Terence R. Flotte

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, Tomás S. Alemán, Shalesh Kaushal et al. · Human Gene Therapy · 2008 · 947 citations · Full text

Medicine, Molecular Biology, Ophthalmology +15

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Artur V. Cideciyan, Tomás S. Alemán, Sanford L. Boye et al. · Proceedings of the National Academy of Sciences · 2008 · 664 citations · Full text

Medicine, Genetics, Ophthalmology +17

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

Sergei Zolotukhin, Mark Potter, Irene Zolotukhin et al. · Methods · 2002 · 577 citations

Life Sciences, Biotechnology, Molecular Biology +12

Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

Terence R. Flotte, Sandra Afione, C. Conrad et al. · Proceedings of the National Academy of Sciences · 1993 · 489 citations

Pharmacy, Molecular Biology, Molecular Genetics +17

Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR

Marie E. Egan, Terence R. Flotte, Sandra Afione et al. · Nature · 1992 · 421 citations

Natural Sciences, Molecular Biology, Molecular Genetics +14