Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy-Reference-Cited by-同舟云学术

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

Published:2024-06 Issue:2 Volume:32 Page:101248
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Audouard Emilie,Khefif Nicolas,Mansat Charlotte,Nelcha Océane,Banchi Elena-Gaia,Lupiet Camille,Farabos Dominique,Lamaziere Antonin,Sevin Caroline,Piguet Françoise

Funder

ELA

Publisher

Elsevier BV

Reference50 articles.

1. Metachromatic Leukodystrophy;Gieselmann,2006

2. A clinical approach to the diagnosis of patients with leukodystrophies and genetic leukoencephelopathies;Parikh;Mol. Genet. Metab.,2015

3. Metachromatic leukodystrophy an update;Gieselmann;Neuropediatrics,2010

4. Metachromatic leukodystrophy: Disease spectrum and approaches for treatment;Van Rappard;Best Pract. Res. Clin. Endocrinol. Metab.,2015

5. Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches;Shaimardanova;Front. Med.,2020

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