Surrogate Cerebrospinal Fluid Biomarkers for Assessing the Efficacy of Gene Therapy in Hurler Syndrome

Mucopolysaccharidosis type I (MPS I) is caused by a deficiency of the lysosomal hydroxylase alpha-l-iduronidase (IDUA). The resulting accumulation of dermatan and heparan sulfate induces intellectual disabilities and pre-mature death, and only a few treatment options are available. In a previous stu...

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Bibliografski detalji
Izdano u:Front Neurol
Glavni autori: Haseloff, Reiner F., Trudel, Stephanie, Birke, Ramona, Schümann, Michael, Krause, Eberhard, Gomila, Cathy, Heard, Jean-Michel, Blasig, Ingolf E., Ausseil, Jérôme
Format: Artigo
Jezik:Inglês
Izdano: Frontiers Media S.A. 2021
Teme:
Neurology
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8155356/
https://ncbi.nlm.nih.gov/pubmed/34054689
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fneur.2021.640547
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