Cystinosis - Symptoms, Causes, Treatment | NORD
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A misstep in the kidney’s key component genesis revealed to cause infantile cystinosis, a rare disease that significantly shortens patients’ lifespans. Researchers at the University of Buffalo reveal insights into addressing and potentially curing the disease through CRISPR genome editing. This breakthrough may render kidney transplants, currently the most effective treatment for infantile cystinosis patients, unnecessary. Published in the open access International Journal of Molecular Sciences,
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