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Francesca Ferrua

vita-salute san raffaele university
H指数:25
被引数:3,560

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Busulfan exposure variability and acute toxicity in pediatric patients undergoing hematopoietic stem cell gene therapy

接受造血干细胞基因治疗的儿童患者的白消安暴露变异性和急性毒性

2026-07-260
OAAI
The Homozygous p.(Arg215Ter) Variant in XRCC2 Is Associated With Atypical Fanconi Anemia Without Major Hematological Abnormalities in Childhood

XRCC2基因同源p.(Arg215Ter)变异与无儿童期主要血液学异常的不典型范可尼贫血相关

2026-01-010
OAAI
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome
2024-05-012
PRE
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
2024-02-145
OA
Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1
2023-11-022
OAAI
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review
2023-06-263
OAAI
Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series
2023-06-242
OAAI
Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency
2022-09-294
OAAI
A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease
2022-08-101
OAAI
Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort

严重联合免疫缺陷的造血细胞移植: SCETIDE 2006 2014的欧洲队列

2022-05-0159
OA
Thymic Epithelial Cell Alterations and Defective Thymopoiesis Lead to Central and Peripheral Tolerance Perturbation in MHCII Deficiency
2021-06-156
OAAI
Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200
2021-02-011
PREAI
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome
2021-01-2137
OAAI
Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report
2020-11-249
OAAI
Consensus of the Italian Primary Immunodeficiency Network on transition management from pediatric to adult care in patients affected with childhood-onset inborn errors of immunity

意大利初级免疫缺陷网络关于儿童期先天性免疫缺陷患者从儿科到成人护理过渡管理的共识

2020-11-0114
OAAI
Urogenital Abnormalities in Adenosine Deaminase Deficiency
2020-04-199
OAAI