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ARVO Annual Meeting Abstract June 2022

Impaired Cathepsin D evidenced in iPSC-derived RPE cells of STGD1 patients

Eunice Sze Yin Ng; Jane Hu; Katherine Frei; Zhichun Jiang; Michael B Gorin; Anna Matynia; Roxana A Radu

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):3894 – A0096. doi:

ARVO Annual Meeting Abstract June 2022

Preliminary Results from a First-in-human Phase I/II Gene Therapy Study (FOCUS) of Subretinally Delivered GT005, an Investigational AAV2 Vector, in Patients with Geographic Atrophy Secondary to Age-related Macular Degeneration

Jared Nielsen; Robert E MacLaren; Jeffrey S Heier; David Steel; Tsveta Ivanova; Sobha Sivaprasad; Paulo Stanga; Clare Bailey; Peter Charbel Issa; Luisa Mendonca; James Francis; Darin Curtiss; Jane Hughes; Nadia K Waheed

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):1504. doi:

ARVO Annual Meeting Abstract June 2022

Preclinical pharmacology and safety of GT005, an investigational gene therapy targeting the complement pathway for the treatment of Geographic Atrophy

Julian Esteve-Rudd; James Francis; Jane Hughes; Scott Ellis

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):294 – F0097. doi:

ARVO Annual Meeting Abstract June 2022

In vivo expression and efficacy following subretinal delivery of GT005, an investigational gene therapy for the treatment of geographic atrophy (GA)

Dimitrios Stampoulis; Josephine Joel; Anna Dreismann; Emanuela Gardenal; Jane Hughes; Scott Ellis; Julian Esteve-Rudd

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):293 – F0096. doi:

Articles March 2022

Assessing Variant Causality and Severity Using Retinal Pigment Epithelial Cells Derived from Stargardt Disease Patients

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Anna Matynia; Jun Wang; Sangbae Kim; Yumei Li; Anupama Dimashkie; Zhichun Jiang; Jane Hu; Samuel P. Strom; Roxana A. Radu; Rui Chen; Michael B. Gorin
TAGS: abca4 gene, proteins, retinal pigment cell, stargardt's disease, pathogenicity, idiopathic pneumonia syndrome, alleles
Trans. Vis. Sci. Tech.. 2022; 11(3):33. doi: https://doi.org/10.1167/tvst.11.3.33

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2021

MeCP2-mediated mitochondrial impairment evidenced in Stargardt disease

Arpita R. Dave; Eunice Sze Yin Ng; Zhichun Jiang; Jane G Hu; Michael B Gorin; Anna Matynia; Linsey Stiles; Roxana A Radu

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):269. doi:

ARVO Annual Meeting Abstract June 2021

Cathepsin D deficiency in RPE cells mediates Stargardt Disease pathogenesis

Eunice Sze Yin Ng; Jane G Hu; Zhichun Jiang; Michael B Gorin; Anna Matynia; Roxana A Radu

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):3113. doi:

ARVO Annual Meeting Abstract June 2021

Membrane attack complex induces RPE cell death in Stargardt Disease

Roxana A Radu; Nermin Kady; Michael B Gorin; Anna Matynia; Jane G Hu

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):2994. doi:

Retinal Cell Biology July 2020

Geographic Atrophy: Confocal Scanning Laser Ophthalmoscopy, Histology, and Inflammation in the Region of Expanding Lesions

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Vera L. Bonilha; Brent A. Bell; Jane Hu; Caroline Milliner; Gayle J. Pauer; Stephanie A. Hagstrom; Roxana A. Radu; Joe G. Hollyfield
TAGS: atrophy, diagnostic imaging, donors, eye, granules, histology, inflammation, scanning laser ophthalmoscopy, body tissue, retina
Invest. Ophthalmol. Vis. Sci.. 2020; 61(8):15. doi: https://doi.org/10.1167/iovs.61.8.15

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2020

Complement Dysregulation is Evidenced in iPSC-derived RPE Cells from Stargardt Disease patients

Jane Hu; Nermin Kady; Ashley Macabasco; Michael B Gorin; Anna Matynia; Roxana A Radu

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):1507. doi:

ARVO Annual Meeting Abstract June 2020

GT005, a gene therapy for the treatment of dry age-related macular degeneration (AMD)

Scott Ellis; Anna Buchberger; Julie Holder; elise Orhan; Jane Hughes

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):2295. doi:

ARVO Annual Meeting Abstract June 2020

Proteomic analysis of vitreous humor samples obtained from donors with and without age-related macular degeneration (AMD) reveal an overactivation of complement protein expression.

