Data Sheet 1_Preclinical biodistribution and toxicology assessment of an AAV5-based subretinal modifier gene therapy for retinitis pigmentosa.pdf
<p>Retinitis pigmentosa (RP) is a multifactorial disease caused by mutations in over 100 genes, affecting ~1 in 4,000 people worldwide, and is characterized by abnormalities in the rod and cone photoreceptors. Gene therapy approaches are promising and have been established to provide an unprec...
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2025
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