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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:

ARVO Annual Meeting Abstract July 2019

QR-421a, an antisense oligonucleotide, for the treatment of retinitis pigmentosa due to USH2A exon 13 mutations

Hester van Diepen; Kalyan Dulla; Hee Lam Chan; Iris Schulkens; Wouter Beumer; Lars Vorthoren; Cathaline den Besten; Levi Buil; Janne Turunen; Jiayi Miao; Sanne Broekman; Erik de Vrieze; Margo Dona; Silvia Albert; Erwin van Wijk; Peter S Adamson

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):3250. doi:

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:

ARVO Annual Meeting Abstract July 2018

Relationship of severity of Fuchs endothelial corneal dystrophy with triplet repeat expansion in TCF4 and The Potential Implications to Surgical Intervention

Kirithika Muthusamy; Beatriz Sanchez-Pintado; Nathaniel Hafford Tear; Petra Liskova; Peter S Adamson; Alison J Hardcastle; Alice E Davidson; Stephen J. Tuft

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):2920. doi:

ARVO Annual Meeting Abstract July 2018

RNA toxicity induced by TCF4 CTG expansions is ameliorated by antisense therapeutics in a patient-derived cell model of Fuchs corneal endothelial dystrophy (FECD).

Alice E Davidson; Christina Zarouchlioti; Beatriz Sanchez-Pintado; Nathaniel Hafford Tear; Pontus Klein; Petra Liskova; Kalyan Dulla; Kirithika Muthusamy; Lubica Dudakova; Pavlina Skalicka; Pirro G Hysi; Michael E. Cheetham; Stephen J. Tuft; Peter S Adamson; Alison J Hardcastle

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):3022. doi:

ARVO Annual Meeting Abstract July 2018

Oligonucleotide-based splice correction of the ABCA4 c.5461-10T>C mutation in Stargardt disease type 1

Kalyan Dulla; Seda Yilmaz-Elis; Jiayi Miao; Iris Schmidt; Iris Schulkens; Hee Lam Chan; Peter S Adamson

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):5315. doi:

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:

ARVO Annual Meeting Abstract June 2017

QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A>G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2

Hester van Diepen; Heelam Chan; Janne Turunen; Ma'ayan Semo; Anthony Vugler; Ralph Slijkerman; Erwin van Wijk; Peter Adamson

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):1203. doi:

ARVO Annual Meeting Abstract June 2017

Systemic Pharmacodynamic Efficacy of a Complement Factor B Antisense Oligonucleotide in Preclinical and Phase 1 Clinical Studies

Michael McCaleb; Tamar R Grossman; Peter Adamson; Scott Henry; Richard S Geary; Steve G Hughes; Brett P Monia

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):1952. doi:

ARVO Annual Meeting Abstract June 2017

Antisense Oligonucleotide-induced Skipping of USH2A exon13 Restores Visual Function in Zebrafish

Erwin van Wijk; Margo Dona; Ralph Slijkerman; Peter Adamson; Janne Turunen; Maarten Kamermans; Stephan C F Neuhauss; Hester van Diepen

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):2490. doi:

ARVO Annual Meeting Abstract June 2017

QRX-421, an Antisense Oligonucleotide (AON) Targeting Mutations in Exon 13 of USH2A , Associated with Retinitis Pigmentosa in Usher Syndrome Type 2, is Effective in Skipping Exon 13 in the USH2A mRNA of Patients Fibroblasts and Patient-Derived Optic Cups

Peter Adamson; Heelam Chan; Janne Turunen; Jiayi Miao; Erik de Vrieze; Margo Dona; Silvia Albert; Erwin van Wijk; Hester van Diepen

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):2969. doi:

ARVO Annual Meeting Abstract June 2017

Diabetes induced neurodegeneration in the retina and the brain of mice are associated and independent of microvasculopathy

Chunhua Jiao; Michael David Abramoff; Kyungmoo Lee; Ipek Oguz; Peter Adamson; Adam Hedberg-Buenz; Michael G Anderson; Elliott Sohn

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):5195. doi:

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:

Physiology and Pharmacology June 2016

Blockage of PI3K/mTOR Pathways Inhibits Laser-Induced Choroidal Neovascularization and Improves Outcomes Relative to VEGF-A Suppression Alone

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Jie Ma; Yu Sun; Francisco J. López; Peter Adamson; Edit Kurali; Kameran Lashkari
TAGS: vascular endothelial growth factor a, 1-phosphatidylinositol 3-kinase, choroidal neovascularization, lasers, mtor serine-threonine kinases, aflibercept
Invest. Ophthalmol. Vis. Sci.. 2016; 57(7):3138-3144. doi: 10.1167/iovs.15-18795

Letters to the Editor December 2015

Author Response: The Role of IL-18 in the Treatment of AMD

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Sarah L. Doyle; Francisco J. López; Peter Humphries; Peter Adamson; Matthew Campbell
TAGS: interleukin-18
Invest. Ophthalmol. Vis. Sci.. 2015; 56(13):8237-8238. doi: 10.1167/iovs.15-18714

