Francesca Tucci

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora et al. · The Lancet · 2022 · 284 citations · Full text

Medicine, Hematology, Immunology +15

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry

Maria Elena Maccari, Hassan Abolhassani, Asghar Aghamohammadi et al. · Frontiers in Immunology · 2018 · 182 citations · Full text

Life Sciences, Immunology, Pharmacotherapy +17

MicroRNA-449a Overexpression, Reduced NOTCH1 Signals and Scarce Goblet Cells Characterize the Small Intestine of Celiac Patients

Laura Iaffaldano, Nadia Tinto, Donatella Montanaro et al. · PLoS ONE · 2011 · 103 citations · Full text

Life Sciences, Bioinformatics, Genetics +17

Treatment with rapamycin can restore regulatory T-cell function in IPEX patients

Laura Passerini, Federica Barzaghi, Rosalia Curto et al. · Journal of Allergy and Clinical Immunology · 2019 · 73 citations · Full text

Life Sciences, Immunology, Pharmacotherapy +12

Potential Celiac Patients: A Model of Celiac Disease Pathogenesis

Maria Pia Sperandeo, Antonella Tosco, Valentina Izzo et al. · PLoS ONE · 2011 · 61 citations · Full text

Life Sciences, Genetics, Immunology +17