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Michael Jaskolka
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Journal Articles

CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial

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Rabi Hanna, Haydar Frangoul, Luis Pineiro, Christopher McKinney, Markus Mapara, Jignesh Dalal, Hemalatha Rangarajan, Harold Atkins, Monica Bhatia, Deepakbabu Chellapandian, Jennifer Jaroscak, Lakshmanan Krishnamurti, Janet Kwiatkowski, Lyndsay Molinari, Elizabeth Stenger, Michael Jaskolka, Qifeng Yu, Nnenna Badamosi, Brieana Cox-Buckley, Baisong Mei, Olubunmi Afonja, Mark Walters
Journal: Blood
Blood (2025) 146 (Supplement 1): 4314.
https://doi.org/10.1182/blood-2025-4314
Published: 2025
Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Free
Haydar Frangoul, Rabi Hanna, Mark C. Walters, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Baisong Mei, Olubunmi Afonja, Alexis Thompson
Journal: Blood
Blood (2024) 144 (Supplement 1): 7456.
https://doi.org/10.1182/blood-2024-208623
Published: 2024
Journal Articles

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Free
Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Daphne L. Farrington, Maha Wally, Baisong Mei, Adebayo Lawal, Olubunmi O. Afonja, Mark C. Walters
Journal: Blood
Blood (2023) 142 (Supplement 1): 4996.
https://doi.org/10.1182/blood-2023-187397
Published: 2023