Which specific application utilizes *in vivo* therapy via direct injection into the eye to restore function?
Answer
Inherited retinal diseases like Leber congenital amaurosis (LCA).
The treatment strategy is closely matched to the target tissue accessibility and the nature of the disease. For inherited retinal diseases such as Leber congenital amaurosis (LCA), which affect localized, accessible tissue within the eye, the *in vivo* method is highly effective. The vector carrying the functional gene copy is injected directly into the ocular environment, targeting the light-sensing cells. This localized delivery ensures high concentration at the site of the defect without necessitating the systemic administration required for treatments targeting widespread tissues like the blood system or motor neurons.
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