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ARVO Annual Meeting Abstract June 2022

Findings on visual photosensitivity in two phase 1/2 clinical trials of subretinal gene therapy with AGTC-401 and AGTC-402 for CNGB3 and CNGA3 achromatopsia

Rachel M Huckfeldt; Jason Comander; Mark E Pennesi; Paul Yang; Andreas Lauer; Robert Sisk; Edward Averbukh; Eyal Banin; Ninel Z Gregori; Janet L Davis; Byron L Lam; Christine Nichols Kay; Jessica Ijams Wolfing Morgan; Joseph Carroll; Bright Senyo Ashimatey; Matthew Feinsod

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):450. doi:

ARVO Annual Meeting Abstract June 2022

Characterizing the refractive error during the first 2 decades of life in patients with congenital stationary night blindness

Austin David Igelman; Thomas Khuu; Alaa Tayyib; Milda Reith; Birgit Lorenz; Erin O'Neil; Shaden Yassin; Andrew Stephenson; Emily Krauss; Ogul Uner; Justin Grassmeyer; Paul Yang; Lorri Wilson; Douglas Fredrick; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):253 – A0107. doi:

ARVO Annual Meeting Abstract June 2022

Characterization of refractive error progression in patients with blue cone monochromacy

Thomas Huynh Khuu; Austin David Igelman; Paul Yang; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):1720 – F0038. doi:

ARVO Annual Meeting Abstract June 2022

Interim Safety Results in Two Phase 1/2 Open-label, Dose-escalation Clinical Trials of Subretinal Gene Therapy with AGTC-401 (rAAV2tYF-PR1.7-hCNGB3) and AGTC-402 (rAAV2tYF-PR1.7-hCNGA3) in Subjects with Achromatopsia (ACHM)

Alessandro Iannaccone; Mark E Pennesi; Paul Yang; Andreas Lauer; Robert Sisk; Ninel Z Gregori; Janet L Davis; Byron L Lam; Christine Nichols Kay; Mauro Goldbaum; Bright Senyo Ashimatey; Feng Zhu; Matthew Feinsod; Lejla Vajzovic

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):2829 – A0345. doi:

ARVO Annual Meeting Abstract June 2022

18 Month Analysis of Macular Structure using Optical Coherence Tomography (OCT) from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa

Paul Yang; Andreas Lauer; Mark E Pennesi; David G Birch; Rand Spencer; Robert Sisk; Alessandro Iannaccone; Matthew Feinsod; Mauro Goldbaum

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):4399 – F0078. doi:

ARVO Annual Meeting Abstract June 2022

Commercially available, unmodified Octopus perimeters can characterize rod-photoreceptor function in two-color dark-adapted perimetry

Lesley Everett; Austin David Igelman; Jason Changbum Park; Robert Alexander Hyde; J Jason McAnany; Paul Yang; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2022; 63(7):2246 – F0454. doi:

Articles December 2021

Effect of Pharmacological Pupil Dilation on Dark-Adapted Perimetric Sensitivity in Healthy Subjects Using an Octopus 900 Perimeter

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Austin D. Igelman; Cristy Ku; Sam Mershon; Mariana Matioli da Palma; J. Jason McAnany; Robert A. Hyde; Jason C. Park; Paul Yang; Mark E. Pennesi
TAGS: color, dilatation, pathologic, mydriasis, perimetry, pharmacology, pupil dilation, octopus
Trans. Vis. Sci. Tech.. 2021; 10(14):18. doi: https://doi.org/10.1167/tvst.10.14.18

Genetics June 2021

Characterization of the Spectrum of Ophthalmic Changes in Patients With Alagille Syndrome

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Mariana Matioli da Palma; Austin D. Igelman; Cristy Ku; Amanda Burr; Jia Yue You; Emily M. Place; Nan-Kai Wang; Jin Kyun Oh; Kari E. Branham; Xinxin Zhang; Jeeyun Ahn; Michael B. Gorin; Byron L. Lam; Cecinio C. Ronquillo; Paul S. Bernstein; Aaron Nagiel; Rachel Huckfeldt; Michelle T. Cabrera; John P. Kelly; Benjamin Bakall; Alessandro Iannaccone; Robert B. Hufnagel; Wadih M. Zein; Robert K. Koenekoop; David G. Birch; Paul Yang; Abigail T. Fahim; Mark E. Pennesi
TAGS: arteriohepatic dysplasia, eye
Invest. Ophthalmol. Vis. Sci.. 2021; 62(7):27. doi: https://doi.org/10.1167/iovs.62.7.27

