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Advanced technologies for differentiating kidney organoids from hPSCs and efficient genome-editing systems with the clustered regularly interspaced short palindromic repeat CRISPR-Cas9 have enabled modeling of human kidney diseases 15,16,17

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In China, supportive policies like accelerated approval pathways for urgently needed orphan drugs (UNOD) and Care for Rare Diseases Encouragement (CARE) plan 28 , have been implemented to improve access to therapies for rare conditions