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Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders

Lysosomal storage disorders (LSDs) represent a group of more than 50 severe metabolic diseases caused by the deficiency of specific lysosomal hydrolases, activators, carriers, or lysosomal integral membrane proteins, leading to the abnormal accumulation of substrates within the lysosomes. Numerous m...

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Dettagli Bibliografici
Pubblicato in:Genes (Basel)
Autori principali: Dardis, Andrea, Buratti, Emanuele
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2018
Soggetti:
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5852569/
https://ncbi.nlm.nih.gov/pubmed/29415500
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/genes9020073
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