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Shen et al. combine a genome-wide CRISPR screen in mice with an adult human retina model to identify broadly protective gene therapies for retinitis pigmentosa. They show that boosting UFD1 or UXT preserves photoreceptors and vision by helping the retina withstand stress from misfolded proteins.
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A genome-wide in vivo CRISPR screen identifies neuroprotective strategies in the mouse and human retina