Genetic rescue, a ‘natural gene therapy,’ suggests possible new approach to pediatric MDS

Researchers have discovered a battle waged within the blood systems of children who inherit mutations in the genes SAMD9 and SAMD9L. The outcomes have life-or-death consequences for patients. Investigators are working to learn how to influence the results.,Researchers have discovered a battle waged within the blood systems of children who inherit mutations in the genes SAMD9 and SAMD9L. The outcomes have life-or-death consequences for patients. Investigators are working to learn how to influence the results.

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