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2021

Phenotypic and genotypic spectrum of HGSNAT-related retinopathy

Mariana Matioli da Palma; Austin D. Igelman; Amanda Burr; Cristy Ann Ku; Paul Yang; Juliana M.F. Sallum; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):3225. doi:

ARVO Annual Meeting Abstract June 2021

Six Month Findings from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Show Clinically Meaningful Improvement in Macular Sensitivity

Paul Yang; Andreas Lauer; Mark E Pennesi; David G Birch; rand spencer; Robert A Sisk; Alessandro Iannaccone; Erin Leone; Deanine Halliman; Nnenna Ihekoromadu; Matthew Feinsod

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):1481. doi:

ARVO Annual Meeting Abstract June 2021

Twelve-month Findings from Two Phase 1/2 Clinical Studies of Subretinal Gene Therapy Drugs for Achromatopsia

Mark E Pennesi; Paul Yang; Andreas Lauer; Robert A Sisk; Jason M Comander; Rachel M. Huckfeldt; Edward Averbuhk; Eyal Banin; Ninel Gregori; Janet L Davis; Byron L Lam; Christine Kay; Erin Leone; Deanine Halliman; Nnenna Ihekoromadu; Matthew Feinsod

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):3321. doi:

ARVO Annual Meeting Abstract June 2021

Treatment outcomes in 11 patients with RPE65- retinopathy receiving voritegene neparvovec-rzyl

Cristy Ann Ku; Mariana Matioli da Palma; Austin Igelman; Andreas Lauer; Steven Bailey; Jacque L Duncan; Paul Yang; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):3320. doi:

ARVO Annual Meeting Abstract June 2021

Effect of pupil dilation on dark adapted perimetry using an Octopus 900 perimeter

Austin Igelman; Cristy Ann Ku; Mariana Matioli da Palma; Paul Yang; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2021; 62(8):499. doi:

Retina November 2020

Genotype Phenotype Correlation and Variability in Microcephaly Associated With Chorioretinopathy or Familial Exudative Vitreoretinopathy

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Maria F. Shurygina; Joseph M. Simonett; Maria A. Parker; Amanda Mitchell; Florin Grigorian; Jacob Lifton; Aaron Nagiel; Alexander A. Shpak; Elena L. Dadali; Irina A. Mishina; Richard G. Weleber; Paul Yang; Mark E. Pennesi

Invest. Ophthalmol. Vis. Sci.. 2020; 61(13):2. doi: https://doi.org/10.1167/iovs.61.13.2

Includes: Supplemental Content

Articles October 2020

A Ketogenic & Low-Protein Diet Slows Retinal Degeneration in rd10 Mice

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Renee C. Ryals; Samuel J. Huang; Dahlia Wafai; Claire Bernert; William Steele; Makayla Six; Shasank Bonthala; Hope Titus; Paul Yang; Melanie Gillingham; Mark E. Pennesi
TAGS: diet, diet, protein-restricted, mice, proteins, retina, retinal degeneration, electroretinography, serum albumin
Trans. Vis. Sci. Tech.. 2020; 9(11):18. doi: https://doi.org/10.1167/tvst.9.11.18

Includes: Multimedia, Supplemental Content

Retina August 2020

Suppression of cGMP-Dependent Photoreceptor Cytotoxicity With Mycophenolate Is Neuroprotective in Murine Models of Retinitis Pigmentosa

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Paul Yang; Rachel Lockard; Hope Titus; Jordan Hiblar; Kyle Weller; Dahlia Wafai; Richard G. Weleber; Robert M. Duvoisin; Catherine W. Morgans; Mark E. Pennesi
TAGS: cyclic gmp, cytotoxicity, mice, photoreceptors, mycophenolate mofetil, retinitis pigmentosa
Invest. Ophthalmol. Vis. Sci.. 2020; 61(10):25. doi: https://doi.org/10.1167/iovs.61.10.25

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2020

Neuroprotective Mechanisms of Dark Rearing on cGMP in rd10 mice

Paul Yang; Hope Titus; Jordan Hiblar; Dahlia Wafai; Robert M Duvoisin; Richard G Weleber; Catherine W Morgans; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):2482. doi:

