Francesca Ferrua

Affiliations
  • San Raffaele Telethon Institute for Gene Therapy and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Publications (36)

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.

The Journal of allergy and clinical immunology
Avec: Emilia Cirillo , Giuliana Giardino , Silvia Ricci , Viviana Moschese , Vassilios Lougaris , Francesca Conti , Chiara Azzari , Federica Barzaghi , Clementina Canessa , Baldassarre Martire , Raffaele Badolato , Laura Dotta , Annarosa Soresina , Caterina Cancrini , Andrea Finocchi , Davide Montin , Roberta Romano , Donato Amodio , Francesca Ferrua , Alberto Tommasini , Lucia Augusta Baselli , Rosa Maria Dellepiane , Agata Polizzi , Luciana Chessa , Antonio Marzollo , Maria Pia Cicalese , Maria Caterina Putti , Andrea Pession , Alessandro Aiuti , Franco Locatelli , Alessandro Plebani , Claudio Pignata

Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.

The Journal of allergy and clinical immunology
Avec: Lucia Sereni , Maria Carmina Castiello , Dario Di Silvestre , Patrizia Della Valle , Chiara Brombin , Francesca Ferrua , Maria Pia Cicalese , Loris Pozzi , Maddalena Migliavacca , Maria Ester Bernardo , Claudio Pignata , Roula Farah , Lucia Dora Notarangelo , Nufar Marcus , Lorella Cattaneo , Marco Spinelli , Stefania Giannelli , Marita Bosticardo , Koen van Rossem , Armando D'Angelo , Alessandro Aiuti , Pierluigi Mauri , Anna Villa

Thymic Epithelium Abnormalities in DiGeorge and Down Syndrome Patients Contribute to Dysregulation in T Cell Development.

Frontiers in immunology
Avec: Genni Enza Marcovecchio , Ileana Bortolomai , Francesca Ferrua , Elena Fontana , Luisa Imberti , Erika Conforti , Donato Amodio , Sonia Bergante , Giulia Macchiarulo , Veronica D'Oria , Francesca Conti , Silvia Di Cesare , Georgia Fousteri , Adriano Carotti , Alessandro Giamberti , Pietro Luigi Poliani , Luigi D Notarangelo , Caterina Cancrini , Anna Villa , Marita Bosticardo

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome.

EMBO molecular medicine
Avec: Valentina Vavassori , Elisabetta Mercuri , Genni E Marcovecchio , Maria C Castiello , Giulia Schiroli , Luisa Albano , Carrie Margulies , Frank Buquicchio , Elena Fontana , Stefano Beretta , Ivan Merelli , Andrea Cappelleri , Paola Mv Rancoita , Vassilios Lougaris , Alessandro Plebani , Maria Kanariou , Arjan Lankester , Francesca Ferrua , Eugenio Scanziani , Cecilia Cotta-Ramusino , Anna Villa , Luigi Naldini , Pietro Genovese

Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report.

Frontiers in immunology
Avec: Sabina Cenciarelli , Valeria Calbi , Federica Barzaghi , Maria Ester Bernardo , Chiara Oltolini , Maddalena Migliavacca , Vera Gallo , Francesca Tucci , Federico Fraschetta , Elena Albertazzi , Elena Sophia Fratini , Giulia Consiglieri , Stefania Giannelli , Francesca Dionisio , Claudia Sartirana , Sara Racca , Chiara Camesasca , Giovanni Peretto , Rita Daverio , Antonio Esposito , Francesco De Cobelli , Paolo Silvani , Marco Rabusin , Andrea Cara , Daria Trabattoni , Stefania Dispinseri , Gabriella Scarlatti , Lorenzo Piemonti , Vito Lampasona , Maria Pia Cicalese , Alessandro Aiuti , Francesca Ferrua

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.

