Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up

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Veröffentlicht in:	Molecular genetics and metabolism 2024-02, Vol.141 (2), p.107831, Article 107831
Hauptverfasser:	Fumagalli, Francesca, Calbi, Valeria, De Mattia, Fabiola, Zambon, Alberto Andrea, Gallo, Vera, Recupero, Salvatore, Fratini, Elena Sophia, Ippolito, Alessia, Ciotti, Francesca, Fraschini, Maddalena, Sarzana, Marina, Scarparo, Stefano, Montini, Eugenio, Locatelli, Sara, Facchini, Marcella, Clerici, Alessandra, Morena, Francesco, Martino, Sabata, Yarzi, Muska, Moro, Sean, Shenker, Andrew, Brooks, Jean, Richardson, Alan, Campbell, Laura, Aiuti, Alessandro
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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