Finding cures for children's genetic diseases

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Your gift will enable life changing research

Your gift will enable life changing research

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Accurate diagnosis can save lives At CMRI, we are tackling bone marrow failure syndromes with a two-pronged strategy: Diagnosis and Therapy. We are working with leaders in the field to create and test methods for correcting these telomere problems, in order to prevent bone marrow failure and related cancers. CMRI’s research on the precise diagnosis and treatment of bone marrow failure disorders desperately needs research staff and resources. Gene therapy requires the highest level of expertise, and gene editing technology is still very expensive, as is growing and studying a patient’s stem cells. This is cutting-edge work, for which your support is essential.