Lancet (London, England)
Avec: Francesca Fumagalli , Valeria Calbi , Maria Grazia Natali Sora , Maria Sessa , Cristina Baldoli , Paola Maria V Rancoita , Francesca Ciotti , Marina Sarzana , Maddalena Fraschini , Alberto Andrea Zambon , Serena Acquati , Daniela Redaelli , Vanessa Attanasio , Simona Miglietta , Fabiola De Mattia , Federica Barzaghi , Francesca Ferrua , Maddalena Migliavacca , Francesca Tucci , Vera Gallo , Ubaldo Del Carro , Sabrina Canale , Ivana Spiga , Laura Lorioli , Salvatore Recupero , Elena Sophia Fratini , Francesco Morena , Paolo Silvani , Maria Rosa Calvi , Marcella Facchini , Sara Locatelli , Ambra Corti , Stefano Zancan , Gigliola Antonioli , Giada Farinelli , Michela Gabaldo , Jesus Garcia-Segovia , Laetitia C Schwab , Gerald F Downey , Massimo Filippi , Maria Pia Cicalese , Sabata Martino , Clelia Di Serio , Fabio Ciceri , Maria Ester Bernardo , Luigi Naldini , Alessandra Biffi , Alessandro Aiuti

Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series.

Biomedicines
Avec: Daniele Canarutto , Chiara Oltolini , Federica Barzaghi , Valeria Calbi , Maddalena Migliavacca , Francesca Tucci , Vera Gallo , Giulia Consiglieri , Francesca Ferrua , Salvatore Recupero , Maria Celia Cervi , Hamoud Al-Mousa , Anna Pituch-Noworolska , Chiara Tassan Din , Paolo Scarpellini , Paolo Silvani , Claudia Fossati , Miriam Casiraghi , Daniela Maria Cirillo , Antonella Castagna , Maria Ester Bernardo , Alessandro Aiuti , Maria Pia Cicalese

Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.

Nature medicine
Avec: Maddalena Migliavacca , Federica Barzaghi , Claudia Fossati , Paola M V Rancoita , Michela Gabaldo , Francesca Dionisio , Stefania Giannelli , Federica Andrea Salerio , Francesca Ferrua , Francesca Tucci , Valeria Calbi , Vera Gallo , Salvatore Recupero , Giulia Consiglieri , Roberta Pajno , Maria Sambuco , Alessio Priolo , Chiara Ferri , Vittoria Garella , Ilaria Monti , Paolo Silvani , Silvia Darin , Miriam Casiraghi , Ambra Corti , Stefano Zancan , Margherita Levi , Daniela Cesana , Filippo Carlucci , Anna Pituch-Noworolska , Dalia AbdElaziz , Ulrich Baumann , Andrea Finocchi , Caterina Cancrini , Saverio Ladogana , Andrea Meinhardt , Isabelle Meyts , Davide Montin , Lucia Dora Notarangelo , Fulvio Porta , Marlène Pasquet , Carsten Speckmann , Polina Stepensky , Alberto Tommasini , Marco Rabusin , Zeynep Karakas , Miguel Galicchio , Lucia Leonardi , Marzia Duse , Sukru Nail Guner , Clelia Di Serio , Fabio Ciceri , Maria Ester Bernardo , Alessandro Aiuti , Maria Pia Cicalese

Gene Modification and Three-Dimensional Scaffolds as Novel Tools to Allow the Use of Postnatal Thymic Epithelial Cells for Thymus Regeneration Approaches.

Stem cells translational medicine
Avec: Ileana Bortolomai , Monica Sandri , Elena Draghici , Elena Fontana , Elisabetta Campodoni , Genni Enza Marcovecchio , Francesca Ferrua , Laura Perani , Antonello Spinelli , Tamara Canu , Marco Catucci , Tiziano Di Tomaso , Lucia Sergi Sergi , Antonio Esposito , Angelo Lombardo , Luigi Naldini , Anna Tampieri , Georg A Hollander , Anna Villa , Marita Bosticardo

Corrigendum: Targeted NGS Platforms for Genetic Screening and Gene Discovery in Primary Immunodeficiencies.

