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Targeting the Cure: Advances in Gene Editing for Retinal Diseases

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The Retina International World Congress 2024, hosted by Fighting Blindness, brought together patients, caregivers, clinicians, researchers, and patient advocates to discuss the latest advancements in gene editing for inherited retinal dystrophies (IRDs). IRDs are a group of genetic diseases that damage the retina, leading to progressive visual impairment and blindness. With over 250 IRD-causing genes identified, the urgency to develop new treatments is evident.

In Vivo Correction of CEP290 Mutations: Data from Editas Medicine’s EDIT-101 Trial

Professor Mark Pennesi, Oregon Health & Science University (OHSU) Casey Eye Institute and Retina Foundation of the Southwest, presented data from Editas Medicine’s Phase 1/2 BRILLIANCE trial of EDIT-101, a CRISPR-Cas9-based therapeutic candidate designed to remove the intronic mutation IVS26 in the CEP290 gene. The mutation leads to a premature stop codon in the CEP290 mRNA and a truncated, non-functional CEP290 protein.

The trial involved 14 participants, who received a single dose of EDIT-101 via sub-retinal injection. The results showed that six participants exhibited a meaningful improvement in cone-mediated vision, and four participants showed a significant improvement in visual acuity. Additionally, four participants demonstrated improvements in visual navigation, and six participants reported meaningful improvements in quality-of-life scores.

In Vivo Modelling of Retinitis Pigmentosa

Assistant Professor Leah Byrne, University of Pittsburgh School of Medicine, presented her team’s work on developing in vivo models of PRPF31 retinitis pigmentosa (RP). The team used CRISPR-Cas9 gene editing to introduce various PRPF31 deletions in mice and observed that sub-retinal injection of the gene-editing cargo led to efficient targeting of photoreceptors and better represented the human RP11 situation.

The results showed that co-injection of the PRPF31 knockout construct and a Cas9-resistant construct containing a full-length wild-type PRPF31 sequence resulted in rescue of RP symptoms 5 weeks after treatment. The data support PRPF31 gene augmentation as a therapeutic approach for RP11.

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