Dulla K, Aguilà M, Lane A, Jovanovic K, Parfitt D, Schulkens I, et al
. Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models. Mol Ther Nucleic Acids. 2018;12:730-740 pubmed publisher
..In conclusion, the pharmacodynamic, pharmacokinetic, and safety properties make QR-110 a promising candidate for treating LCA10, and clinical development is currently ongoing. ..