Frontiers in immunology
Avec: Cristina Cifaldi , Immacolata Brigida , Federica Barzaghi , Matteo Zoccolillo , Valentina Ferradini , Davide Petricone , Maria Pia Cicalese , Dejan Lazarevic , Davide Cittaro , Maryam Omrani , Enrico Attardi , Francesca Conti , Alessia Scarselli , Maria Chiriaco , Silvia Di Cesare , Francesco Licciardi , Montin Davide , Francesca Ferrua , Clementina Canessa , Claudio Pignata , Silvia Giliani , Simona Ferrari , Georgia Fousteri , Graziano Barera , Pietro Merli , Paolo Palma , Simone Cesaro , Marco Gattorno , Antonio Trizzino , Viviana Moschese , Loredana Chini , Anna Villa , Chiara Azzari , Andrea Finocchi , Franco Locatelli , Paolo Rossi , Federica Sangiuolo , Alessandro Aiuti , Caterina Cancrini , Gigliola Di Matteo

Urogenital Abnormalities in Adenosine Deaminase Deficiency.

Journal of clinical immunology
Avec: Roberta Pajno , Lucia Pacillo , Salvatore Recupero , Maria P Cicalese , Francesca Ferrua , Federica Barzaghi , Silvia Ricci , Antonio Marzollo , Silvia Pecorelli , Chiara Azzari , Andrea Finocchi , Caterina Cancrini , Gigliola Di Matteo , Gianni Russo , Massimo Alfano , Arianna Lesma , Andrea Salonia , Stuart Adams , Claire Booth , Alessandro Aiuti

Hematopoietic Stem Cell Transplantation for Combined Immunodeficiencies, on Behalf of IEWP-EBMT.

Frontiers in pediatrics
Avec: Benedicte Neven , Francesca Ferrua

A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease.

Journal of clinical immunology
Avec: Maddalena Migliavacca , Luca Basso Ricci , Giada Farinelli , Valeria Calbi , Francesca Tucci , Federica Barzaghi , Francesca Ferrua , Maria Pia Cicalese , Silvia Darin , Lina Raffaella Barzaghi , Fabio Giglio , Jacopo Peccatori , Francesca Fumagalli , Roberto Nicoletti , Stefania Giannelli , Claudia Sartirana , Alessandro Bandiera , Maria Esposito , Raffaella Milani , Benedetta Mazzi , Andrea Finocchi , Sarah Marktel , Andrea Assanelli , Franco Locatelli , Fabio Ciceri , Alessandro Aiuti , Maria Ester Bernardo

Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity.

Blood
Avec: Tanja C Vallée , Jannik S Glasmacher , Hannes Buchner , Peter D Arkwright , Uta Behrends , Anastasia Bondarenko , Michael J Browning , David K Buchbinder , Alessandro Cattoni , Liudmyla Chernyshova , Peter Ciznar , Theresa Cole , Wojciech Czogala , Gregor Dueckers , John David M Edgar , Fatih Erbey , Anders Fasth , Francesca Ferrua , Renata Formankova , Eleonora Gambineri , Andrew R Gennery , Frederick D Goldman , Luis Ignacio Gonzalez-Granado , Carsten Heilmann , Tarja Heiskanen-Kosma , Hanna Juntti , Leena Kainulainen , Hirokazu Kanegane , Neslihan E Karaca , Sara Sebnem Kilic , Christoph Klein , Sylwia Koltan , Irina Kondratenko , Isabelle Meyts , Gulnara M Nasrullayeva , Lucia Dora Notarangelo , Srdjan Pasic , Isabelle Pellier , Claudio Pignata , Siraj Ahmed Misbah , Ansgar S Schulz , Gesmar Rs Segundo , Anna Shcherbina , Mary A Slatter , Robert Sokolic , Pere Soler-Palacin , Polina Stepensky , Joris M van Montfrans , Samppa Ryhänen , Beata Wolska-Kuśnierz , John B Ziegler , Xiaodong Zhao , Alessandro Aiuti , Hans D Ochs , Michael H Albert

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.

