grant
Gene Therapy for LCA5-Assocated Inherited Retinal Degeneration: Extension to Pediatric Cohorts
Organization UNIVERSITY OF PENNSYLVANIALocation PHILADELPHIA, UNITED STATESPosted 15 Sept 2024Deadline 31 Jul 2028
FDANIHUS FederalResearch GrantFY2025
Description preview
PROJECT SUMMARY / ABSTRACT
Advances in genetics and genomics have raised hopes for the development of gene-based treatments for
inherited retinal diseases (IRDs) which are single-gene defects that cause vision loss. Some of the greatest
successes for gene-based treatments has occurred in the most severe subset of IRDs that fall into the rubric of…
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