Recent Publications

• Bi-allelic pathogenic variants in NR2E3 may be associated with a subtle enhanced S-cone syndrome phenotype.
  Ophthalmic genetics. 2026 Aug;47(4):328-334 Epub 2026 Mar 30; Authors: Hüther A, Sherman CL, Sumaroka A, O'Neil EC, Roman AJ, Kim R, Weber ML, Garafalo AV, Cideciyan AV, Aleman TS

• PRCD-associated retinitis pigmentosa in dogs and humans.
  Experimental eye research. 2026 Aug;269:111045 Epub 2026 May 8; Authors: Dufour VL, Sato Y, Sumaroka A, Sudharsan R, Banin E, Yu Y, Ripolles-Garcia A, Kwok JC, Aleman TS, Ying GS, Sharon D, Cideciyan AV, Beltran WA, Aguirre GD

• Inherited retinal degenerations: clinical phenotypes and emerging therapies.
  Lancet (London, England). 2026 Jul 25;408(10552):367-377 Epub 2026 Jul 6; Authors: Uyhazi KE, O'Neil EC, Aleman TS, Cideciyan AV

• Interim Results From a Multicenter Observational Safety Study of Patients Treated With Voretigene Neparvovec-rzyl in the United States.
  Ophthalmology. 2026 Jul 22; Authors: Nagiel A, Russell SR, Lam BL, Comander JI, Aleman TS, Besirli CG, Sisk RA, Yang P, Weng CY, Chung DC, Thakre A, Savola JM, Rousso DL, VN PASS Study Group

• Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing BEST1 Delivered by Subretinal Injection in a Canine Model of Human Bestrophinopathy.
  Human gene therapy. 2026 Jul 6;:10430342261458076; Authors: Gray A, Kwok JC, Sato Y, Assenmacher CA, Choudhary M, Jayagopal A, Hua P, Ying GS, Swider M, Sumaroka A, Cideciyan AV, Gustavo AD, Beltran WA

• INTRAFAMILIAL VARIABILITY OF IMPG1 -ASSOCIATED VITELLIFORM DYSTROPHY.
  Retinal cases & brief reports. 2026 Jul 1;20(4):510-514; Authors: Chan L, Adams M, Aleman TS, Bloom SM

• Lens Opacity in ABCA4-Associated Stargardt Disease Patients in the ProgStar Study.
  Investigative ophthalmology & visual science. 2026 Jul 1;67(8):47; Authors: Ibukun F, Chan BXJ, Khan J, Wang Y, Ervin AM, Kühlewein L, Strauss RW, Aychoua N, Birch D, Cideciyan A, Singh M, Traboulsi E, Bernstein P, Michaelides M, Kong X

• Clinical endpoints special session, ISCEV 2025, Utrecht.
  Documenta ophthalmologica. Advances in ophthalmology. 2026 May 20; Authors: Hamilton R, Birch D, Stingl K, Fujinami K, Holopigian K, Boon CJF, Moseley J, McAnany JJ, Cideciyan AV, van Genderen MM

• Longitudinal Changes of Fundus Autofluorescence and Correlation With Visual Acuity in ABCA4-Associated Stargardt Disease.
  Investigative ophthalmology & visual science. 2026 Mar 2;67(3):6; Authors: Ibukun F, Strauss RW, Rustam Z, Khan MJ, Cideciyan AV, Birch DG, Ho A, Sunness JS, Audo IS, Michaelides M, Zrenner E, Traboulsi EI, Bernstein P, Sadda S, Ip MS, West S, Kong X

• Measuring Vision in Children Undergoing Retinal Gene Therapy.
  JAMA ophthalmology. 2026 Feb 1;144(2):165-166; Authors: Aleman TS, Cideciyan AV

• A Spectrum of Severity of a Unifying Retinal Phenotype in TUBB4B-Associated Inherited Retinal Degeneration.
  Retinal cases & brief reports. 2026 Jan 22; Authors: Zhang S, Avery RA, Wu V, Sumaroka A, Kim RJ, Maripuri DP, Datz E, Weber ML, Roman AJ, Tsang SH, Cideciyan AV, Aleman TS, O'Neil EC

• Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial.
  Molecular therapy : the journal of the American Society of Gene Therapy. 2025 Oct 1;33(10):4784-4798 Epub 2025 Jul 1; Authors: Aleman TS, Uyhazi KE, Roman AJ, Weber ML, O'Neil EC, Swider M, Sumaroka A, Maguire KH, Aleman EM, Santos AJ, Kim RJ, Parchinski KM, Billek A, Fradin M, Chung W, Margaritis P, Sun J, Scoles DH, Wu V, Garafalo AV, Jayagopal A, Yerxa B, Tuller S, Maguire AM, Bennett J, Cideciyan AV

• Measuring Rod- and Cone-Photoreceptor-Specific Vision in Inherited Retinal Diseases Using a Commercial Perimeter.
  Investigative ophthalmology & visual science. 2025 Oct 1;66(13):31; Authors: Wu V, Roman AJ, Galsterer EL, Ansari G, Erdinest I, Righetti G, Viarbitskaya I, Russell RC, Kim RJ, Charlier J, Pfau K, Stingl K, Banin E, Pfau M, Stingl K, Aleman TS, Cideciyan AV

• Location and Identity of Photoreceptors Contributing to the Full-Field Stimulus Test (FST).
  Investigative ophthalmology & visual science. 2025 Oct 1;66(13):46; Authors: Cideciyan AV, Neuhauser H, Roman AJ, Aleman TS