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9 Publications listed

Synthesis, Biological Activity, and Molecular Dynamics Simulations of LNA-Charge Neutral Linkages for Enhanced Splice-Switching Antisense Oligonucleotides

Mapping chromatin structure at base-pair resolution unveils a unified model of cis-regulatory element interactions

Central Retinal Sensitivity Decline in RPGR-related Retinal Phenotypes

Purpose: To characterize the natural history of central retinal sensitivity decline in RPGR-associated retinopathy, and to evaluate and quantify phenotypic differences in disease progression using microperimetry-derived metrics.

Design: A retrospective cohort study SUBJECTS: 50 patients with genetically confirmed RPGR mutations (36 rod-cone, 14 cone-rod dystrophy [CORD]), contributing 357 microperimetry assessments.

Potential CRISPR Base Editing Therapeutic Options in a Sorsby Fundus Dystrophy Patient