Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD)
Vera Gallo,Valeria Calbi,Fabiola De Mattia,Alberto Andrea Zambon, Salvatore Recupero,Elena Sophia Fratini,Francesca Ciotti,Maddalena Fraschini, Mara Sangalli, Marina Sarzana, Alessandra Clerici,Francesco Morena,Sabata Martino, Jean Brooks, Alan Richardson,Laura Campbell,Alessandro Aiuti,Francesca Fumagalli MOLECULAR GENETICS AND METABOLISM（2024）
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