Review June 2020

Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium

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Debra A. Thompson; Alessandro Iannaccone; Robin R. Ali; Vadim Y. Arshavsky; Isabelle Audo; James W. B. Bainbridge; Cagri G. Besirli; David G. Birch; Kari E. Branham; Artur V. Cideciyan; Steven P. Daiger; Deniz Dalkara; Jacque L. Duncan; Abigail T. Fahim; John G. Flannery; Roberto Gattegna; John R. Heckenlively; Elise Heon; K. Thiran Jayasundera; Naheed W. Khan; Henry Klassen; Bart P. Leroy; Robert S. Molday; David C. Musch; Mark E. Pennesi; Simon M. Petersen-Jones; Eric A. Pierce; Rajesh C. Rao; Thomas A. Reh; Jose A. Sahel; Dror Sharon; Paul A. Sieving; Enrica Strettoi; Paul Yang; David N. Zacks; The Monaciano Consortium
TAGS: outcome measures, retinal diseases, surrogate endpoints, genetics, vision, natural history, ethics, gene therapy
Trans. Vis. Sci. Tech.. 2020; 9(7):2. doi: https://doi.org/10.1167/tvst.9.7.2

ARVO Annual Meeting Abstract July 2019

An analysis of intraoperative optical coherence tomography findings in subretinal gene therapy surgery

Huber Vasconcelos Junior; Paul Yang; Mark E Pennesi; Brandon J Lujan; Andreas K Lauer

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):6399. doi:

ARVO Annual Meeting Abstract July 2019

Expanded clinical spectrum of pentosan polysulfate sodium-associated pigmentary maculopathy

Adam Marcus Hanif; Stanford Taylor; Stephen Armenti; Rachel Shah; Austin Igelman; Mark E Pennesi; Thiran Jayasundera; Paul Yang; Thomas S Hwang; Christina J Flaxel; Rahul Khurana; Joshua D Stein; Nieraj Jain

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):3245. doi:

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Findings on visual photosensitivity in two phase 1/2 clinical trials of subretinal gene therapy with AGTC-401 and AGTC-402 for CNGB3 and CNGA3 achromatopsia

Characterizing the refractive error during the first 2 decades of life in patients with congenital stationary night blindness

Characterization of refractive error progression in patients with blue cone monochromacy

Interim Safety Results in Two Phase 1/2 Open-label, Dose-escalation Clinical Trials of Subretinal Gene Therapy with AGTC-401 (rAAV2tYF-PR1.7-hCNGB3) and AGTC-402 (rAAV2tYF-PR1.7-hCNGA3) in Subjects with Achromatopsia (ACHM)

18 Month Analysis of Macular Structure using Optical Coherence Tomography (OCT) from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa

Commercially available, unmodified Octopus perimeters can characterize rod-photoreceptor function in two-color dark-adapted perimetry

Effect of Pharmacological Pupil Dilation on Dark-Adapted Perimetric Sensitivity in Healthy Subjects Using an Octopus 900 Perimeter

Characterization of the Spectrum of Ophthalmic Changes in Patients With Alagille Syndrome

Phenotypic and genotypic spectrum of HGSNAT-related retinopathy

Six Month Findings from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Show Clinically Meaningful Improvement in Macular Sensitivity

Twelve-month Findings from Two Phase 1/2 Clinical Studies of Subretinal Gene Therapy Drugs for Achromatopsia

Treatment outcomes in 11 patients with RPE65- retinopathy receiving voritegene neparvovec-rzyl

Effect of pupil dilation on dark adapted perimetry using an Octopus 900 perimeter

Genotype Phenotype Correlation and Variability in Microcephaly Associated With Chorioretinopathy or Familial Exudative Vitreoretinopathy

A Ketogenic & Low-Protein Diet Slows Retinal Degeneration in rd10 Mice

Suppression of cGMP-Dependent Photoreceptor Cytotoxicity With Mycophenolate Is Neuroprotective in Murine Models of Retinitis Pigmentosa

Neuroprotective Mechanisms of Dark Rearing on cGMP in rd10 mice

Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium

An analysis of intraoperative optical coherence tomography findings in subretinal gene therapy surgery

Expanded clinical spectrum of pentosan polysulfate sodium-associated pigmentary maculopathy

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