Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

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Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

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Defective myosin VIIA gene responsible for Usher syndrome type IB

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Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

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