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
William W. Hauswirth(University of Florida), Samuel G. Jacobson(Penn Presbyterian Medical Center), Tomás S. Alemán(Children's Hospital of Philadelphia), Thomas J. Conlon(Adelson Foundation), Artur V. Cideciyan(University of Pennsylvania), Barry J. Byrne(University of Florida), Lili Wang(Hunan Agricultural University), Sharon Schwartz(Penn Presbyterian Medical Center), Sanford L. Boye(University of Florida), Shalesh Kaushal(University of Florida), Terence R. Flotte(University of Massachusetts Chan Medical School)
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