Last Updated: 09/26/2026
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Found 12 publications
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.
Journal: Obstetrical & gynecological survey
Published: December 30, 2025
Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia.
Journal: Frontiers in pediatrics
Published: May 09, 2025
Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.
Journal: The New England journal of medicine
Published: April 30, 2025
Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV.
Journal: European journal of haematology
Published: April 20, 2025
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.
Journal: Lancet (London, England)
Published: March 12, 2024
Busulfan and subsequent malignancy: An evidence-based risk assessment.
Journal: Pediatric blood & cancer
Published: August 18, 2023
Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Journal: Haematologica
Published: November 16, 2022
Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD.
Journal: American journal of hematology
Published: September 30, 2021
Donor-Host Lineage-Specific Chimerism Monitoring and Analysis in Pediatric Patients Following Allogeneic Stem Cell Transplantation: Influence of Pretransplantation Variables and Correlation with Post-Transplantation Outcomes.
Journal: Transplantation and cellular therapy
Published: February 18, 2021
Natural gene therapy by reverse mosaicism leads to improved hematology in Fanconi anemia patients.
Journal: American journal of hematology
Published: February 17, 2021
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Journal: Molecular therapy. Methods & clinical development
Published: October 28, 2020
Mosaicism in Fanconi anemia: concise review and evaluation of published cases with focus on clinical course of blood count normalization.
Journal: Annals of hematology
Published: July 25, 2019
Showing 1-12 of 12
Last Updated: 09/26/2026