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Genetics January 2025

Safety of Human USH1C Transgene Expression Following Subretinal Injection in Wild-Type Pigs

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Peter Kiraly; Joshua Klein; Immanuel P. Seitz; Felix F. Reichel; Tobias Peters; Taras Ardan; Jana Juhasova; Stefan Juhás; Zdenka Ellederova; Yaroslav Nemesh; Ruslan Nyshchuk; Nikolai Klymiuk; Kerstin Nagel-Wolfrum; Ashley R. Winslow; Uwe Wolfrum; Jan Motlik; M. Dominik Fischer
TAGS: disease vectors, gene therapy, injections, safety, spatial vectors, suidae, transgenes, retina, histopathology tests, electroretinography
Invest. Ophthalmol. Vis. Sci.. 2025; 66(1):48. https://doi.org/10.1167/iovs.66.1.48

Includes: Supplemental Content

Retina October 2024

Optimizing Subretinal Bleb Formation for Visual Streak Involvement in a Porcine Model for Retinal Gene Therapy

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Immanuel P. Seitz; Tobias Peters; Felix Reichel; Andrea Bähr; Hanna Auch; Jan Motlik; Taras Ardan; Jana Juhasova; Yaroslav Nemesh; Stefan Juhás; Nikolai Klymiuk; Manuel Dominik Fischer
TAGS: blister, eye, gene therapy, injection site, injections, suidae, anisotropy
Invest. Ophthalmol. Vis. Sci.. 2024; 65(12):41. https://doi.org/10.1167/iovs.65.12.41

ARVO Annual Meeting Abstract June 2024

Retention rates of subretinal gene therapeutic agents based on AAV serotype 2 and 8 using different drug-delivery materials

Felix Reichel; Peter Kiraly; Immanuel Philipp Seitz; M Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2024; 65(7):911.

ARVO Annual Meeting Abstract June 2024

Real-world safety and effectiveness of voretigene neparvovec: Year 3 interim results from the PERCEIVE study

M Dominik Fischer; Francesca Simonelli; Isabelle S Audo; Bart Peter Leroy; Line Kessel; Joao Pedro Marques; Mirjana Bjelos; James Bainbridge; Manar Aoun; Rainer Maier; Rehna Khan; Andreas Clemens; Frank G Holz

Invest. Ophthalmol. Vis. Sci.. 2024; 65(7):3290.

ARVO Annual Meeting Abstract June 2024

Host cell protein impurities are not a relevant driver of gene therapy-associated immune response

Immanuel Philipp Seitz; Eduardo Bocanegra; Felix Reichel; Stylianos Michalakis; Martin Biel; Karl Ulrich Bartz-Schmidt; Tobias Peters; M Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2024; 65(7):5345.

ARVO Annual Meeting Abstract June 2024

A new subretinal gene therapy for USH1C is well tolerated in wild type pigs

Peter Kiraly; Matthias Mai; Tobias Peters; Immanuel P Seitz; Taras Ardan; Zdenka Ellederova; Yaroslav Nemesh; Ruslan Nyshchuk; Nikolai Klymiuk; Ashley Winslow; Joshua Klein; Kerstin Nagel-Wolfrum; Uwe Wolfrum; Jan Motlik; M Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2024; 65(7):5341.

ARVO Annual Meeting Abstract June 2024

Pro Re Nata versus treat-and-extend anti-VEGF dosing strategies for the treatment of macular oedema in retinal vein occlusion: 12-month outcomes

Vasil Kostin; Martin Horak; Wiktoria Zamirska; Yasmin Bakr; M Dominik Fischer; Peter Charbel Issa; Christine Kiire; Niamh Stone; Samantha R De Silva

Invest. Ophthalmol. Vis. Sci.. 2024; 65(7):6303.

ARVO Annual Meeting Abstract June 2024

Slowly degenerating retina of Rpgr- deficient mouse remain immunologically quiescent

Molly John; Cristina Martinez-Fernandez Dela Camara; Federica Staurenghi; M Dominik Fischer; Kanmin Xue; Robert E MacLaren

Invest. Ophthalmol. Vis. Sci.. 2024; 65(7):5051.

