Gene Therapy for Inherited Retinal Diseases: From Laboratory Bench to Patient Bedside and Beyond.

ABSTRACT: This comprehensive review provides a thorough examination of inherited retinal diseases (IRDs), encompassing their classification, genetic underpinnings, and the promising landscape of gene therapy trials. IRDs, a diverse group of genetic conditions causing vision loss through photoreceptor cell death, are explored through various angles, including inheritance patterns, gene involvement, and associated systemic disorders. The focal point is gene therapy, which offers hope for halting or even reversing the progression of IRDs. The review highlights ongoing clinical trials spanning retinal cell replacement, neuroprotection, pharmacological interventions, and optogenetics. While these therapies hold tremendous potential, they face challenges like timing optimization, standardized assessment c

SUBMITTER: Brar AS 

PROVIDER: S-EPMC10776519 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature