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Donald Kohn
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Journal Articles

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Free
Erica Esrick, Leslie Lehmann, Amy Federico, Heather Daley, Colleen Dansereau, Satiro DeOliveira, John Everett, Pei-Chi Kao, Boya Liu, Theodore Moore, Emily Morris, Helene Negre, Kit Shaw, Gavin Roach, Jerome Ritz, Aoife Doto, Olivia Silva, Helene Vincon, Frederic Bushman, Donald Kohn, Akshat Jain, Wendy London, David Justus, Myriam Armant, John Manis, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 2533.
https://doi.org/10.1182/blood-2025-2533
Published: 2025
Journal Articles

Synthetic oligonucleotides contain synthesis errors that are propagated into the genome by editing through homology-directed repair

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Stacia Wyman, Zulema Romero Garcia, Seok-Jin Heo, Marian Navarrete, Netravathi Krishnappa, Donald Kohn, David Martin, Mark Walters, Dario Boffelli
Journal: Blood
Blood (2025) 146 (Supplement 1): 4321.
https://doi.org/10.1182/blood-2025-4321
Published: 2025
Journal Articles

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

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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

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

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John Manis, David Justus, Erica Esrick, Edmund Waller, Mary Horowitz, Leslie Lehmann, Amy Federico, Colleen Dansereau, Emily Morris, Leah Cheng, Mehrdad Abedi, Michael Pulsipher, Neena Kapoor, Gary Schiller, Angela Treml, Sonali Chaudhury, Victoria Coleman-Cowger, Kit Shaw, Jerome Ritz, Heather Daley, Donald Kohn, Akshat Jain, Jeanne Hendrickson, Carly Howard, Aimee Sarnie, Ellen Proeung, Helene Vincon, Myriam Armant, Joseph Antin, Mark Walters, Mary Eapen, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 6094.
https://doi.org/10.1182/blood-2025-6094
Published: 2025
Journal Articles

Comparative Results of Gene Therapy for Adenosine Deaminase Deficiency with or without PEG-ADA Withdrawal and Myelosuppressive Chemotherapy.

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Robert Sokolic, MD, Greg Podsakoff, MD, Linda Muul, PhD, Barbara Engel, MD, Jayashree Jagadeesh, BS, Elizabeth Garabedian, RN, MSLS, Denise Carbonaro, Laura Tuschong, BS, Joanna Ireland, Michael Hershfield, MD, John Tisdale, MD, Cynthia Dunbar, MD, Alan Wayne, MD, Donald Kohn, MD, Fabio Candotti, MD
Journal: Blood
Blood (2007) 110 (11): 501.
https://doi.org/10.1182/blood.V110.11.501.501
Published: 2007
Journal Articles

In Vivo Biosafety Model To Assess Risk of Adverse Events from Retroviral and Lentiviral Vectors.

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Gerhard Bauer, Mo Dao, Ph.D., Scott Case, Ph.D., Todd Meyerrose, Ph.D., Louisa Wirthlin, Phillip Herrbrich, Ph.D., Ping Zhou, Ph.D., Xiuli Wang, Ph.D., Susie Csik, Jesusa Arevalo, Dianne Skelton, Jon Walker, Karen Pepper, Gay Crooks, MD, Donald Kohn, MD, Jan Nolta, Ph.D.
Journal: Blood
Blood (2007) 110 (11): 2595.
https://doi.org/10.1182/blood.V110.11.2595.2595
Published: 2007
Journal Articles

Human Progenitor and Stem Cell Expansion through Selective, Reversible Cytokine Receptor Signaling.

Free
Hisham Abdel-Azim, MD, Yuhua Zhu, Roger Hollis, PhD, Xuili Wang, PhD, Qian-Lin Hao, Lora Barsky, Donald Kohn, MD, Gay Crooks, MD
Journal: Blood
Blood (2005) 106 (11): 31.
https://doi.org/10.1182/blood.V106.11.31.31
Published: 2005
Journal Articles

Genetically Corrected Autologous Stem Cells Engraft, But Host Immune Responses Limit Their Utility in Canine -L-iduronidase Deficiency

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Carolyn Lutzko, Stephen Kruth, Anthony C.G. Abrams-Ogg, Kathy Lau, Liheng Li, Brian R. Clark, Christine Ruedy, Shaherose Nanji, Robert Foster, Donald Kohn, Robert Shull, Ian D. Dubé
Journal: Blood
Blood (1999) 93 (6): 1895–1905.
https://doi.org/10.1182/blood.V93.6.1895.406k02_1895_1905
Published: 1999