ARVO Annual Meeting Abstract June 2023

Evidence of potential early-stage ocular tertiary lymphoid tissue formation following high dose retinal AAV8 gene therapy

Kirsten Bucher; Daniela Süsskind; Martin Biel; Stylianos Michalakis; Karl-Ulrich Bartz-Schmidt; Tobias Peters; M Dominik Fischer; Simon J Clark

Invest. Ophthalmol. Vis. Sci.. 2023; 64(8):3911.

ARVO Annual Meeting Abstract June 2023

Functional outcomes and adverse effects of voretigene neparvovec for biallelic RPE65-mediated inherited retinal dystrophies in a cohort of patients from a single centre

Peter Kiraly; Charles Cottriall; Laura J Taylor; Jasleen K Jolly; Jasmina Cehajic-Kapetanovic; Imran H Yusuf; Cristina Martinez-Fernandez dela Camara; Morag Shanks; Susan M Downes; Robert E MacLaren; Dominik M Fischer

Invest. Ophthalmol. Vis. Sci.. 2023; 64(8):801.

ARVO Annual Meeting Abstract June 2023

Safety and Vision Outcomes of subretinal gene supplementation therapy in PDE6A -associated Retinitis pigmentosa

Felix Friedrich Lambert Reichel; Stylianos Michalakis; Dominik M Fischer; laura kuehlewein; Tobias Peters; Susanne Kohl; Karl-Ulrich Bartz-Schmidt; Marius Ueffing; Eberhart Zrenner; Martin Biel; Katarina Stingl; Bernd Wissinger

Invest. Ophthalmol. Vis. Sci.. 2023; 64(8):782.

ARVO Annual Meeting Abstract June 2023

Optimizing Subretinal Bleb Formation in a Porcine Model for Retinal Gene Therapy

Immanuel Philipp Seitz; Tobias Peters; Bähr Andrea; Hannah Auch; Motlik Jan; Taras Ardan; Yaroslav Nemesh; Stefan Juhas; Nikolai Klymiuk; Dominik M Fischer

Invest. Ophthalmol. Vis. Sci.. 2023; 64(8):778.

ARVO Annual Meeting Abstract June 2022

Neural plasticity of the human visual system reflects treatment effect in gene therapy for Achromatopsia

Immanuel Philipp Seitz; Michael Erb; Thomas Ethofer; Andriani Rina; Georgios Keliris; M Dominik Fischer; Karl-Ulrich Bartz-Schmidt

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):452.

ARVO Annual Meeting Abstract June 2022

Development of a functional assay for the assessment of two common CRB1 mutations

Julia-Sophia Bellingrath; Michelle E. McClements; M. Dominik Fischer; Robert E MacLaren

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):1766 – F0315.

ARVO Annual Meeting Abstract June 2022

Generation of qualitative evidence to support the content validity of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis

Christine Nichols Kay; Isabelle S Audo; Helena Bradley; Melissa Barclay; Joel Sims; Kieran Boparai; Francesco Patalano; Christel Naujoks; Claudio Spera; Daniel Viriato; M Dominik Fischer; Jane Green; Todd Durham; Nicola Williamson; Judit Banhazi

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):1711 – F0029.

ARVO Annual Meeting Abstract June 2022

Psychometric validation of patient-reported outcome (PRO) and observer-reported outcome (ObsRO) measures in Retinitis Pigmentosa and Leber Congenital Amaurosis

Isabelle S Audo; Nicola Williamson; Melissa Barclay; Joel Sims; Kieran Boparai; Francesco Patalano; Christel Naujoks; Claudio Spera; Judit Banhazi; Paul O’Brien; Christine Kay; Jane Green; Todd Durham; Helena Bradley; M Dominik Fischer

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):2663.

ARVO Annual Meeting Abstract June 2022

Central Visual Function and Genotype-Phenotype Correlations in PDE6A -Associated Retinitis Pigmentosa in Preparation for a Gene Supplemental Trial

Ditta Zobor; Torsten Strasser; Gunnar Blumenstock; Katarina Stingl; M. Dominik Fischer; Barbara Wilhelm; Eberhart Zrenner; Bernd Wissinger; Susanne Kohl; Nicole Weisschuh; Laura Kuehlewein

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):4499 – F0286.

ARVO Annual Meeting Abstract June 2022

PERCEIVE study report: Real-world safety and effectiveness of voretigene neparvovec

M Dominik Fischer; Rainer Maier; Andrea Suhner; Daniel Stiehl; Christina Fasser; Bart P Leroy

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):451.

Genetics May 2022

Central Visual Function and Genotype–Phenotype Correlations in PDE6A -Associated Retinitis Pigmentosa

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Laura Kuehlewein; Torsten Straßer; Gunnar Blumenstock; Katarina Stingl; M. Dominik Fischer; Barbara Wilhelm; Eberhart Zrenner; Bernd Wissinger; Susanne Kohl; Nicole Weisschuh; Ditta Zobor; for the RD-CURE Consortium
TAGS: genotype, genotype-phenotype associations, pde6a gene, phenotype, retinitis pigmentosa, vision, color vision, genetics, eye
Invest. Ophthalmol. Vis. Sci.. 2022; 63(5):9. https://doi.org/10.1167/iovs.63.5.9

ARVO Annual Meeting Abstract June 2021

NIGHT study: natural progression of choroideremia

Byron L Lam; Robert E MacLaren; M. Dominik Fischer; Frank G Holz; Mark E Pennesi; David G Birch; Eeva-Marja Sankila; Isabelleanne Meunier; Kimberly E Stepien; Juliana M.F. Sallum; Cindy Lu; Jiajun Liu; Dan Yoon

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):34.

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Safety of Human USH1C Transgene Expression Following Subretinal Injection in Wild-Type Pigs

Optimizing Subretinal Bleb Formation for Visual Streak Involvement in a Porcine Model for Retinal Gene Therapy

Retention rates of subretinal gene therapeutic agents based on AAV serotype 2 and 8 using different drug-delivery materials

Real-world safety and effectiveness of voretigene neparvovec: Year 3 interim results from the PERCEIVE study

Host cell protein impurities are not a relevant driver of gene therapy-associated immune response

A new subretinal gene therapy for USH1C is well tolerated in wild type pigs

Pro Re Nata versus treat-and-extend anti-VEGF dosing strategies for the treatment of macular oedema in retinal vein occlusion: 12-month outcomes

Slowly degenerating retina of Rpgr- deficient mouse remain immunologically quiescent

Evidence of potential early-stage ocular tertiary lymphoid tissue formation following high dose retinal AAV8 gene therapy

Functional outcomes and adverse effects of voretigene neparvovec for biallelic RPE65-mediated inherited retinal dystrophies in a cohort of patients from a single centre

Safety and Vision Outcomes of subretinal gene supplementation therapy in PDE6A -associated Retinitis pigmentosa

Optimizing Subretinal Bleb Formation in a Porcine Model for Retinal Gene Therapy

Neural plasticity of the human visual system reflects treatment effect in gene therapy for Achromatopsia

Development of a functional assay for the assessment of two common CRB1 mutations

Generation of qualitative evidence to support the content validity of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis

Psychometric validation of patient-reported outcome (PRO) and observer-reported outcome (ObsRO) measures in Retinitis Pigmentosa and Leber Congenital Amaurosis

Central Visual Function and Genotype-Phenotype Correlations in PDE6A -Associated Retinitis Pigmentosa in Preparation for a Gene Supplemental Trial

PERCEIVE study report: Real-world safety and effectiveness of voretigene neparvovec

Central Visual Function and Genotype–Phenotype Correlations in PDE6A -Associated Retinitis Pigmentosa

NIGHT study: natural progression of choroideremia

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