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Orphanet Journal of Rare Diseases|August 3, 2023
How to START? Four pillars to optimally begin your orphan drug developmentAnneliene Hechtelt Jonker, Liliana Batista, Michela Gabaldo, et al.
Veterinary Pathology|January 26, 2026
A comprehensive review of humanized mice applications in regulatory submissions for cell and gene therapy productsGiovanni Pellegrini, Lucia Minoli, Sara Degl'Innocenti, et al.
Orphanet Journal of Rare Diseases|April 8, 2018
Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCIDHeide Stirnadel-Farrant, Mahesh Kudari, Nadia Garman, et al.
Lancet (London, England)|June 13, 2016
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trialMaria Sessa, Laura Lorioli, Francesca Fumagalli, et al.
Nature Medicine|January 22, 2019
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemiaSarah Marktel, Samantha Scaramuzza, Maria Pia Cicalese, et al.
Lancet (London, England)|January 23, 2022
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 accessFrancesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, et al.
The New England Journal of Medicine|November 17, 2021
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler SyndromeBernhard Gentner, Francesca Tucci, 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.
Stem Cell Reports|January 23, 2026
Charting the translational pathway: ISSCR best practices for the development of PSC-derived therapiesJaqueline Barry, Elsa Abranches, Ricardo P Baptista, et al.
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