Publications by authors named "Donald Kohn"

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Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report.
Jessie L Alexander, Blachy J Dávila Saldaña, Ruta Brazauskas, Sravya Gethika Dammalapati, Linda M Griffith, Donald B Kohn

Blood Adv· December 2025

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Prime Editing for p47-Deficient Chronic Granulomatous Disease.
Jennifer L Gori, Elie Haddad, Haydar Frangoul, Donald B Kohn, Emma C Morris

N Engl J Med· December 2025

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Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers.
Shanmuganathan Chandrakasan, Adrianna Westbrook, Linda M Griffith, David Hagin, Sumathi Iyengar, Donald B Kohn

Clin Immunol· December 2025

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TriLeukeVax: A CD80/IL-15/IL-15Rα Expressing Autologous AML Cell Vaccine Elicits Robust Anti-Leukemic Cytolytic Activity.
Jacob Du, Uthpala N Wijayaratna, Xinyue Wang, Jeffrey P Fung, Belinda Huang, Donald B Kohn

bioRxiv· November 2025

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Outcomes Following Matched Sibling Donor Transplantation for Severe Combined Immunodeficiency: A Report from the PIDTC.
Ahmad Rayes, Brent R Logan, Xuerong Liu, Jasmeen Dara, Rebecca H Buckley, Donald B Kohn

Blood Adv· November 2025

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Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.
Claire Booth, Julián Sevilla, Elena Almarza, Caroline Y Kuo, Josune Zubicaray, Donald B Kohn

Obstet Gynecol Surv· November 2025

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Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.
Eva E R Segura, Kevyn Hart, Beatriz Campo Fernandez, Devin Brown, Kevin Tam, Donald B Kohn

Cell Rep Med· October 2025

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Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report.
Danielle E Arnold, Jennifer W Leiding, Brent Logan, Rebecca A Marsh, Linda M Griffith, Donald B Kohn

Transplant Cell Ther· October 2025

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Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.
Claire Booth, Katelyn Masiuk, Konstantinos Vazouras, Augustine Fernandes, Jinhua Xu-Bayford, Donald B Kohn

N Engl J Med· October 2025

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Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.
Kevyn L Hart, Ralph Valentine Crisostomo, Annika Mittelhauser, Lingyu Zhan, Nika Kononov, Donald B Kohn

Mol Ther· September 2025

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Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia.
Christopher R Luthers, Annika Mittelhauser, Aurelien Colamartino, Xiaomeng Wu, Samuel Cirigliano, Donald B Kohn

Mol Ther Methods Clin Dev· September 2025

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Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny.
Theodore S Nowicki, Nataly Naser Al Deen, Cole W Peters, Begoña Comin-Anduix, Egmidio Medina, Donald B Kohn

Nat Commun· July 2025

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Synthesis errors in oligonucleotides are propagated into the genome by homology-directed repair.
Stacia K Wyman, Zulema Romero, Seok-Jin Heo, Marian Navarrete, Netravathi Krishnappa, Donald B Kohn

bioRxiv· July 2025

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Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.
David A Williams, Donald B Kohn, Adrian J Thrasher

Mol Ther· May 2025

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Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID.
Eugenio Montini, Luigi Naldini, Claire Booth, Donald B Kohn, Alessandro Aiuti

Mol Ther· May 2025

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Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.
Claire Booth, Julián Sevilla, Elena Almarza, Caroline Y Kuo, Josune Zubicaray, Donald B Kohn

N Engl J Med· May 2025

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Stage-specific CAR-mediated signaling generates naïve-like, TCR-null CAR T cells from induced pluripotent stem cells.
Sang Pil Yoo, Xuegang Yuan, Claire Engstrom, Patrick Chang, Suwen Li, Donald B Kohn

bioRxiv· April 2025

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Making sense of adenosine deaminase variants and their clinical implications.
Eyal Grunebaum, Robyn Loves, Donald B Kohn

J Allergy Clin Immunol· January 2025

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Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy.
Eugenio Montini, Luigi Naldini, Claire Booth, Donald B Kohn, Alessandro Aiuti

Mol Ther· January 2025

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Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease.
Paul G Ayoub, Julia Gensheimer, Lindsay Lathrop, Colin Juett, Jason Quintos, Donald B Kohn

Mol Ther Methods Clin Dev· December 2024

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DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34 cell clonogenic potential.
Christopher R Luthers, Sung-Min Ha, Annika Mittelhauser, Marco Morselli, Joseph D Long, Donald B Kohn

Mol Ther Methods Clin Dev· December 2024

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Myelodysplasia after Lentiviral Gene Therapy.
Donald B Kohn, Claire Booth, Luigi Naldini

N Engl J Med· December 2024

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High symptom burden in female X-linked chronic granulomatous disease carriers.
Mary Ann Miranda, Athanasios Tsalatsanis, Jessica R Trotter, Danielle E Arnold, Jacqueline D Squire, Donald B Kohn

Clin Immunol· November 2024

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Enhancing pediatric access to cell and gene therapies.
Crystal L Mackall, Catherine M Bollard, Nancy Goodman, Casey Carr, Rebecca Gardner, Donald B Kohn

Nat Med· July 2024

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A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease.
Kevyn L Hart, Boya Liu, Devin Brown, Beatriz Campo-Fernandez, Kevin Tam, Donald B Kohn

Mol Ther Methods Clin Dev· June 2024

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