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Andrea Assanelli
Researcher at Vita-Salute San Raffaele University
Publications - 89
Citations - 4652
Andrea Assanelli is an academic researcher from Vita-Salute San Raffaele University. The author has contributed to research in topics: Hematopoietic stem cell transplantation & Transplantation. The author has an hindex of 24, co-authored 77 publications receiving 3785 citations. Previous affiliations of Andrea Assanelli include Università telematica San Raffaele.
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Journal ArticleDOI
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Alessandra Biffi,Eugenio Montini,Laura Lorioli,Martina Cesani,Francesca Fumagalli,Tiziana Plati,Cristina Baldoli,Sabata Martino,Andrea Calabria,Sabrina Canale,Fabrizio Benedicenti,Giuliana Vallanti,Luca Biasco,Simone Leo,Nabil Kabbara,Gianluigi Zanetti,William B. Rizzo,Nalini Mehta,Maria Pia Cicalese,Miriam Casiraghi,Jaap Jan Boelens,Ubaldo Del Carro,David J. Dow,Manfred Schmidt,Andrea Assanelli,Victor Neduva,Clelia Di Serio,Elia Stupka,Jason P. Gardner,Christof von Kalle,Claudio Bordignon,Claudio Bordignon,Fabio Ciceri,Attilio Rovelli,Maria Grazia Roncarolo,Alessandro Aiuti,Maria Sessa,Luigi Naldini +37 more
TL;DR: The reconstitution of ARSA activity in the cerebrospinal fluid and the arrested progression of neurodegenerative disease in the three treated patients demonstrate that the transplanted cells, or their progeny, can seed the nervous system and deliver therapeutic levels of active enzyme.
Journal ArticleDOI
Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
Alessandro Aiuti,Luca Biasco,Samantha Scaramuzza,Francesca Ferrua,Maria Pia Cicalese,Cristina Baricordi,Francesca Dionisio,Andrea Calabria,Stefania Giannelli,Maria Carmina Castiello,Marita Bosticardo,Costanza Evangelio,Andrea Assanelli,Miriam Casiraghi,Sara Di Nunzio,Luciano Callegaro,Claudia Benati,Paolo Rizzardi,Danilo Pellin,Clelia Di Serio,Manfred G. Schmidt,Christof von Kalle,Jason P. Gardner,Nalini Mehta,Victor Neduva,David J. Dow,Anne Galy,Roberto Miniero,Andrea Finocchi,Ayse Metin,Pinaki P. Banerjee,Jordan S. Orange,Stefania Galimberti,Maria Grazia Valsecchi,Alessandra Biffi,Eugenio Montini,Anna Villa,Fabio Ciceri,Maria Grazia Roncarolo,Luigi Naldini +39 more
TL;DR: A clinical protocol based on lentiviral vector (LV) gene transfer into autologous hematopoietic stem/progenitor cells (HSCs) resulted in robust, stable, and long-term engraftment of gene-corrected HSCs in the patients’ bone marrow, and the findings support the use of LV gene therapy to treat patients with WAS.
Journal ArticleDOI
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
Maria Sessa,Laura Lorioli,Francesca Fumagalli,Serena Acquati,Daniela Redaelli,Cristina Baldoli,Sabrina Canale,Ignazio Diego Lopez,Francesco Morena,Andrea Calabria,Rossana Fiori,Paolo Silvani,Paola M.V. Rancoita,Michela Gabaldo,Fabrizio Benedicenti,Gigliola Antonioli,Andrea Assanelli,Maria Pia Cicalese,Ubaldo Del Carro,Maria Grazia Natali Sora,Sabata Martino,Angelo Quattrini,Eugenio Montini,Clelia Di Serio,Fabio Ciceri,Maria Grazia Roncarolo,Alessandro Aiuti,Luigi Naldini,Alessandra Biffi +28 more
TL;DR: The authors' ad-hoc findings provide preliminary evidence of safety and therapeutic benefit of HSC-GT, which resulted in protection from CNS demyelination in eight patients and, in at least three patients, amelioration of peripheral nervous system abnormalities, with signs of remyelinated at both sites.
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The first case of COVID-19 treated with the complement C3 inhibitor AMY-101.
Sara Mastaglio,Annalisa Ruggeri,Antonio M. Risitano,Piera Angelillo,Despina Yancopoulou,Dimitrios C. Mastellos,Markus Huber-Lang,Simona Piemontese,Andrea Assanelli,Cecilia Garlanda,John D. Lambris,Fabio Ciceri +11 more
TL;DR: The clinical course of a patient with severe ARDS due to COVID-19 pneumonia who was safely and successfully treated with the compstatin-based complement C3 inhibitor AMY-101 is reported.
Journal ArticleDOI
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.
Sarah Marktel,Samantha Scaramuzza,Maria Pia Cicalese,Fabio Giglio,Stefania Galimberti,Maria Rosa Lidonnici,Valeria Calbi,Andrea Assanelli,Maria Ester Bernardo,Claudia Rossi,Andrea Calabria,Raffaella Milani,Salvatore Gattillo,Fabrizio Benedicenti,Giulio Spinozzi,Annamaria Aprile,Alessandra Bergami,Miriam Casiraghi,Giulia Consiglieri,Nicoletta Masera,Emanuela D'Angelo,Nadia Mirra,Raffaella Origa,Immacolata Tartaglione,Silverio Perrotta,Robert Winter,Milena Coppola,Gianluca Viarengo,Luca Santoleri,Giovanna Graziadei,Michela Gabaldo,Maria Grazia Valsecchi,Eugenio Montini,Luigi Naldini,Maria Domenica Cappellini,Fabio Ciceri,Alessandro Aiuti,Giuliana Ferrari +37 more
TL;DR: In a phase 1/2 clinical trial, gene therapy with autologous hematopoietic stem cells significantly reduced transfusion requirement in adults and children with transfusion dependent ß-thalassemia.