Biochemistry and Molecular Biology October 2015

Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration

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Susan F. Murray; Ali Jazayeri; Michael T. Matthes; Douglas Yasumura; Haidong Yang; Raechel Peralta; Andy Watt; Sue Freier; Gene Hung; Peter S. Adamson; Shuling Guo; Brett P. Monia; Matthew M. LaVail; Michael L. McCaleb
TAGS: alleles, animals, transgenic, antisense oligonucleotides, injection of therapeutic agent, photoreceptors, rhodopsin, eye, mice, rna, tissue degeneration
Invest. Ophthalmol. Vis. Sci.. 2015; 56(11):6362-6375. doi: 10.1167/iovs.15-16400

Biochemistry and Molecular Biology August 2015

IL-18 Immunotherapy for Neovascular AMD: Tolerability and Efficacy in Nonhuman Primates

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Sarah L. Doyle; Francisco J. López; Lucia Celkova; Kiva Brennan; Kelly Mulfaul; Ema Ozaki; Paul F. Kenna; Edit Kurali; Natalie Hudson; Teresa Doggett; Thomas A. Ferguson; Peter Humphries; Peter Adamson; Matthew Campbell
TAGS: immunotherapy, interleukin-18, primates, exudative age-related macular degeneration
Invest. Ophthalmol. Vis. Sci.. 2015; 56(9):5424-5430. doi: 10.1167/iovs.15-17264

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2015

Six months effective ocular drug levels and protection against laser CNV in a cynomolgus monkey after a single ocular administration of a biodegradable microsphere delivery system releasing a highly potent anti-VEGF domain antibody based molecule.

Ian R Catchpole; Peter Adamson; Thomas Wilde; Eric Dobrzynski; Caroline Sychertz; Rodd Polsky; Richard Haworth; Chi-Man Tang; Jos Smal; Judith Van der Zwan

Invest. Ophthalmol. Vis. Sci.. 2015; 56(7 ):1498. doi:

ARVO Annual Meeting Abstract June 2015

IL-18 immunotherapy for neovascular AMD; Tolerability and efficacy in non-human primates

Matthew Campbell; Peter S Adamson; Francisco J Lopez; Edit Kurali; Peter Humphries; Sarah Doyle

Invest. Ophthalmol. Vis. Sci.. 2015; 56(7 ):4803. doi:

ARVO Annual Meeting Abstract June 2015

Identification of a Human Clinical Candidate ASO Selectively Targeting the P23H Variant of Rhodopsin for the Treatment of P23H Rhodopsin-mediated ADRP

Sue F Murray; Ali Jazayeri; Raechel Peralta; Andy Watt; Timothy Vickers; Shuling Guo; Scott P Henry; Peter S Adamson; Brett p Monia; Michael McCaleb

Invest. Ophthalmol. Vis. Sci.. 2015; 56(7 ):5402. doi:

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

QR-421a, an antisense oligonucleotide, for the treatment of retinitis pigmentosa due to USH2A exon 13 mutations

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)

Relationship of severity of Fuchs endothelial corneal dystrophy with triplet repeat expansion in TCF4 and The Potential Implications to Surgical Intervention

RNA toxicity induced by TCF4 CTG expansions is ameliorated by antisense therapeutics in a patient-derived cell model of Fuchs corneal endothelial dystrophy (FECD).

Oligonucleotide-based splice correction of the ABCA4 c.5461-10T>C mutation in Stargardt disease type 1

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

QRX-411, an Antisense Oligonucleotide (AON) Directing Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A>G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2

Systemic Pharmacodynamic Efficacy of a Complement Factor B Antisense Oligonucleotide in Preclinical and Phase 1 Clinical Studies

Antisense Oligonucleotide-induced Skipping of USH2A exon13 Restores Visual Function in Zebrafish

QRX-421, an Antisense Oligonucleotide (AON) Targeting Mutations in Exon 13 of USH2A , Associated with Retinitis Pigmentosa in Usher Syndrome Type 2, is Effective in Skipping Exon 13 in the USH2A mRNA of Patients Fibroblasts and Patient-Derived Optic Cups

Diabetes induced neurodegeneration in the retina and the brain of mice are associated and independent of microvasculopathy

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

Blockage of PI3K/mTOR Pathways Inhibits Laser-Induced Choroidal Neovascularization and Improves Outcomes Relative to VEGF-A Suppression Alone

Author Response: The Role of IL-18 in the Treatment of AMD

Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration

IL-18 Immunotherapy for Neovascular AMD: Tolerability and Efficacy in Nonhuman Primates

Six months effective ocular drug levels and protection against laser CNV in a cynomolgus monkey after a single ocular administration of a biodegradable microsphere delivery system releasing a highly potent anti-VEGF domain antibody based molecule.

IL-18 immunotherapy for neovascular AMD; Tolerability and efficacy in non-human primates

Identification of a Human Clinical Candidate ASO Selectively Targeting the P23H Variant of Rhodopsin for the Treatment of P23H Rhodopsin-mediated ADRP

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