarnie, Jen Fortin, Erica Esrick, Matthew Heeney, Daniel Bauer, Sarah Nikiforow, Jose Cancelas, Leslie Lehmann, David Williams, John Manis
Journal: Blood
Blood (2025) 146 (Supplement 1): 6096.
https://doi.org/10.1182/blood-2025-6096
Published: 2025