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D

Daniele Canarutto

faculty of medicine and surgery
H指数:16
被引数:1,147

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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
Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters

通过瞬态和门报告基因选择具有预期功能编辑的人类造血干细胞

2026-06-010
OAAI
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome
2024-05-012
PRE
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
2024-02-075
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
Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series
2023-06-242
OAAI
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
2022-10-0159
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
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
2019-05-01155
OAAI
Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis
2018-11-1313
OAAI