Gene Therapy Updates for Inherited Retinal Dystrophies
Abstract
Inherited retinal dystrophies (IRDs) encompass a group of genetically diverse disorders, each uniquely influencing distinct retinal cell pathways and retinal areas. IRDs currently affect an estimated 5.5 million individuals worldwide, exerting a profound impact on the quality of life of those affected. Depending on the mutated gene, typical presentations often manifest as colour or night blindness, or peripheral vision blindness progressing to complete blindness. Consequently, patients grappling with IRDs face not only the physical challenges of their condition, but also endure significant psychosocial and economic repercussions.
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