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ARVO Annual Meeting Abstract July 2019

CEP290 -associated LCA due to a photoreceptor cilium defect treated with an intravitreal antisense oligonucleotide results in improved vision

Artur V Cideciyan; Samuel G Jacobson; Arlene V Drack; Allen C Ho; Alexandra Garafalo; Alejandro J Roman; Mike Schwartz; Patricia Biasutto; Wilma de Wit; Michael E Cheetham; Peter S Adamson; David Rodman; Julie De Zaeytijd; Caroline Van Cauwenbergh; Bart P Leroy; Stephen R Russell

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):1834. doi: https://doi.org/

ARVO Annual Meeting Abstract July 2019

Allele specific knock-down of human P23H rhodopsin mRNA and prevention of retinal degeneration in humanized P23H rhodopsin knock-in mouse, following treatment with an intravitreal GAPmer antisense oligonucleotide (QR-1123)

Patricia Biasutto; Peter S Adamson; Kalyan Dulla; Sue Murray; Brett Monia; Michael McCaleb

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):5719. doi: https://doi.org/

ARVO Annual Meeting Abstract June 2017

QR-110 Treatment for Leber’s Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA10 iPSC-Derived Optic Cups

Patricia Biasutto; Kalyan Dulla; Peter Adamson; Iris Schulkens; Iris Schmidt; Amelia Lane; Monica Aguila; Michael E Cheetham

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):249. doi: https://doi.org/

ARVO Annual Meeting Abstract September 2016

QR-110, an RNA oligonucleotide (AON) directing skipping of the cryptic exon in p.Cys998X CEP290 associated with Leber’s congenital amaurosis, shows increase in wild-type mRNA and an ability to reach the outer nuclear layer (ONL) in mice following intravitreal administration

Patricia Biasutto; Hee Lam Chan; Hester Boersma; Thomas Hoogenboezem; Jim Swildens; Kalyan Dulla; Ma'ayan Mary Semo; Anthony Alexander Vugler; Rob Collin; Peter Adamson

Invest. Ophthalmol. Vis. Sci.. 2016; 57(12):1123. doi: https://doi.org/

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