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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

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. 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:

Articles May 2021

Examining Whether AOSLO-Based Foveal Cone Metrics in Achromatopsia and Albinism Are Representative of Foveal Cone Structure

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Katie M. Litts; Erica N. Woertz; Niamh Wynne; Brian P. Brooks; Alicia Chacon; Thomas B. Connor, Jr.; Deborah Costakos; Alina Dumitrescu; Arlene V. Drack; Gerald A. Fishman; William W. Hauswirth; Christine N. Kay; Byron L. Lam; Michel Michaelides; Mark E. Pennesi; Kimberly E. Stepien; Sasha Strul; C. Gail Summers; Joseph Carroll
TAGS: achromatopsia, albinism, retinal cone, diagnostic imaging, mosaicism
Trans. Vis. Sci. Tech.. 2021; 10(6):22. doi: https://doi.org/10.1167/tvst.10.6.22

Includes: Supplemental Content

Articles January 2021

Optical Coherence Tomography Artifacts Are Associated With Adaptive Optics Scanning Light Ophthalmoscopy Success in Achromatopsia

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Katie M. Litts; Erica N. Woertz; Michalis Georgiou; Emily J. Patterson; Byron L. Lam; Gerald A. Fishman; Mark E. Pennesi; Christine N. Kay; William W. Hauswirth; Michel Michaelides; Joseph Carroll
TAGS: achromatopsia, artifacts, diagnostic imaging, ophthalmoscopy, optical coherence tomography, optics
Trans. Vis. Sci. Tech.. 2021; 10(1):11. doi: https://doi.org/10.1167/tvst.10.1.11

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2020

Baseline visual fields in the RUSH2A study: Associated factors and correlation with measures of disease severity

Jacque L Duncan; Wendi Liang; Isabelle S Audo; David G Birch; Joseph Carroll; Janet K. Cheetham; Laura R Erker; Frederick L Ferris; Elise Heon; Robert Hufnagel; Alessandro Iannaccone; Glenn J Jaffe; Christine N Kay; Maureen Maguire; Michel Michaelides; Mark E Pennesi

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):3364. doi:

ARVO Annual Meeting Abstract June 2020

Optical coherence tomography artifacts predict adaptive optics scanning light ophthalmoscopy success in achromatopsia

Katie M Litts; Erica N. Woertz; Rachel E Linderman; Byron L Lam; Gerald A Fishman; Mark E Pennesi; Christine N Kay; William W Hauswirth; Joseph Carroll

Invest. Ophthalmol. Vis. Sci.. 2020; 61(7):5271. doi:

ARVO Annual Meeting Abstract July 2019

Stargardt disease ALK-001 phase 2 clinical trial: 12-month interim data

Hendrik P Scholl; Stephen H Tsang; Christine Nichols Kay; Thomas B. Connor; Michael B Gorin; Paul S Bernstein; Byron L Lam; Zach Strecker; Justin Tyler Zaremba; Gabrielle DeBartolomeo; Ilyas Washington; Leonide Saad

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):1336. doi:

ARVO Annual Meeting Abstract July 2019

Anatomical and fixation parameters as predictors of reading speed (RS) in Stargardt Disease

Gabrielle DeBartolomeo; Justin Tyler Zaremba; Zach Strecker; Christine Nichols Kay; Stephen H Tsang; Thomas B. Connor; Michael B Gorin; Paul S Bernstein; Byron L Lam; Ilyas Washington; Hendrik P Scholl; Leonide Saad

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):1063. doi:

ARVO Annual Meeting Abstract July 2019

The Visual Function Index (VF-14) reflects a notable loss in Vision-Related Quality of Life (VRQOL) in Stargardt Disease

Justin Tyler Zaremba; Zach Strecker; Gabrielle DeBartolomeo; Stephen H Tsang; Christine Nichols Kay; Thomas B. Connor; Michael B Gorin; Paul S Bernstein; Byron L Lam; Ilyas Washington; Hendrik P Scholl; Leonide Saad

Invest. Ophthalmol. Vis. Sci.. 2019; 60(9):4970. doi:

ARVO Annual Meeting Abstract July 2018

Interocular symmetry of foveal cone topography in achromatopsia (ACHM)

Katie M Litts; Christopher S Langlo; Emily J Patterson; Rebecca Mastey; Jenna A Cava; Patrick May; Byron L Lam; Gerald A Fishman; Mark E Pennesi; Christine N. Kay; Alfredo Dubra; William W Hauswirth; Kathleen N Beasley; Jeffrey D Chulay; Joseph Carroll

Invest. Ophthalmol. Vis. Sci.. 2018; 59(9):4992. doi:

Articles June 2018

Intraobserver Repeatability and Interobserver Reproducibility of Ellipsoid Zone Measurements in Retinitis Pigmentosa

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Margaret R. Strampe; Alison L. Huckenpahler; Brian P. Higgins; Sergey Tarima; Alexis Visotcky; Kimberly E. Stepien; Christine N. Kay; Joseph Carroll
TAGS: retinitis pigmentosa, spectral domain-optical coherence tomography, usher syndrome
Trans. Vis. Sci. Tech.. 2018; 7(3):13. doi: https://doi.org/10.1167/tvst.7.3.13

Includes: Multimedia, Supplemental Content

ARVO Annual Meeting Abstract June 2017

Stargardt Disease Phase 2 Clinical Trial: Design and Baseline Characteristics

Hendrik P Scholl; Christine N Kay; Stephen H Tsang; Kimberly E Stepien; Thomas B Connor; Paul S Bernstein; Byron L Lam; Michael B Gorin; Ilyas Washington; Leonide Saad

