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Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

Metachromatic leukodystrophy (MLD) is a rare, autosomal recessive neurodegenerative disease caused by deficient activity of the lysosomal enzyme arylsulfatase A (ARSA), resulting in sulfatide accumulation and subsequent demyelination and neuronal damage within the central and peripheral nervous syst...

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Principais autores: Emilie Audouard, Nicolas Khefif, Charlotte Mansat, Océane Nelcha, Elena-Gaia Banchi, Camille Lupiet, Dominique Farabos, Antonin Lamaziere, Caroline Sevin, Françoise Piguet
Format: Artigo
Sprog:Inglês
Udgivet: Elsevier 2024-06-01
Serier:Molecular Therapy: Methods & Clinical Development
Fag:
metachromatic leukodystrophy
gene therapy
intravenous
AAV
Online adgang:http://www.sciencedirect.com/science/article/pii/S2329050124000640
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