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Alessandra Biffi
Researcher at University of Padua
Publications - 155
Citations - 7756
Alessandra Biffi is an academic researcher from University of Padua. The author has contributed to research in topics: Genetic enhancement & Metachromatic leukodystrophy. The author has an hindex of 35, co-authored 130 publications receiving 6150 citations. Previous affiliations of Alessandra Biffi include Harvard University & Dana Corporation.
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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
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Jinkuk Kim,Chunguang Hu,Christelle Moufawad El Achkar,Lauren E. Black,Julie Douville,Austin Larson,Mary K. Pendergast,Sara F. Goldkind,Eunjung Lee,Eunjung Lee,Ashley Kuniholm,Aubrie Soucy,Jai Vaze,Nandkishore R. Belur,Kristina Fredriksen,Iva Stojkovska,Alla V. Tsytsykova,Myriam Armant,Renata L. DiDonato,Jaejoon Choi,Laura Cornelissen,Luis M. Pereira,Erika F. Augustine,Casie A. Genetti,Kira A. Dies,Brenda Barton,Brenda Barton,Lucinda Williams,Lucinda Williams,Benjamin D. Goodlett,Bobbie L. Riley,Amy Pasternak,Emily Berry,Kelly A. Pflock,Stephen Chu,Chantal Reed,Kimberly Tyndall,Pankaj B. Agrawal,Alan H. Beggs,P. Ellen Grant,David K. Urion,Richard O. Snyder,Susan E. Waisbren,Annapurna Poduri,Peter J. Park,Al Patterson,Alessandra Biffi,Joseph R. Mazzulli,Olaf Bodamer,Olaf Bodamer,Charles B. Berde,Timothy W. Yu,Timothy W. Yu +52 more
TL;DR: This study describes how molecular diagnosis of a rare, fatal neurodegenerative condition led to the rational design, testing, and manufacture of milasen, a splice-modulating antisense oligonucleotide drug tailored to a particular patient.
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.
Journal ArticleDOI
Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters
TL;DR: Synthetic bidirectional promoters are developed that mediate coordinate transcription of two mRNAs in a ubiquitous or a tissue-specific manner that enabled efficient dual gene transfer in several tissues in vivo after direct delivery or transgenesis, and in a human gene therapy model.