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Blood|May 1, 2016
Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiencyMaria Pia Cicalese, Francesca Ferrua, Laura Castagnaro, et al.
Growth Hormone & IGF Research : Official Journal of the Growth Hormone Research Society and the International IGF Research Society|September 6, 2019
Uniparental disomy and pretreatment IGF-1 may predict elevated IGF-1 levels in Prader-Willi patients on GH treatmentViviana Valeria Palmieri, Antonella Lonero, Sarah Bocchini, et al.
Frontiers in Endocrinology|May 13, 2024
Endocrine features of Prader-Willi syndrome: a narrative review focusing on genotype-phenotype correlationSimona F Madeo, Luca Zagaroli, Sara Vandelli, et al.
The Lancet. Haematology|April 15, 2019
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical studyFrancesca Ferrua, Maria Pia Cicalese, Stefania Galimberti, et al.
Nature Medicine|February 15, 2024
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiencyMaddalena Migliavacca, Federica Barzaghi, Claudia Fossati, et al.
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