Susana Navarro
Madrid, MD 28040, Spain
Last Updated: 09/26/2026
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Found 25 publications
The potential of gene therapy to rescue telomeric and extratelomeric defects in X-linked dyskeratosis congenita.
Journal: Molecular therapy. Advances
Published: January 21, 2026
Case Report: Eltrombopag in mosaic and gene therapy-treated patients with Fanconi anemia.
Journal: Frontiers in pediatrics
Published: May 09, 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
TKI-mediated inhibition of NLRP1 inflammasome restores erythropoiesis in DBA syndrome.
Journal: EMBO molecular medicine
Published: March 05, 2025
Prognostic significance of mutation type and chromosome fragility in Fanconi anemia.
Journal: American journal of hematology
Published: July 24, 2024
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
Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Journal: Haematologica
Published: November 16, 2022
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes.
Journal: Molecular therapy. Methods & clinical development
Published: February 21, 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
Last Updated: 09/26/2026