Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

Infantile globoid cell leukodystrophy (GLD, Krabbe disease) is a demyelinating disease caused by the deficiency of the lysosomal enzyme galactosylceramidase (GALC) and the progressive accumulation of the toxic metabolite psychosine. We showed previously that central nervous system (CNS)-directed, ad...

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Udgivet i:Mol Ther
Main Authors: Li, Yedda, Miller, Christopher A., Shea, Lauren K., Jiang, Xuntian, Guzman, Miguel A., Chandler, Randy J., Ramakrishnan, Sai M., Smith, Stephanie N., Venditti, Charles P., Vogler, Carole A., Ory, Daniel S., Ley, Timothy J., Sands, Mark S.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2021
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7854295/
https://ncbi.nlm.nih.gov/pubmed/33388420
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.12.031
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