The Lancet. Haematology
Avec: Francesca Ferrua , Maria Pia Cicalese , Stefania Galimberti , Stefania Giannelli , Francesca Dionisio , Federica Barzaghi , Maddalena Migliavacca , Maria Ester Bernardo , Valeria Calbi , Andrea Angelo Assanelli , Marcella Facchini , Claudia Fossati , Elena Albertazzi , Samantha Scaramuzza , Immacolata Brigida , Serena Scala , Luca Basso-Ricci , Roberta Pajno , Miriam Casiraghi , Daniele Canarutto , Federica Andrea Salerio , Michael H Albert , Antonella Bartoli , Hermann M Wolf , Rossana Fiori , Paolo Silvani , Salvatore Gattillo , Anna Villa , Luca Biasco , Christopher Dott , Emily J Culme-Seymour , Koenraad van Rossem , Gillian Atkinson , Maria Grazia Valsecchi , Maria Grazia Roncarolo , Fabio Ciceri , Luigi Naldini , Alessandro Aiuti

Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection.

Haematologica
Avec: Francesca Tucci , Vera Gallo , Federica Barzaghi , Francesca Ferrua , Maddalena Migliavacca , Valeria Calbi , Matteo Doglio , Elena Sophia Fratini , Zeynep Karakas , Sukru Guner , Matilde Zambelli , Cristina Parisi , Raffaella Milani , Salvatore Gattillo , Benedetta Mazzi , Chiara Oltolini , Maurizio Barbera , Cristina Baldoli , Daniela Maria Cirillo , Veronica Asnaghi , Cristina De Min , Maria Pia Cicalese , Fabio Ciceri , Alessandro Aiuti , Maria Ester Bernardo

Thymic Epithelial Cell Alterations and Defective Thymopoiesis Lead to Central and Peripheral Tolerance Perturbation in MHCII Deficiency.

Frontiers in immunology
Avec: Francesca Ferrua , Ileana Bortolomai , Elena Fontana , Dario Di Silvestre , Rosita Rigoni , Genni Enza Marcovecchio , Elena Draghici , Francesca Brambilla , Maria Carmina Castiello , Gloria Delfanti , Despina Moshous , Capucine Picard , Tom Taghon , Victoria Bordon , Ansgar S Schulz , Catharina Schuetz , Silvia Giliani , Annarosa Soresina , Andrew R Gennery , Sara Signa , Blachy J Dávila Saldaña , Ottavia M Delmonte , Luigi D Notarangelo , Chaim M Roifman , Pietro Luigi Poliani , Paolo Uva , Pier Luigi Mauri , Anna Villa , Marita Bosticardo

Premature Senescence and Increased Oxidative Stress in the Thymus of Down Syndrome Patients.

Frontiers in immunology
Avec: Genni Enza Marcovecchio , Francesca Ferrua , Elena Fontana , Stefano Beretta , Marco Genua , Ileana Bortolomai , Anastasia Conti , Davide Montin , Maria Teresa Cascarano , Sonia Bergante , Veronica D'Oria , Alessandro Giamberti , Donato Amodio , Caterina Cancrini , Adriano Carotti , Raffaella Di Micco , Ivan Merelli , Marita Bosticardo , Anna Villa

Follicular helper T cell signature of replicative exhaustion, apoptosis, and senescence in common variable immunodeficiency.

