Therapeutic strategies based on modified U1 snRNAs and chaperones for Sanfilippo C splicing mutations
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A. Pshezhetsky | M. Prata | P. Jordan | D. Grinberg | L. Vilageliu | L. Matos | S. Alves | B. Pérez | L. Desviat | I. Canals | L. Dridi | Yoo Choi