Rate My Professor Arlene Drack

Dr. Drack's research focuses on inherited eye diseases affecting children, particularly the development of novel treatments. Her laboratory work involves mouse models of retinal degeneration, subretinal injection of therapeutic molecules, and translational studies preparing for human trials, with a special emphasis on Bardet-Biedl Syndrome. She serves as Principal Investigator for the Phase III RPE65 Leber congenital amaurosis gene therapy trial at the University of Iowa and Children's Hospital of Philadelphia. Her contributions include participation in research leading to FDA consideration of the first ocular gene therapy approval. Dr. Drack chairs the inaugural Genetic Eye Disease task force for the American Association for Pediatric Ophthalmology and Strabismus. Key publications include 'Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial' (The Lancet, 2017), 'Mutations in NYX, encoding the leucine-rich proteoglycan nyctalopin, cause X-linked complete congenital stationary night blindness' (Nature Genetics, 2000), 'Infantile cataracts' (Survey of Ophthalmology, 1996), 'Patient-specific iPSC-derived photoreceptor precursor cells as a means to investigate retinitis pigmentosa' (eLife, 2013), and 'Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect' (Nature Medicine, 2019). Her work has advanced genetic testing recommendations and gene therapy for blinding retinal disorders.