Alessandra Biffi

Voices of biotech

Jun Qin, Bonnie Berger, Drew Endy, 2016, Nature Biotechnology.

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Jinkuk Kim, Annapurna Poduri, P Ellen Grant, 2019, The New England journal of medicine.

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Chunyan Ren, Luca Pinello, Luca Biasco, 2019, Nature Medicine.

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Luca Biasco, Alessandro Aiuti, Maria Grazia Valsecchi, 2013, Science.

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection.

Gianluigi Zanetti, Christof von Kalle, Alessandra Biffi, 2011, Blood.

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.

Andrea Calabria, Alessandra Biffi, Luigi Naldini, 2016, Human gene therapy.

Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

Alessandra Biffi, Luigi Naldini, Angelo Quattrini, 2010, Science Translational Medicine.

Biffi Metachromatic Leukodystrophy Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits

Gianluigi Zanetti, Luca Biasco, Alessandro Aiuti, 2013 .