Anna Buchberger; James Francis; Ashley Frazer-Abel; Selina McHarg; Paul N Bishop; Scott Ellis; Jane Hughes

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):3650. doi:

ARVO Annual Meeting Abstract July 2019

Reduced expression of “normal” ABCA4 alleles in iPS-derived RPE cells from Stargardt Disease patients

Anna Matynia; Saravanan Karumbayaram; Samuel Strom; Jane Hu; Nermin Kady; Roxana A Radu; Michael B Gorin

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):388. doi:

ARVO Annual Meeting Abstract July 2019

Disease mechanisms elucidated by genetic regulation of human RPE gene expression

Douglas Vollrath; Boxiang Liu; Melissa A Calton; Nathan S. Abell; Gillie Benchorin; Michael J. Gloudemans; Ming Chen; Jane Hu; Xin Li; Brunilda Balliu; Dean Bok; Stephen B. Montgomery

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):1759. doi:

ARVO Annual Meeting Abstract July 2019

Retinoid recycling is impaired in Stargardt iPSC-derived RPE

Roxana A Radu; Nermin Kady; Jane Hu; Jacqueline Pei; Marcia Lloyd; Dean Bok; Michael B Gorin; Saravanan Karumbayaram; Anna Matynia

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):2343. doi:

ARVO Annual Meeting Abstract July 2018

Characterization of Stargardt iPSC-derived RPE cells

Nermin Kady; Anna Matynia; Saravanan Karumbayaram; Jane Hu; Marcia Lloyd; Dean Bok; Michael B Gorin; Roxana A. Radu

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):3045. doi:

ARVO Annual Meeting Abstract July 2018

Localization of ABCA4 in the Retinal Pigment Epithelium and its implications for Stargardt Disease

Tamara Lee Lenis; Sze Yin Ng; Jane Hu; Zhichun Jiang; Marcia Lloyd; Nicholas Esposito; Dean Bok; Silvia C Finnemann; Gabriel H Travis; Roxana A. Radu

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):4502. doi:

Vision Sciences Society Annual Meeting Abstract August 2017

What enhances/reduces holistic processing in perceptual expertise: experience in writing/drawing versus component composition

Ricky Van-yip Tso; Wai-ming Cheung; Terry Kit-fong Au; Janet Hui-wen Hsiao

Journal of Vision. 2017; 17(10):1039. doi: https://doi.org/10.1167/17.10.1039

ARVO Annual Meeting Abstract June 2017

Expression of ABCA4 in Retinal Pigment Epithelium cells and its implications for Stargardt disease

Tamara L Lenis; Shanta Sarfare; Jane Hu; Zhichun Jiang; Marcia Lloyd; Dean Bok; Gabriel H Travis; Roxana A Radu

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):607. doi:

ARVO Annual Meeting Abstract June 2017

Elevated intraocular pressure following silicone oil injection for complicated retinal detachments

Hiroaki Ozaki; Jane Yuh-Huey Huang; Ryota Ko; Eiichi Uchio

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):4173. doi:

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Impaired Cathepsin D evidenced in iPSC-derived RPE cells of STGD1 patients

Preliminary Results from a First-in-human Phase I/II Gene Therapy Study (FOCUS) of Subretinally Delivered GT005, an Investigational AAV2 Vector, in Patients with Geographic Atrophy Secondary to Age-related Macular Degeneration

Preclinical pharmacology and safety of GT005, an investigational gene therapy targeting the complement pathway for the treatment of Geographic Atrophy

In vivo expression and efficacy following subretinal delivery of GT005, an investigational gene therapy for the treatment of geographic atrophy (GA)

Assessing Variant Causality and Severity Using Retinal Pigment Epithelial Cells Derived from Stargardt Disease Patients

MeCP2-mediated mitochondrial impairment evidenced in Stargardt disease

Cathepsin D deficiency in RPE cells mediates Stargardt Disease pathogenesis

Membrane attack complex induces RPE cell death in Stargardt Disease

Geographic Atrophy: Confocal Scanning Laser Ophthalmoscopy, Histology, and Inflammation in the Region of Expanding Lesions

Complement Dysregulation is Evidenced in iPSC-derived RPE Cells from Stargardt Disease patients

GT005, a gene therapy for the treatment of dry age-related macular degeneration (AMD)

Proteomic analysis of vitreous humor samples obtained from donors with and without age-related macular degeneration (AMD) reveal an overactivation of complement protein expression.

Reduced expression of “normal” ABCA4 alleles in iPS-derived RPE cells from Stargardt Disease patients

Disease mechanisms elucidated by genetic regulation of human RPE gene expression

Retinoid recycling is impaired in Stargardt iPSC-derived RPE

Characterization of Stargardt iPSC-derived RPE cells

Localization of ABCA4 in the Retinal Pigment Epithelium and its implications for Stargardt Disease

What enhances/reduces holistic processing in perceptual expertise: experience in writing/drawing versus component composition

Expression of ABCA4 in Retinal Pigment Epithelium cells and its implications for Stargardt disease

Elevated intraocular pressure following silicone oil injection for complicated retinal detachments

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