Splice-Modulating Oligonucleotide QR-110 Restores CEP290...

Molecular Therapy — Nucleic Acids 2018 / 09 Vol. 12

Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

Dulla, Kalyan, Aguila, Monica, Lane, Amelia, Jovanovic, Katarina, Parfitt, David A., Schulkens, Iris, Chan, Hee Lam, Schmidt, Iris, Beumer, Wouter, Vorthoren, Lars, Collin, Rob W.J., Garanto, Alejandr
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Volume:
12
Language:
english
Journal:
Molecular Therapy - Nucleic Acids
DOI:
10.1016/j.omtn.2018.07.010
Date:
September, 2018
File:
PDF, 1.71 MB
english, 2018
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