In vivo dynamics of human hematopoietic stem cells: novel concepts and future directions

Por Scala, Serena, Aiuti, Alessandro
Publicado no Blood Adv (2019)

Clinical Applications of Gene Therapy for Primary Immunodeficiencies

Por Cicalese, Maria Pia, Aiuti, Alessandro
Publicado no Hum Gene Ther (2015)

Retroviral Integrations in Gene Therapy Trials

Por Biasco, Luca, Baricordi, Cristina, Aiuti, Alessandro
Publicado em 2012

Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

Por Aiuti, Alessandro, Roncarolo, Maria Grazia, Naldini, Luigi
Publicado no EMBO Mol Med (2017)

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

Por Staal, Frank J. T., Aiuti, Alessandro, Cavazzana, Marina
Publicado no Front Pediatr (2019)

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

Por Tucci, Francesca, Scaramuzza, Samantha, Aiuti, Alessandro, Mortellaro, Alessandra
Publicado no Mol Ther (2021)

Autoimmune Dysregulation and Purine Metabolism in Adenosine Deaminase Deficiency

Por Sauer, Aisha Vanessa, Brigida, Immacolata, Carriglio, Nicola, Aiuti, Alessandro
Publicado em 2012

Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions

Por Ferrua, Francesca, Marangoni, Francesco, Aiuti, Alessandro, Roncarolo, Maria Grazia
Publicado no J Allergy Clin Immunol (2020)

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I

Por Penati, Rachele, Fumagalli, Francesca, Calbi, Valeria, Bernardo, Maria Ester, Aiuti, Alessandro
Publicado no J Inherit Metab Dis (2017)

How I treat ADA deficiency

Por Gaspar, H. Bobby, Aiuti, Alessandro, Porta, Fulvio, Candotti, Fabio, Hershfield, Michael S., Notarangelo, Luigi D.
Publicado em 2009

Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy

Por Sauer, Aisha V., Morbach, Henner, Brigida, Immacolata, Ng, Yen-Shing, Aiuti, Alessandro, Meffre, Eric
Publicado em 2012

Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches

Por Fraldi, Alessandro, Serafini, Marta, Sorrentino, Nicolina Cristina, Gentner, Bernhard, Aiuti, Alessandro, Bernardo, Maria Ester
Publicado no Ital J Pediatr (2018)

Multiparametric Whole Blood Dissection: A one‐shot comprehensive picture of the human hematopoietic system

Por Basso‐Ricci, Luca, Scala, Serena, Milani, Raffaella, Migliavacca, Maddalena, Rovelli, Attilio, Bernardo, Maria Ester, Ciceri, Fabio, Aiuti, Alessandro, Biasco, Luca
Publicado no Cytometry A (2017)

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

Por Kohn, Donald B., Hershfield, Michael S., Puck, Jennifer M., Aiuti, Alessandro, Blincoe, Annaliesse, Gaspar, H. Bobby, Notarangelo, Luigi D., Grunebaum, Eyal
Publicado no J Allergy Clin Immunol (2018)

Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients

Por Cassani, Barbara, Mirolo, Massimiliano, Cattaneo, Federica, Benninghoff, Ulrike, Hershfield, Michael, Carlucci, Filippo, Tabucchi, Antonella, Bordignon, Claudio, Roncarolo, Maria Grazia, Aiuti, Alessandro
Publicado em 2008

Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in ADA-SCID

Por Cancrini, Caterina, Ferrua, Francesca, Scarselli, Alessia, Brigida, Immacolata, Romiti, Maria Luisa, Barera, Graziano, Finocchi, Andrea, Roncarolo, Maria Grazia, Caniglia, Maurizio, Aiuti, Alessandro
Publicado em 2010

Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome

Por Catucci, Marco, Prete, Francesca, Bosticardo, Marita, Castiello, Maria Carmina, Draghici, Elena, Locci, Michela, Roncarolo, Maria Grazia, Aiuti, Alessandro, Benvenuti, Federica, Villa, Anna
Publicado em 2011

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

Por Cicalese, Maria Pia, Ferrua, Francesca, Castagnaro, Laura, Rolfe, Katie, De Boever, Erika, Reinhardt, Rickey R., Appleby, Jonathan, Roncarolo, Maria Grazia, Aiuti, Alessandro
Publicado no Mol Ther (2018)

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

Por Biasco, Luca, Ambrosi, Alessandro, Pellin, Danilo, Bartholomae, Cynthia, Brigida, Immacolata, Roncarolo, Maria Grazia, Di Serio, Clelia, von Kalle, Christof, Schmidt, Manfred, Aiuti, Alessandro
Publicado em 2011

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

Por Zonari, Erika, Desantis, Giacomo, Petrillo, Carolina, Boccalatte, Francesco E., Lidonnici, Maria Rosa, Kajaste-Rudnitski, Anna, Aiuti, Alessandro, Ferrari, Giuliana, Naldini, Luigi, Gentner, Bernhard
Publicado no Stem Cell Reports (2017)