Choroid plexus-targeted viral gene therapy for alpha-mannosidosis, a prototypical neurometabolic lysosomal storage disease

Choi, E.-Y., Wolfe, J. H., & Kaler, S. G. (2025). Choroid plexus-targeted viral gene therapy for alpha-mannosidosis, a prototypical neurometabolic lysosomal storage disease. Human Molecular Genetics. https://doi.org/10.1093/hmg/ddae201
Authors:
Eun-Young Choi
John H Wolfe
Stephen G Kaler
Affiliated Authors:
Stephen G Kaler
Columbia Affiliation:
Author Keywords:
choroid plexus
lysosome
adeno-associated virus
alpha-mannosidosis
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Publication Type:
Article
Unique ID:
10.1093/hmg/ddae201
PMID:
39815619
DOI:
10.1093/hmg/ddae201
Journal:
Human Molecular Genetics
Publication Date:
Data Source:
PubMed
Source Link:
View PubMed Record
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Record Created: