Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations

Title
Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations
Authors
Keywords
Splicing mutations, Modified U1 snRNAs, Glucosamine, Sanfilippo C syndrome, Lysosomal storage disorder
Journal
Orphanet Journal of Rare Diseases
Volume 9, Issue 1, Pages -
Publisher
Springer Nature
Online
2014-12-10
DOI
10.1186/s13023-014-0180-y

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