HIGHLIGHTS
- who: Kamil Kruczek from the National Eye Institute, USA have published the research: Gene Therapy of Dominant CRX-Leber Congenital Amaurosis using Patient Stem Cell-Derived Retinal Organoids, in the Journal: (JOURNAL)
- what: The studies provide a proof-of-concept for developing gene therapy of dominant CRX-LCA and other CRX retinopathies. The authors established a retinal organoid model of CRX-LCA from patient-derived iPSCs and demonstrate perturbation in molecular phenotype of photoreceptors, including diminished expression of visual opsins, in concordance with clinically observed loss of light responses. The contribution of these mechanisms may . . .
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