P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up* Article Swipe

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Francesca Fumagalli , Valeria Calbi , Fabiola De Mattia , Alberto A. Zambon , Vera Gallo , Salvatore Recupero , Elena Sophia Fratini , Alessia Ippolito , Francesca Ciotti , Maddalena Fraschini , Marina Sarzana , Stefano Scarparo , Eugenio Montini , Sara Locatelli , Marcella Facchini , Alessandra Clerici , Francesco Morena , Sabata Martino , Muska Yarzi , Sean Moro , Andrew Shenker , Jean Brooks , Alan E. Richardson , Leslie Meltzer , Laura Campbell , Alessandro Aiuti ·

YOU? · · 2024 · Open Access · · DOI: https://doi.org/10.1016/j.gimo.2024.100882

Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disorder caused by biallelic pathogenic variants in the arylsulfatase A (ARSA) gene that result in deficiency of the ARSA enzyme. ARSA deficiency leads to accumulation of the undegraded substrate (sulfatides) in the central and peripheral nervous systems, and subsequently progressive demyelination, neurodegeneration, loss of motor and cognitive skills, and early death. MLD is a devastating and debilitating disease with profound burden and poor quality of life (QoL) for affected patients and their caregivers.

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Metachromatic leukodystrophy Genetic enhancement Haematopoiesis Stem cell Hematopoietic stem cell Neuroscience Medicine Biology Gene Pathology Genetics

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Type: article
Language: en
Landing Page: https://doi.org/10.1016/j.gimo.2024.100882
PDF: http://www.gimopen.org/article/S2949774424000281/pdf
OA Status: diamond
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OpenAlex ID: https://openalex.org/W4392606763

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DOI: https://doi.org/10.1016/j.gimo.2024.100882

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Title: P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*

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Language: en

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Publication year: 2024

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Authors: Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto A. Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan E. Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti

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Landing page: https://doi.org/10.1016/j.gimo.2024.100882

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Concepts: Metachromatic leukodystrophy, Genetic enhancement, Haematopoiesis, Stem cell, Hematopoietic stem cell, Neuroscience, Medicine, Biology, Gene, Pathology, Genetics

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