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Vera Gallo

pediatric immunohematology unit and bmt program
H指数:18
被引数:1,352

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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
Effects of atidarsagene autotemcel gene therapy on peripheral nerves in late-infantile metachromatic leukodystrophy

阿蒂达萨基因自体细胞基因疗法对晚婴儿型异染性脑白质营养不良外周神经的影响

BRAIN
IF11.7
2025-09-012
PRE
Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognition, language, and speech and slows brain demyelination and atrophy in early-onset metachromatic leukodystrophy
2025-02-010
PRE
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
Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD)
2024-02-010
PRE
Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up
2024-02-010
PRE
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
Long-term clinical outcomes of atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up
2023-02-010
PRE
Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use
2023-02-010
PRE
Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy
2021-02-012
PREAI
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
Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report
2020-11-249
OAAI
Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guerin infection
2020-08-1318
OAAI
Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients
2020-02-0110
PREAI
Rituximab Unveils Hypogammaglobulinemia and Immunodeficiency in Children with Autoimmune Cytopenia

利妥昔单抗揭示了自身免疫性血细胞减少症儿童的低丙种球蛋白血症和免疫缺陷

2020-01-0144
PREAI