Invest. Ophthalmol. Vis. Sci.. 2017; 58(8):4654. doi:

ARVO Annual Meeting Abstract September 2016

TEASE: a phase 2 clinical trial assessing the tolerability and effects of oral once-a-day ALK-001 on Stargardt disease

Hendrik P Scholl; Syed Mahmood Shah; Christine Nichols Kay; Stephen H Tsang; Kimberly E Stepien; Paul S Bernstein; Byron L Lam; Michael B Gorin; Ilyas Washington; Leonide Saad

Invest. Ophthalmol. Vis. Sci.. 2016; 57(12):2685. doi:

ARVO Annual Meeting Abstract September 2016

Assessing Cone Structure in Achromatopsia With AOSLO and SDOCT

Christopher S Langlo; Laura R Erker; Maria A Parker; Emily J Patterson; Byron L Lam; Christine Nichols Kay; Mark E Pennesi; Richard G Weleber; Gerald A Fishman; William W Hauswirth; Joseph Carroll

Invest. Ophthalmol. Vis. Sci.. 2016; 57(12) doi:

ARVO Annual Meeting Abstract September 2016

Assessing color vision in congenital achromatopsia

Emily J Patterson; Christopher S Langlo; Byron L Lam; Christine Nichols Kay; Mark E Pennesi; Richard G Weleber; Gerald A Fishman; William W Hauswirth; Joseph Carroll

Invest. Ophthalmol. Vis. Sci.. 2016; 57(12) doi:

Retina August 2016

Residual Foveal Cone Structure in CNGB3 -Associated Achromatopsia

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Christopher S. Langlo; Emily J. Patterson; Brian P. Higgins; Phyllis Summerfelt; Moataz M. Razeen; Laura R. Erker; Maria Parker; Frederick T. Collison; Gerald A. Fishman; Christine N. Kay; Jing Zhang; Richard G. Weleber; Paul Yang; David J. Wilson; Mark E. Pennesi; Byron L. Lam; John Chiang; Jeffrey D. Chulay; Alfredo Dubra; William W. Hauswirth; Joseph Carroll; for the ACHM-001 Study Group
TAGS: achromatopsia, retinal cone
Invest. Ophthalmol. Vis. Sci.. 2016; 57(10):3984-3995. doi: https://doi.org/10.1167/iovs.16-19313

Includes: Supplemental Content

Retina May 2016

Assessing Photoreceptor Structure in Retinitis Pigmentosa and Usher Syndrome

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Lynn W. Sun; Ryan D. Johnson; Christopher S. Langlo; Robert F. Cooper; Moataz M. Razeen; Madia C. Russillo; Alfredo Dubra; Thomas B. Connor, Jr; Dennis P. Han; Mark E. Pennesi; Christine N. Kay; David V. Weinberg; Kimberly E. Stepien; Joseph Carroll
TAGS: photoreceptors, retinal cone, retinitis pigmentosa, usher syndrome, parafoveal region
Invest. Ophthalmol. Vis. Sci.. 2016; 57(6):2428-2442. doi: https://doi.org/10.1167/iovs.15-18246

Includes: Supplemental Content

ARVO Annual Meeting Abstract June 2015

Gene therapy for a mouse model of Achromatopsia

Shanta Sarfare; Seok-Hong Min; Sanford L Boye; Shannon Elizabeth Boye; William W Hauswirth; Christine Nichols Kay

Invest. Ophthalmol. Vis. Sci.. 2015; 56(7 ):5469. doi:

ARVO Annual Meeting Abstract April 2014

Expression of CNGB3 provides long-term rescue in the cone-only CNGB3 x NRL double knock-out (DKO) mouse

Seok-Hong Min; Qing Ruan; Sanford L Boye; Shannon Boye; William W Hauswirth; Christine Nichols Kay

Invest. Ophthalmol. Vis. Sci.. 2014; 55(13):3309. 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

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

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)

Examining Whether AOSLO-Based Foveal Cone Metrics in Achromatopsia and Albinism Are Representative of Foveal Cone Structure

Optical Coherence Tomography Artifacts Are Associated With Adaptive Optics Scanning Light Ophthalmoscopy Success in Achromatopsia

Baseline visual fields in the RUSH2A study: Associated factors and correlation with measures of disease severity

Optical coherence tomography artifacts predict adaptive optics scanning light ophthalmoscopy success in achromatopsia

Stargardt disease ALK-001 phase 2 clinical trial: 12-month interim data

Anatomical and fixation parameters as predictors of reading speed (RS) in Stargardt Disease

The Visual Function Index (VF-14) reflects a notable loss in Vision-Related Quality of Life (VRQOL) in Stargardt Disease

Interocular symmetry of foveal cone topography in achromatopsia (ACHM)

Intraobserver Repeatability and Interobserver Reproducibility of Ellipsoid Zone Measurements in Retinitis Pigmentosa

Stargardt Disease Phase 2 Clinical Trial: Design and Baseline Characteristics

TEASE: a phase 2 clinical trial assessing the tolerability and effects of oral once-a-day ALK-001 on Stargardt disease

Assessing Cone Structure in Achromatopsia With AOSLO and SDOCT

Assessing color vision in congenital achromatopsia

Residual Foveal Cone Structure in CNGB3 -Associated Achromatopsia

Assessing Photoreceptor Structure in Retinitis Pigmentosa and Usher Syndrome

Gene therapy for a mouse model of Achromatopsia

Expression of CNGB3 provides long-term rescue in the cone-only CNGB3 x NRL double knock-out (DKO) mouse

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