The Changing Face of Adrenoleukodystrophy. Endocr Rev. 2020..
How we deal with the COVID-19 epidemic in an Italian pediatric onco-hematology clinic located in a region at high density of cases. Br J Haematol. 2020..
Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.
Biodegradable polymeric nanoparticles administered in the cerebrospinal fluid: Brain biodistribution, preferential internalization in microglia and implications for cell-selective drug release. Biomaterials. 2019;209:25-40..
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. Lancet. 2016.
Voices of biotech. Nat Biotechnol. 2016;34(3):270-275.
Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy. Mol Ther Methods Clin Dev. 2015;2:15038..