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Journal Articles

First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD: Initial results of PRIME-0101

Free
Elie Haddad, Haydar Frangoul, Donald Kohn, Emma Morris, Jennifer Gori, Bradley Martin, Briana Deary, McKinley Nickerson, Alexandria Petrusich, Pierre Teira, Karine Leveille, Isabel Fernandez, Stuart Turvey, Elizabeth Kang, Suk See De Ravin, Marie Pierzynski, Tyra Estwick-McCann, Patricia Littel, Douglas Kuhns, Debra Long-Priel, Meghann McManus, Misty Evans, Ben Carpenter, Jordyn Arnold, Barrett Nehilla, Jacob Stewart-Ornstein, Tiernan O'Malley, Jack Heath, Mohammed Asmal, Harry Malech
Journal: Blood
Blood (2025) 146 (Supplement 1): 4319.
https://doi.org/10.1182/blood-2025-4319
Published: 2025
Journal Articles

Robust Pre-Clinical Results and Large-Scale Manufacturing Process for Edit-301: An Autologous Cell Therapy for the Potential Treatment of SCD

Free
Edouard De Dreuzy, PhD, Jack Heath, Patricia Sousa, Tusneem Janoudi, Harry An, PhD, Scott Hansen, David K. Wood, PhD, Charles F Albright, PhD, Sandra Teixeira, PhD, Tamara Monesmith, Kate Zhang, PhD, Kai-Hsin Chang, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 45–46.
https://doi.org/10.1182/blood-2020-140073
Published: 2020
Journal Articles

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

Free
Edouard De Dreuzy, PhD, Jack Heath, John A Zuris, PhD, Patricia Sousa, Ramya Viswanathan, Sean Scott, Jen Da Silva, Terence Ta, PhD, Stacy Capehart, PhD, Tongyao Wang, Cecilia Fernandez, PhD, Vic E Myer, PhD, Charles F Albright, PhD, Christopher J Wilson, PhD, Sandra Teixeira, PhD, Kai-Hsin Chang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4636.
https://doi.org/10.1182/blood-2019-130256
Published: 2019
Journal Articles

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

Free
Kai-Hsin Chang, PhD, Minerva Sanchez, Jack Heath, Edouard deDreuzy, PhD, Scott Haskett, MS, Abigail Vogelaar, Kiran Gogi, Jen Da Silva, Tongyao Wang, Andrew Sadowski, Gregory Gotta, Jamaica Siwak, Ramya Viswanathan, Katherine Loveluck, Hoson Chao, Eric Tillotson, Aditi Chalishazar, MS, Abhishek Dass, Frederick Ta, Emily Brennan, Diana Tabbaa, Eugenio Marco, PhD, John A Zuris, PhD, Deepak Reyon, PhD, Meltem Isik, PhD, Ari Friedland, PhD, Terence Ta, PhD, Fred Harbinski, Georgia Giannoukos, PhD, Sandra Teixeira, PhD, Christopher J Wilson, PhD, Charlie Albright, PhD, Haiyan Jiang, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 409.
https://doi.org/10.1182/blood-2018-99-114780
Published: 2018
Journal Articles

Expanding CRISPR Genome Editing Strategies in Hematopoietic Stem and Progenitor Cells for the Treatment of Hematologic Diseases

Free
Edouard deDreuzy, PhD, Aditi Chalishazar, MS, Jack Heath, Carrie M Margulies, PhD, John Labella, Ramya Viswanathan, Katherine Loveluck, KaiHsin Chang, PhD, Hariharan Jayaram, PhD, John A Zuris, PhD, Vic E Myer, PhD, Charlie Albright, PhD, Cecilia Cotta-Ramusino, PhD, Jennifer L Gori, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4619.
https://doi.org/10.1182/blood.V130.Suppl_1.4619.4619
Published: 2017