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Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations 由 Liliana Matos, Isaac Canals, Larbi Dridi, Yoo Choi, Maria João Prata, Peter Jordan, Lourdes R. Desviat, Belén Pérez, Alexey V. Pshezhetsky, Daniel Grinberg, Sandra Alves, Lluı̈sa Vilageliu
出版 2014Artigo
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Biology
Cell biology
Gene
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Glycosaminoglycan
Heparan sulfate
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Lysosomal storage disease
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Protein splicing
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Splice site mutation
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