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Description

R341C versus WT: 0.7530 ( i )

Am J Kidney Dis 76:S1S107

Zheng, Y., Cheung, C

The Roadmap for a rare disease should identify experiments necessary to understand the disease, identify drug repurposing opportunities, and explore the use of emerging technologies like gene therapy, ASOs, CRISPR/Cas9 and others to treat this disease