| Literature DB >> 33795869 |
Artur V Cideciyan1, Samuel G Jacobson2, Allen C Ho3, Alexandra V Garafalo2, Alejandro J Roman2, Alexander Sumaroka2, Arun K Krishnan2, Malgorzata Swider2, Michael R Schwartz4, Aniz Girach4.
Abstract
Leber congenital amaurosis due to CEP290 ciliopathy is being explored by treatment with the antisense oligonucleotide (AON) sepofarsen. One patient who was part of a larger cohort (ClinicalTrials.gov NCT03140969 ) was studied for 15 months after a single intravitreal sepofarsen injection. Concordant measures of visual function and retinal structure reached a substantial efficacy peak near 3 months after injection. At 15 months, there was sustained efficacy, even though there was evidence of reduction from peak response. Efficacy kinetics can be explained by the balance of AON-driven new CEP290 protein synthesis and a slow natural rate of CEP290 protein degradation in human foveal cone photoreceptors.Entities:
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Year: 2021 PMID： 33795869 PMCID： PMC8127404 DOI： 10.1038/s41591-021-01297-7
Source DB: PubMed Journal: Nat Med ISSN： 1078-8956 Impact factor: 53.440