European journal of immunology
Avec: Giulia Milardi , Biagio Di Lorenzo , Jolanda Gerosa , Federica Barzaghi , Gigliola Di Matteo , Maryam Omrani , Tatiana Jofra , Ivan Merelli , Matteo Barcella , Matteo Filippini , Anastasia Conti , Francesca Ferrua , Francesco Pozzo Giuffrida , Francesca Dionisio , Patrizia Rovere-Querini , Sarah Marktel , Andrea Assanelli , Simona Piemontese , Immacolata Brigida , Matteo Zoccolillo , Emilia Cirillo , Giuliana Giardino , Maria Giovanna Danieli , Fernando Specchia , Lucia Pacillo , Silvia Di Cesare , Carmela Giancotta , Francesca Romano , Alessandro Matarese , Alfredo Antonio Chetta , Matteo Trimarchi , Andrea Laurenzi , Maurizio De Pellegrin , Silvia Darin , Davide Montin , Maddalena Marinoni , Rosa Maria Dellepiane , Valeria Sordi , Vassilios Lougaris , Angelo Vacca , Raffaella Melzi , Rita Nano , Chiara Azzari , Lucia Bongiovanni , Claudio Pignata , Caterina Cancrini , Alessandro Plebani , Lorenzo Piemonti , Constantinos Petrovas , Raffaella Di Micco , Maurilio Ponzoni , Alessandro Aiuti , Maria Pia Cicalese , Georgia Fousteri

Circulating Hematopoietic Stem/Progenitor Cells subsets contribute to human hematopoietic homeostasis.

Blood
Avec: Pamela Quaranta , Luca Basso-Ricci , Raisa Jofra Jofra Hernández , Guido Pacini , Matteo Maria Naldini , Matteo Barcella , Luca Seffin , Giulia Pais , Giulio Spinozzi , Fabrizio Benedicenti , Carlo Pietrasanta , Jin-Gyu Cheong , Andrea Ronchi , Lorenza Pugni , Francesca Dionisio , Ilaria Monti , Stefania Giannelli , Silvia Darin , Federico Fraschetta , Graziano Barera , Francesca Ferrua , Valeria Calbi , Marco Ometti , Raffaella Di Micco , Fabio Mosca , Steven Zvi Josefowicz , Eugenio Montini , Andrea Calabria , Maria Ester Bernardo , Maria Pia Cicalese , Bernhard Gentner , Ivan Merelli , Alessandro Aiuti , Serena Scala

Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.

Science translational medicine
Avec: Giulia Consiglieri , Francesca Tucci , Maurizio De Pellegrin , Barbara Guerrini , Alessandro Cattoni , Giulia Risca , Stefano Scarparo , Marina Sarzana , Silvia Pontesilli , Renata Mellone , Serena Gasperini , Stefania Galimberti , Paolo Silvani , Chiara Filisetti , Silvia Darin , Giulia Forni , Simona Miglietta , Ludovica Santi , Marcella Facchini , Ambra Corti , Francesca Fumagalli , Maria Pia Cicalese , Valeria Calbi , Maddalena Migliavacca , Federica Barzaghi , Francesca Ferrua , Vera Gallo , Salvatore Recupero , Daniele Canarutto , Matteo Doglio , Lucia Tedesco , Nicola Volpi , Attilio Rovelli , Giancarlo la Marca , Maria Grazia Valsecchi , Stefano Zancan , Fabio Ciceri , Luigi Naldini , Cristina Baldoli , Rossella Parini , Bernhard Gentner , Alessandro Aiuti , Maria Ester Bernardo

Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review.

Frontiers in immunology
Avec: Elena Sophia Fratini , Maddalena Migliavacca , Federica Barzaghi , Claudia Fossati , Stefania Giannelli , Ilaria Monti , Miriam Casiraghi , Francesca Ferrua , Salvatore Recupero , Giulia Consiglieri , Valeria Calbi , Francesca Tucci , Vera Gallo , Maria Ester Bernardo , Sabina Cenciarelli , Monica Palmoni , Margherita Moni , Luca Galimberti , Marzia Duse , Lucia Leonardi , Elena Sieni , Elena Soncini , Fulvio Porta , Lucia Dora Notarangelo , Raffaella De Santis , Saverio Ladogana , Alessandro Aiuti , Maria Pia Cicalese

Interleukin-1 blockade in patients with Wiskott-Aldrich Syndrome: a retrospective multinational case series.

