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

Crossref DOI link: https://doi.org/10.1186/s13023-014-0180-y

Published Online: 2014-12-10

Published Print: 2014-12

Update policy: https://doi.org/10.1007/springer_crossmark_policy

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