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This systematic review evaluates gene therapy's efficacy and safety in XLRP, indicating promising but risky outcomes.
XLRP, a severe hereditary retinal dystrophy primarily caused by RPGR gene mutations, lacks curative treatments. AAV-mediated RPGR gene therapy shows promise in early trials, but systemic evaluation of its efficacy and safety is absent. This evaluates gene therapy as a novel approach, noting short-term efficacy but ocular AE risks.
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Yang et al. (2026) studied this question.
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