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CNS-Targeting Therapies for Lysosomal Storage Diseases: Current Advances and Challenges

During the past decades, several therapeutic approaches have been developed and made rapidly available for many patients afflicted with lysosomal storage disorders (LSDs), inborn organelle disorders with broad clinical manifestations secondary to the progressive accumulation of undegraded macromolec...

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Vydáno v:Front Mol Biosci
Hlavní autoři: Edelmann, Mariola J., Maegawa, Gustavo H. B.
Médium: Artigo
Jazyk:Inglês
Vydáno: Frontiers Media S.A. 2020
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7693645/
https://ncbi.nlm.nih.gov/pubmed/33304924
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fmolb.2020.559804
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