Blood
Avec: Samuele Naviglio , Maria Pia Cicalese , Elizabeth Rivers , Francesca Ferrua , Carmem M S Bonfim , Sabina Cenciarelli , Kai-Ning Cheong , Maura Faraci , Stefano Giardino , Sujal Ghosh , Pamela P Lee , Paula Teixeira Lyra , Roland Meisel , Valentina Sofia , Antimo Tessitore , Alberto Tommasini , Erica Valencic , Tanja C Vallée , Stefano Volpi , Austen J Worth , Marco Rabusin , Michael H Albert , Adrian J Thrasher , Alessandro Aiuti

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.

Bone marrow transplantation
Avec: Francesca Tucci , Marta Frittoli , Federica Barzaghi , Valeria Calbi , Maddalena Migliavacca , Francesca Ferrua , Francesca Fumagalli , Laura Lorioli , Laura Castagnaro , Marcella Facchini , Claudia Fossati , Stefano Zancan , Paola Massariello , Michele Manfredini , Giulia Consiglieri , Daniele Canarutto , Salvatore Recupero , Francesco Calzatini , Miriam Casiraghi , Silvia Darin , Gigliola Antonioli , Roberto Miniero , Rossana Fiori , Paolo Silvani , Matilde Zambelli , Sarah Marktel , Salvatore Gattillo , Raffaella Milani , Luca Santoleri , Fabio Ciceri , Alessandra Biffi , Maria Pia Cicalese , Maria Ester Bernardo , Alessandro Aiuti

Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort.

The Journal of allergy and clinical immunology
Avec: Arjan C Lankester , Benedicte Neven , Nizar Mahlaoui , Erik G J von Asmuth , Virginie Courteille , Mikael Alligon , Michael H Albert , Isabelle Badell Serra , Peter Bader , Dmitry Balashov , Rita Beier , Yves Bertrand , Stephane Blanche , Victoria Bordon , Robbert G Bredius , Andrew Cant , Marina Cavazzana , Cristina Diaz-de-Heredia , Figen Dogu , Karoline Ehlert , Natacha Entz-Werle , Anders Fasth , Francesca Ferrua , Alina Ferster , Renata Formankova , Wilhelm Friedrich , Marta Gonzalez-Vicent , Jolanta Gozdzik , Tayfun Güngör , Manfred Hoenig , Aydan Ikinciogullari , Krzysztof Kalwak , Savas Kansoy , Alphan Kupesiz , Arnalda Lanfranchi , Caroline A Lindemans , Roland Meisel , Gerard Michel , Nuno A A Miranda , Jose Moraleda , Despina Moshous , Herbert Pichler , Kanchan Rao , Petr Sedlacek , Mary Slatter , Elena Soncini , Carsten Speckmann , Mikael Sundin , Amos Toren , Kim Vettenranta , Austen Worth , Mehmet A Yeşilipek , Marco Zecca , Fulvio Porta , Ansgar Schulz , Paul Veys , Alain Fischer , Andrew R Gennery

Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4

The EMBO journal
Avec: Daniele Canarutto , Claudia Asperti , Valentina Vavassori , Simona Porcellini , Elisabetta Rovelli , Marianna Paulis , Samuele Ferrari , Angelica Varesi , Martina Fiumara , Aurelien Jacob , Lucia Sergi Sergi , Ilaria Visigalli , Francesca Ferrua , Luis Ignacio González-Granado , Vassilios Lougaris , Andrea Finocchi , Anna Villa , Marina Radrizzani , Luigi Naldini

Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated

Molecular therapy. Methods & clinical development
Avec: Claudia Asperti , Daniele Canarutto , Simona Porcellini , Francesca Sanvito , Francesca Cecere , Valentina Vavassori , Samuele Ferrari , Elisabetta Rovelli , Luisa Albano , Aurelien Jacob , Lucia Sergi Sergi , Elisa Montaldo , Francesca Ferrua , Luis Ignacio González-Granado , Vassilios Lougaris , Raffaele Badolato , Andrea Finocchi , Anna Villa , Marina Radrizzani , Luigi Naldini

Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency.

Frontiers in immunology
Avec: Federica Barzaghi , Maria Pia Cicalese , Matteo Zoccolillo , Immacolata Brigida , Matteo Barcella , Ivan Merelli , Claudia Sartirana , Monica Zanussi , Valeria Calbi , Maria Ester Bernardo , Francesca Tucci , Maddalena Migliavacca , Fabio Giglio , Matteo Doglio , Daniele Canarutto , Francesca Ferrua , Giulia Consiglieri , Giulia Prunotto , Francesco Saettini , Sonia Bonanomi , Patrizia Rovere-Querini , Giulia Di Colo , Tatiana Jofra , Georgia Fousteri , Federica Penco , Marco Gattorno , Michael S Hershfield , Lucia Bongiovanni , Maurilio Ponzoni , Sarah Marktel , Raffaella Milani , Jacopo Peccatori , Fabio Ciceri , Alessandra Mortellaro , Alessandro Aiuti

Hematopoietic stem cell transplantation for CD40 ligand deficiency: Results from an EBMT/ESID-IEWP-SCETIDE-PIDTC study.

The Journal of allergy and clinical immunology
Avec: Francesca Ferrua , Stefania Galimberti , Virginie Courteille , Mary Anne Slatter , Claire Booth , Despina Moshous , Benedicte Neven , Stephane Blanche , Marina Cavazzana , Alexandra Laberko , Anna Shcherbina , Dmitry Balashov , Elena Soncini , Fulvio Porta , Hamoud Al-Mousa , Bandar Al-Saud , Hasan Al-Dhekri , Rand Arnaout , Renata Formankova , Yves Bertrand , Andrzej Lange , Joanne Smart , Beata Wolska-Kusnierz , Victor M Aquino , Christopher C Dvorak , Anders Fasth , Fanny Fouyssac , Carsten Heilmann , Manfred Hoenig , Catharina Schuetz , Jadranka Kelečić , Robbert G M Bredius , Arjan C Lankester , Caroline A Lindemans , Felipe Suarez , Kathleen E Sullivan , Michael H Albert , Krzysztof Kałwak , Vincent Barlogis , Monica Bhatia , Victoria Bordon , Wojciech Czogala , Laura Alonso , Figen Dogu , Jolanta Gozdzik , Aydan Ikinciogullari , Gergely Kriván , Per Ljungman , Isabelle Meyts , Peter Mustillo , Angela R Smith , Carsten Speckmann , Mikael Sundin , Steven John Keogh , Peter John Shaw , Jaap Jan Boelens , Ansgar S Schulz , Petr Sedlacek , Paul Veys , Nizar Mahlaoui , Ales Janda , E Graham Davies , Alain Fischer , Morton J Cowan , Andrew Richard Gennery , Auteur inconnu

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.

The New England journal of medicine
Avec: Bernhard Gentner , Francesca Tucci , Stefania Galimberti , Francesca Fumagalli , Maurizio De Pellegrin , Paolo Silvani , Chiara Camesasca , Silvia Pontesilli , Silvia Darin , Francesca Ciotti , Marina Sarzana , Giulia Consiglieri , Chiara Filisetti , Giulia Forni , Laura Passerini , Daniela Tomasoni , Daniela Cesana , Andrea Calabria , Giulio Spinozzi , Maria-Pia Cicalese , Valeria Calbi , Maddalena Migliavacca , Federica Barzaghi , Francesca Ferrua , Vera Gallo , Simona Miglietta , Erika Zonari , Patali S Cheruku , Claudia Forni , Marcella Facchini , Ambra Corti , Michela Gabaldo , Stefano Zancan , Serena Gasperini , Attilio Rovelli , Jaap-Jan Boelens , Simon A Jones , Robert Wynn , Cristina Baldoli , Eugenio Montini , Silvia Gregori , Fabio Ciceri , Maria G Valsecchi , Giancarlo la Marca , Rossella Parini , Luigi Naldini , Alessandro Aiuti , Maria-Ester Bernardo , Auteur inconnu

