Publications by authors named "Jonathan D Schwartz"

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Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Paula Río, Josune Zubicaray, Susana Navarro, Eva Gálvez, Rebeca Sánchez-Domínguez, Jonathan D Schwartz

Lancet· December 2025

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A posterior fossa mass in a 4-year-old female.
Vy Huynh, M Adelita Vizcaino, Jonathan D Schwartz, J Zachary Wilson, David J Daniels

Brain Pathol· 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, Jonathan D Schwartz

Obstet Gynecol Surv· November 2025

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Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV.
June Iriondo, Josune Zubicaray, Paula Río, Albert Català, María Solsona, Jonathan D Schwartz

Eur J Haematol· October 2025

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Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia.
Josune Zubicaray, June Iriondo, Elena Sebastián, Alejandro Sanz, Paula Rio, Jonathan D Schwartz

Front Pediatr· September 2025

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Characterizing the molecular and spatial heterogeneity of midline gliomas in adults: a single institution analysis.
Bryan J Neth, Robert M Kraft, Kathryn L Eschbacher, Derek R Johnson, Paul A Decker, Jonathan D Schwartz

J Neurooncol· June 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, Jonathan D Schwartz

N Engl J Med· May 2025

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Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease.
Barry Greenberg, Matthew Taylor, Eric Adler, Steven Colan, David Ricks, Jonathan D Schwartz

N Engl J Med· March 2025

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Memantine to Reduce Cognitive Impairment After Radiation in Children: A Pilot Study Evaluating the Feasibility of Memantine in Reducing Cognitive Impairment in Pediatric Patients after Radiation Therapy for Central Nervous System Tumors.
Elizabeth L McKone, William G Breen, Nathan R Foster, Aaron W Bogan, Margaret A Connors, Jonathan D Schwartz

Int J Radiat Oncol Biol Phys· November 2024

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Busulfan and subsequent malignancy: An evidence-based risk assessment.
Janel R Long-Boyle, Donald B Kohn, Ami J Shah, Sueli Marques Spencer, Julian Sevilla, Jonathan D Schwartz

Pediatr Blood Cancer· January 2024

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Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies.
Stephanie M Salabarria, Manuela Corti, Kirsten E Coleman, Megan B Wichman, Julie A Berthy, Jonathan D Schwartz

J Clin Invest· January 2024

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Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Miren Lasaga, Paula Río, Amaia Vilas-Zornoza, Nuria Planell, Susana Navarro, Jonathan D Schwartz

Haematologica· October 2023

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Predicting disease progression and the need for tumor-directed treatment in tectal plate gliomas.
Megan M J Bauman, Max B Giesken, Ryan M Naylor, Gesina F Keating, Jonathan D Schwartz

J Neurosurg Pediatr· September 2023

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Copeptin levels in hospitalized infants and children with suspected vasopressin-dependent disorders: a case series.
Alaa Al Nofal, Christian Hanna, Aida N Lteif, Siobhan T Pittock, Jonathan D Schwartz

J Pediatr Endocrinol Metab· May 2023

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Advances in NK cell therapy for brain tumors.
Jawad Fares, Zachary B Davis, Julian S Rechberger, Stephanie A Toll, Jonathan D Schwartz

NPJ Precis Oncol· February 2023

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Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.
Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez, Gayatri Rao, Jonathan D Schwartz

Mol Ther Methods Clin Dev· September 2022

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Findings on Optical Coherence Tomography in Malignant Infantile Osteopetrosis.
Giovanni A Campagna, Leila Chew, Moritz Pettenkofer, Eileen Nicoletti, Jonathan D Schwartz

Ophthalmic Surg Lasers Imaging Retina· July 2022

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Drug delivery across the blood-brain barrier for the treatment of pediatric brain tumors - An update.
Erica A Power, Julian S Rechberger, Sumit Gupta, Jonathan D Schwartz, David J Daniels

Adv Drug Deliv Rev· June 2022

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Mitapivat versus Placebo for Pyruvate Kinase Deficiency.
Ami J Shah, Jonathan D Schwartz, Jose C Segovia

N Engl J Med· June 2022

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Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD.
Jonathan D Schwartz, Wilma Barcellini, Rachel F Grace, Paola Bianchi, Alberto Zanella

Am J Hematol· March 2022

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Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome.
Julián Sevilla, June Iriondo, Elena Sebastian, Marta Gonzalez-Vicent, Jonathan D Schwartz

Hum Gene Ther· February 2022

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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Susana Navarro, Oscar Quintana-Bustamante, Rebeca Sanchez-Dominguez, Sergio Lopez-Manzaneda, Isabel Ojeda-Perez, Jonathan D Schwartz

Mol Ther Methods Clin Dev· September 2021

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Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients.
María José Ramírez, Roser Pujol, Juan Pablo Trujillo-Quintero, Jordi Minguillón, Massimo Bogliolo, Jonathan D Schwartz

Am J Hematol· August 2021

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Precision Medicine in Pediatric Bithalamic Glioma: Significance of the EGFR exon 20 Insertion Mutation.
Anshit Goyal, Cody L Nesvick, Aditya Raghunathan, Jonathan D Schwartz, David J Daniels

World Neurosurg· May 2021

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Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal.
Ilana Moscatelli, Elena Almarza, Axel Schambach, David Ricks, Ansgar Schulz, Jonathan D Schwartz

Mol Ther Methods Clin Dev· March 2021

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