Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models
Dulla K., Aguila M., Lane A., Jovanovic K., Parfitt DA., Schulkens I., Chan HL., Schmidt I., Beumer W., Vorthoren L., Collin RWJ., Garanto A., Duijkers L., Brugulat-Panes A., Semo M., Vugler AA., Biasutto P., Adamson P., Cheetham ME.