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Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency

Haematologica (Roma), 2018-02, Vol.103 (2), p.e82-e86

2018

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Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients

EMBO molecular medicine, 2017-11, Vol.9 (11), p.1574-1588

2017

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T-Cell Depleted Haploidentical Transplantation in Children With Hematological Malignancies: A Comparison Between CD3+/CD19+ and TCRαβ+/CD19+ Depletion Platforms

Frontiers in oncology, 2022-06, Vol.12, p.884397-884397

2022

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Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia

Current gene therapy, 2016-10, Vol.16 (5), p.338-348

2016

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Missense SLC25A38 variations play an important role in autosomal recessive inherited sideroblastic anemia

Haematologica (Roma), 2011-06, Vol.96 (6), p.808-813

2011

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OPTICAL GENOME MAPPING: A NEW TOOL TO OVERCOME CONVENTIONAL CYTOGENETICS’ LIMITATIONS IN PATIENTS WITH BONE MARROW FAILURE

EJC paediatric oncology, 2023-12, Vol.2, p.100056, Article 100056

2023

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International Forum on Small‐Volume Transfusions in Neonates and Paediatric Patients: Summary

Vox sanguinis, 2023-03, Vol.118 (3), p.223-229

2023

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International Forum on Small‐Volume Transfusions in Neonates and Paediatric Patients: Responses

Vox sanguinis, 2023-03, Vol.118 (3), p.230-251

2023

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Haploidentical Stem Cell Transplantation in Children With Hematological Malignancies Using αβ+ T-Cell Receptor and CD19+ Cell Depleted Grafts: High CD56dim/CD56bright NK Cell Ratio Early Following Transplantation Is Associated With Lower Relapse Incidence and Better Outcome

Frontiers in immunology, 2019-10, Vol.10, p.2504-2504

2019

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Deferasirox and Complex Proximal Tubulopathy. Presentation of two clinical cases

Andes pediatrica : revista Chilena de pediatría, 2021-08, Vol.92 (4), p.584-589

2021

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Mutations in XLF/NHEJ1/Cernunnos gene results in downregulation of telomerase genes expression and telomere shortening

Human molecular genetics, 2017-05, Vol.26 (10), p.1900-1914

2017

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Small pituitary volume and central nervous system anomalies in Fanconi Anemia

Frontiers in endocrinology (Lausanne), 2024-08, Vol.15, p.1385650

2024

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A typical acute lymphoblastic leukemia JAK2 variant, R683G, causes an aggressive form of familial thrombocytosis when germline

Leukemia, 2021-11, Vol.35 (11), p.3295-3298

2021

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CRISPR-Cas9 Technology as a Tool to Target Gene Drivers in Cancer: Proof of Concept and New Opportunities to Treat Chronic Myeloid Leukemia

CRISPR journal, 2021-08, Vol.4 (4), p.519-535

2021

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Smart human resource analytics for happiness management

The Journal of management development, 2023-08, Vol.42 (6), p.514-525

2023

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Letter to the Editor: Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome

Human gene therapy, 2022-02, Vol.33 (3-4), p.213-214

2022

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Priming of hematopoietic progenitor cells by plerixafor and filgrastim in children with previous failure of mobilization with chemotherapy and/or cytokine treatment

Journal of pediatric hematology/oncology, 2012-03, Vol.34 (2), p.146-150

2012

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NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia

Cell stem cell, 2019-11, Vol.25 (5), p.607-621.e7

2019

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Hb Cibeles [α2 CD25(B6) (Gly → Asp)]: a novel alpha chain variant causing alpha-thalassemia

International journal of hematology, 2014-12, Vol.100 (6), p.599-601

2014

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Analysis of clinical outcome and survival in pediatric patients undergoing extracorporeal photopheresis for the treatment of steroid-refractory GVHD

Journal of pediatric hematology/oncology, 2010-11, Vol.32 (8), p.589-593

2010

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Splice donor site sgRNAs enhance CRISPR/Cas9-mediated knockout efficiency

PloS one, 2019-05, Vol.14 (5), p.e0216674-e0216674

2019

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Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia

The Journal of clinical investigation, 2022-08, Vol.132 (15), p.1-16

2022

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Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency

Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients

T-Cell Depleted Haploidentical Transplantation in Children With Hematological Malignancies: A Comparison Between CD3+/CD19+ and TCRαβ+/CD19+ Depletion Platforms

Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia

Missense SLC25A38 variations play an important role in autosomal recessive inherited sideroblastic anemia

Genetic analyses of aplastic anemia and idiopathic pulmonary fibrosis patients with short telomeres, possible implication of DNA-repair genes

OPTICAL GENOME MAPPING: A NEW TOOL TO OVERCOME CONVENTIONAL CYTOGENETICS’ LIMITATIONS IN PATIENTS WITH BONE MARROW FAILURE

International Forum on Small‐Volume Transfusions in Neonates and Paediatric Patients: Summary

International Forum on Small‐Volume Transfusions in Neonates and Paediatric Patients: Responses

Haploidentical Stem Cell Transplantation in Children With Hematological Malignancies Using αβ+ T-Cell Receptor and CD19+ Cell Depleted Grafts: High CD56dim/CD56bright NK Cell Ratio Early Following Transplantation Is Associated With Lower Relapse Incidence and Better Outcome

Deferasirox and Complex Proximal Tubulopathy. Presentation of two clinical cases

Mutations in XLF/NHEJ1/Cernunnos gene results in downregulation of telomerase genes expression and telomere shortening

Small pituitary volume and central nervous system anomalies in Fanconi Anemia

A typical acute lymphoblastic leukemia JAK2 variant, R683G, causes an aggressive form of familial thrombocytosis when germline

CRISPR-Cas9 Technology as a Tool to Target Gene Drivers in Cancer: Proof of Concept and New Opportunities to Treat Chronic Myeloid Leukemia

Smart human resource analytics for happiness management

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

Letter to the Editor: Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome

Priming of hematopoietic progenitor cells by plerixafor and filgrastim in children with previous failure of mobilization with chemotherapy and/or cytokine treatment

Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells

NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia

Hb Cibeles [α2 CD25(B6) (Gly → Asp)]: a novel alpha chain variant causing alpha-thalassemia

Analysis of clinical outcome and survival in pediatric patients undergoing extracorporeal photopheresis for the treatment of steroid-refractory GVHD

Splice donor site sgRNAs enhance CRISPR/Cas9-mediated knockout efficiency

Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia

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