4.3 Editorial Material

What's next in gene therapy for Crigler-Najjar syndrome?

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Summary: This study investigates the long-term efficacy of AAV8-hUGT1A1 in treating Crigler-Najjar syndrome and explores the possibility of re-treatment using the same AAV vector with immune modulation. Results show that the timing of administration at different ages affects the efficacy in rats, and an immune-suppressive regimen can enhance treatment effectiveness to some extent.

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