Peripheral blood stem and progenitor cell collection in pediatric candidates for

Molecular therapy. Methods & clinical development
Avec: Daniele Canarutto , Francesca Tucci , Salvatore Gattillo , Matilde Zambelli , Valeria Calbi , Bernhard Gentner , Francesca Ferrua , Sarah Marktel , Maddalena Migliavacca , Federica Barzaghi , Giulia Consiglieri , Vera Gallo , Francesca Fumagalli , Paola Massariello , Cristina Parisi , Gianluca Viarengo , Elena Albertazzi , Paolo Silvani , Raffaella Milani , Luca Santoleri , Fabio Ciceri , Maria Pia Cicalese , Maria Ester Bernardo , Alessandro Aiuti

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.

BMJ case reports
Avec: Maria Pia Cicalese , Francesca Ferrua , Federica Barzaghi , Federica Cerri , Matteo Moro , Alessandro Aiuti , Paolo Silvani

Wiskott-Aldrich syndrome: Oral findings and microbiota in children and review of the literature.

Clinical and experimental dental research
Avec: Alessandra Lucchese , Sabina Cenciarelli , Maurizio Manuelli , Marta Marcolina , Federica Barzaghi , Valeria Calbi , Maddalena Migliavacca , Maria Ester Bernardo , Francesca Tucci , Vera Gallo , Federico Fraschetta , Silvia Darin , Miriam Casiraghi , Alessandro Aiuti , Francesca Ferrua , Maria Pia Cicalese

Neonatal alloimmune neutropenia: diagnosis and management of 31 Italian patients.

Blood transfusion = Trasfusione del sangue
Avec: Alessandra Cattaneo , Manuela Liguori , Elena Trombetta , Ferruccio Ceriotti , Lorenza Pugni , Andrea Ronchi , Giulia Carracchia , Lucia D Notarangelo , Francesca Ferrua , Federica Barzaghi , Chiara Giovanettoni , Gianvincenzo Zuccotti , Emilia Cirillo , Claudio Pignata , Federica Meroni , Anna Maietta , Piero Farruggia , Laura Porretti

Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.

Nature communications
Avec: Serena Scala , Francesca Ferrua , Luca Basso-Ricci , Francesca Dionisio , Maryam Omrani , Pamela Quaranta , Raisa Jofra Hernandez , Luca Del Core , Fabrizio Benedicenti , Ilaria Monti , Stefania Giannelli , Federico Fraschetta , Silvia Darin , Elena Albertazzi , Stefania Galimberti , Eugenio Montini , Andrea Calabria , Maria Pia Cicalese , Alessandro Aiuti

Long-term lineage commitment in hematopoietic stem cell gene therapy.

Nature
Avec: Andrea Calabria , Giulio Spinozzi , Daniela Cesana , Elena Buscaroli , Fabrizio Benedicenti , Giulia Pais , Francesco Gazzo , Serena Scala , Maria Rosa Lidonnici , Samantha Scaramuzza , Alessandra Albertini , Simona Esposito , Francesca Tucci , Daniele Canarutto , Maryam Omrani , Fabiola De Mattia , Francesca Dionisio , Stefania Giannelli , Sarah Marktel , Francesca Fumagalli , Valeria Calbi , Sabina Cenciarelli , Francesca Ferrua , Bernhard Gentner , Giulio Caravagna , Fabio Ciceri , Luigi Naldini , Giuliana Ferrari , Alessandro Aiuti , Eugenio Montini

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Alessandro Aiuti 25 collaborations
Maria Pia Cicalese 21 collaborations
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