Mandy Boontanrart - Ariya Bio
Reactivating fetal hemoglobin in B-Hemoglobinopathy patients
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B-Hemoglobinopathies are one of the most common genetic diseases worldwide with still no curative therapy other than allogeneic stem cell transplant. To address this, we are developing a CRISPR/Cas9 therapeutic approach to reactivate fetal hemoglobin that will ameliorate the disease symptoms. The genetic region we are targeting has not been proposed or explored for therapeutic benefit before.
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Mandy Boontanrart