Publications by authors named "Francesca Ferrua"

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Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET).
Annarosa Soresina, Roberto Rondelli, Lucia Dora Notarangelo, Franco Locatelli, Alessandro Aiuti, Francesca Ferrua

EClinicalMedicine· June 2025

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Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
Francesca Fumagalli, Valeria Calbi, Vera Gallo, Alberto Andrea Zambon, Salvatore Recupero, Francesca Ferrua

N Engl J Med· April 2025

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Long-term lineage commitment in haematopoietic stem cell gene therapy.
Andrea Calabria, Giulio Spinozzi, Daniela Cesana, Elena Buscaroli, Fabrizio Benedicenti, Francesca Ferrua

Nature· December 2024

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Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series.
Samuele Naviglio, Maria Pia Cicalese, Elizabeth Rivers, Francesca Ferrua, Carmem Bonfim

Blood· October 2024

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Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival.
Tanja C Vallée, Jannik S Glasmacher, Hannes Buchner, Peter D Arkwright, Uta Behrends, Francesca Ferrua

Blood· June 2024

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Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.
Pamela Quaranta, Luca Basso-Ricci, Raisa Jofra Hernandez, Guido Pacini, Matteo Maria Naldini, Francesca Ferrua

Blood· May 2024

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Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
Giulia Consiglieri, Francesca Tucci, Maurizio De Pellegrin, Barbara Guerrini, Alessandro Cattoni, Francesca Ferrua

Sci Transl Med· May 2024

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Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.
Maddalena Migliavacca, Federica Barzaghi, Claudia Fossati, Paola M V Rancoita, Michela Gabaldo, Francesca Ferrua

Nat Med· February 2024

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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.
Daniele Canarutto, Claudia Asperti, Valentina Vavassori, Simona Porcellini, Elisabetta Rovelli, Francesca Ferrua

EMBO J· December 2023

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Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated and .
Claudia Asperti, Daniele Canarutto, Simona Porcellini, Francesca Sanvito, Francesca Cecere, Francesca Ferrua

Mol Ther Methods Clin Dev· September 2023

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Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review.
Elena Sophia Fratini, Maddalena Migliavacca, Federica Barzaghi, Claudia Fossati, Stefania Giannelli, Francesca Ferrua

Front Immunol· July 2023

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Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.
Daniele Canarutto, Chiara Oltolini, Federica Barzaghi, Valeria Calbi, Maddalena Migliavacca, Francesca Ferrua

Biomedicines· June 2023

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Neonatal alloimmune neutropenia: diagnosis and management of 31 Italian patients.
Alessandra Cattaneo, Manuela Liguori, Elena Trombetta, Ferruccio Ceriotti, Lorenza Pugni, Francesca Ferrua

Blood Transfus· May 2023

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Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.
Serena Scala, Francesca Ferrua, Luca Basso-Ricci, Francesca Dionisio, Maryam Omrani

Nat Commun· May 2023

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Overhydrated hereditary stomatocytosis: A rare cause of familiar persistent macrocytosis due to SLC4A1 variants.
Anna Paola Marcello, Camilla Visconti, Cristina Vercellati, Anna Zaninoni, Paola Bianchi, Francesca Ferrua

Pediatr Blood Cancer· April 2023

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A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease.
Maddalena Migliavacca, Luca Basso Ricci, Giada Farinelli, Valeria Calbi, Francesca Tucci, Francesca Ferrua

J Clin Immunol· November 2022

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Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency.
Federica Barzaghi, Maria Pia Cicalese, Matteo Zoccolillo, Immacolata Brigida, Matteo Barcella, Francesca Ferrua

Front Immunol· October 2022

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Follicular helper T cell signature of replicative exhaustion, apoptosis, and senescence in common variable immunodeficiency.
Giulia Milardi, Biagio Di Lorenzo, Jolanda Gerosa, Federica Barzaghi, Gigliola Di Matteo, Francesca Ferrua

Eur J Immunol· July 2022

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Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort.
Arjan C Lankester, Benedicte Neven, Nizar Mahlaoui, Erik G J von Asmuth, Virginie Courteille, Francesca Ferrua

J Allergy Clin Immunol· May 2022

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A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation.
Giulia Consiglieri, Francesca Ferrua, , Alessandro Aiuti, Maria Pia Cicalese

J Clin Immunol· February 2022

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Correction to: A Case of Two Adult Brothers with Wiskott‑Aldrich Syndrome, One Treated with Gene Therapy and One with HLA‑Identical Hematopoietic Stem Cell Transplantation.
Giulia Consiglieri, Francesca Ferrua, , Alessandro Aiuti, Maria Pia Cicalese

J Clin Immunol· February 2022

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Third cranial nerve palsy in an 88-year-old man after SARS-CoV-2 mRNA vaccination: change of injection site and type of vaccine resulted in an uneventful second dose with humoral immune response.
Maria Pia Cicalese, Francesca Ferrua, Federica Barzaghi, Federica Cerri, Matteo Moro

BMJ Case Rep· February 2022

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Wiskott-Aldrich syndrome: Oral findings and microbiota in children and review of the literature.
Alessandra Lucchese, Sabina Cenciarelli, Maurizio Manuelli, Marta Marcolina, Federica Barzaghi, Francesca Ferrua

Clin Exp Dent Res· February 2022

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Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, Maria Sessa, Cristina Baldoli, Francesca Ferrua

Lancet· January 2022

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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
Bernhard Gentner, Francesca Tucci, Stefania Galimberti, Francesca Fumagalli, Maurizio De Pellegrin, Francesca Ferrua

N Engl J Med· November 2021

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