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An association between variants in the IGF2 gene and BeckwithWiedemann syndrome: interaction between genotype and epigenotype

Voretigene neparvovec-rzyl (Luxturna) was approved by FDA in December of 2017 and is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy

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The therapeutic target is the disease, not the BMI

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