At the recent ASGCT meeting, researchers from Exegenesis Bio Inc. presented preclinical data for EXG-102-031, a novel recombinant adeno-associated virus (rAAV)-gene therapy being developed for the treatment of neovascular age-related macular degeneration (AMD), also called wet AMD (wAMD).
MicroRNA-184 (miR-184) mutations have been previously tied to inherited anterior segment dysgenesis, potentially causing cataracts and keratoconus. To elucidate the involvement of miR-184 in ocular disorders, Chinese researchers have developed a miR-184 knockout zebrafish model. Investigators used CRISPR-Cas9 technology to delete two miR-184 paralogs (dre-mir-184-1 and dre-mir-184-2) in zebrafish, which are highly conserved between human and fly.
Researchers from Senelix Co. Ltd. described the preclinical characterization of the novel adiponectin receptor protein 1/2 (ADIPOR1/2) agonist SL-100, which is being developed for the treatment of dry eye disease (DED).
A study comprising a total of 406 subjects, including both healthy individuals (n=54) and patients with dry eye (DE) disease was performed with the aim of discovering basal tear cytokine markers for disease diagnosis and severity. Based on guidelines, patients with DE were classified as predisposed DE (pDE, n=136), mild-moderate DE (mDE, n=185), and severe DE (sDE, n=31).
The SLC6A6 gene encodes the transporter of the amino acid taurine. In recently presented work, researchers from the Institute of Molecular and Clinical Ophthalmology Basel, University of Basel and affiliated organizations aimed to investigate the molecular pathology of a novel mutation in SLC6A6 and its association with a syndromic form of Leber congenital amaurosis (LCA).