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The RReSTORe Consortium Publications

Below you will find publications which have arisen directly from the work of the RReSTORe Consortium, as well as featured publications from RReSTORe members that are pertinent to our mission of repopulating RGCs to regenerate the diseased visual pathway and restore vision to patients suffering from optic neuropathies.

Retinal ganglion cell repopulation for vision restoration in optic neuropathy: a roadmap from the RReSTORe Consortium.

Soucy JR, Aguzzi EA, Cho J, Gilhooley MJ, Keuthan C, Luo Z, Monavarfeshani A, Saleem MA, Wang XW, Wohlschlegel J; RReSTORe Consortium; Baranov P, Di Polo A, Fortune B, Gokoffski KK, Goldberg JL, Guido W, Kolodkin AL, Mason CA, Ou Y, Reh TA, Ross AG, Samuels BC, Welsbie D, Zack DJ, Johnson TV. Mol Neurodegener. 2023 Sep 21;18(1):64. doi: 10.1186/s13024-023-00655-y. PMID: 37735444

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The Retinal Ganglion Cell Repopulation, Stem Cell Transplantation, & Optic Nerve Regeneration (RReSTORe) Consortium.

Johnson TV, Baranov P, Di Polo A, Fortune B, Gokoffski KK, Goldberg JL, Guido W, Kolodkin AL, Mason CA, Ou Y, Reh TA, Ross AG, Samuels BC, Zack DJ. Ophthalmol Sci. 2023 Aug 24;3(4):100390. doi: 10.1016/j.xops.2023.100390. PMID: 38025164

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The importance of unambiguous cell origin determination in neuronal repopulation studies

Johnson TV, Calkins DJ, Fortune B, Goldberg JL, La Torre A, Lamba DA, Meyer JS, Reh TA, Wallace VA, Zack DJ, Baranov P. iScience. 2023 Mar 9;26(4):106361. doi: 10.1016/j.isci.2023.106361. eCollection 2023 Apr 21. PMID: 37009209

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Recent papers published by RReSTORe Members:

  • Abd Alhadi S, Camacho D, Chaqour B, Rossman JB, Pham JC, Shindler KS, Ross AG. Neuroprotective effects of SIRT1 in human RGCs derived from iPSCs following oxidative stress induction at early and late stages of differentiation. Exp Eye Res. 259:110526. (2025). PMID: 40681033
  • Aguzzi EA, Mary S, Mowlavi Vardanjani M, … Handa JT, Johnson TV. The internal limiting basement membrane inhibits functional engraftment of transplanted human retinal ganglion cells in vivo. Sci Transl Med. 18(847):eadr1062. (2026). PMID: 42054496
  • Alexandris AS, Yi J, Liu C, … Zack DJ, Koliatsos VE. Recovery of retinal terminal fields after traumatic brain injury: evidence of collateral sprouting and sexual dimorphism. J Neurosci. 46(5):e0792252025. (2026). PMID: 41397889
  • Baez HC, LaPorta JM, Walker AD, … Gullapalli V, McGregor JE. Inner limiting membrane peel extends in vivo calcium imaging of retinal ganglion cell activity beyond the fovea in non-human primate. Invest Ophthalmol Vis Sci. 66(12):25. (2025). PMID: 40928310
  • Bian M, Huie EL, Xia X, … Russano K, Goldberg JL. 3’UTR regulation of axon translation and optic nerve regeneration. Mol Neurodegener. 21(1):42. (2026). PMID: 42216210
  • Chaudhari K, Guzman-Clavel LE, Donthi N, Yang J, Harris SC, Lin TH, Dunn FA, Kolodkin AL. Temporally segregated functions for Gfi1 in the development and maintenance of direction selectivity in the vertebrate retina. Curr Biol. 36(15):3877. (2026). PMID: 42442355
  • Cueva Vargas JL, Belforte N, Vidal-Paredes IA, Dotigny F, Vande Velde C, Quintero H, Di Polo A. Stress-induced mitochondrial fragmentation in endothelial cells disrupts blood-retinal barrier integrity causing neurodegeneration. Mol Ther. 34(1):562. (2026). PMID: 40994007
  • Dunn M, Cull G, Reynaud J, Gardiner SK, Di Polo A, Fortune B. Microvascular volume loss exceeds nerve fiber layer but not neuroretinal rim tissue loss during progression of nonhuman primate experimental glaucoma. Invest Ophthalmol Vis Sci. 67(2):24. (2026). PMID: 41665300
  • Gomes C, Huang KC, Lavekar SS, … Zhang C, Meyer JS. Modeling human retinal ganglion cell axonal outgrowth, development, and pathology using pluripotent stem cell-based microfluidic platforms. Proc Natl Acad Sci U S A. 122(37):e2423682122. (2025). PMID: 40924455
  • Ha A, Kim YK, Lee CK, … Kim M, Rho S. Effects of nicotinamide supplementation in normal-tension glaucoma: a crossover placebo-controlled randomised clinical trial. Br J Ophthalmol. 110(4):417. (2026). PMID: 41167798
  • Harkin J, Gomes C, Afify R, … Huang KC, Meyer JS. A human pluripotent stem cell triculture platform to elucidate microglial regulation of retinal ganglion cells in neuroinflammation. Proc Natl Acad Sci U S A. 123(34):e2522958123. (2026). PMID: 42616788
  • Jaggers TN, Ripolles-Garcia A, Moshiri A, … Marsh-Armstrong N, Thomasy SM. Rhesus macaques with an OPA1 mutation demonstrate features of autosomal dominant optic atrophy. Proc Natl Acad Sci U S A. 123(16):e2509165123. (2026). PMID: 41984835
  • Kim T, Iseri E, Peng MG, … Lazzi G, Gokoffski KK. Electric field stimulation directs target-specific axon regeneration and partial restoration of vision after optic nerve crush injury. PLoS One. 20(1):e0315562. (2025). PMID: 39787061
  • Kiraly JK, Balraj A, Leary P, You Z, Harris SC, Hyer JD, Dunn FA, Kolodkin AL. Slit2/Robo1 signaling constrains image stabilization responses to preserve ethologically favorable directional asymmetry. Curr Biol. 35(19):4714. (2025). PMID: 40945510
  • Kriukov E, Soucy JR, Labrecque E, Baranov P. Unraveling the developmental heterogeneity of human retinal ganglion cells within the developing retina to study the continuity of maturation. Dev Biol. 528:91. (2025). PMID: 40912448
  • Li L, Feng X, Fang F, … Zhou R, Hu Y. Actin depolymerization promotes axon regeneration by restoring axonal mitochondrial transport in mouse models of optic neuropathy. Sci Transl Med. 18(838):eadw0908. (2026). PMID: 41739905
  • Liu D, Webber HC, Bian F, … Braun M, Lansky Z. Optineurin-facilitated axonal mitochondria delivery promotes neuroprotection and axon regeneration. Nat Commun. 16(1):1789. (2025). PMID: 39979261
  • Mackin RD, Bhalla RV, Akhanov V, Abdulwahab QT, Burger CA, Samuel MA. Retinal ganglion cell migration and viability requires the kinase LKB1. J Cell Biol. 224(7):e202410023. (2025). PMID: 40498035
  • Masterson SP, Govindaiah G, Guido W, Bickford ME. Cell-type specific binocular interactions in mouse visual thalamus. iScience. 28(7):112956. (2025). PMID: 40687842
  • McCool S, Jain A, Smith JC, Schaal V, Pendyala G, Yelamanchili S, Van Hook MJ. Compensatory responses to glaucoma pathology in the dorsolateral geniculate nucleus. iScience. 29(4):115176. (2026). PMID: 41858631
  • Murphy PJ, McGregor JE, Xu Z, Yang Q, Merigan W, Williams DR. Optogenetic stimulation of single ganglion cells in the living primate fovea. Elife. 12:RP90050. (2025). PMID: 41041715
  • O’Neill N, Sarabu N, Abd Alhadi S, Pham JC, Dine K, Chaqour B, Ross AG, Shindler KS. The role of Nrf2 in SIRT1-mediated RGC neuroprotection in traumatic optic neuropathy. Transl Vis Sci Technol. 15(7):2. (2026). PMID: 42390160
  • Ou Y, Chopra V, Rosdahl JA, … Kim SJ, WuDunn D. Visual electrophysiology for the diagnosis of glaucoma: a report by the American Academy of Ophthalmology. Ophthalmology. 133(8):1036. (2026). PMID: 42287272
  • Pahlavan P, Mayer PS, Simonyan A, … Gokoffski KK, Lazzi G. Toward non-invasive electrical stimulation for guided optic nerve regeneration. IEEE Trans Neural Syst Rehabil Eng. 33:3616. (2025). PMID: 40875415
  • Pavlou M, Probst M, Kaplan L, Filippova E, Prieve AR, Rieke F, Reh TA. AAV-mediated expression of proneural factors stimulates neurogenesis from adult Müller glia in vivo. EMBO Mol Med. 17(4):722. (2025). PMID: 40050705
  • Pereiro X, Ruzafa N, Azkargorta M, … Santiago AR, Vecino E. Müller glial cells located in the peripheral retina are more susceptible to high pressure: implications for glaucoma. Cell Biosci. 14(1):5. (2024). PMID: 38183095
  • Saludares M, Wennberg-Smith Z, Beykin G, Khatib TZ, Nunez M, Wang Q, Di Polo A, Goldberg JL. A phase I randomized trial of topical insulin for glaucoma: safety and efficacy outcomes. Ophthalmol Sci. 6(2):101032. (2025). PMID: 41584099
  • Santamaría-Muñoz D, Reyes RV, Krueger MR, … Simó S, La Torre A. Cell-intrinsic vulnerability and immune activation cooperate to drive degeneration in a mitochondrial complex I deficiency model of optic neuropathy. J Neuroinflammation. 23(1):83. (2026). PMID: 41645183
  • Shil SK, Subramani M, Van Hook MJ, Qiu F, Ahmad I. Disease modeling of myocilin mutation-dependent normal tension glaucoma: human retinal ganglion cell susceptibility to unfolded protein response and mTOR signaling. Stem Cells. 44(3):sxag009. (2026). PMID: 41712748
  • Soucy JR, Oswald J, Kriukov E, Phay M, Masland J, Pernstich C, Baranov P. Sustained neurotrophic factor co-treatment enhances donor and host retinal ganglion cell survival in mice. Transl Vis Sci Technol. 14(9):27. (2025). PMID: 40965436
  • Tribble JR, Hui F, Quintero H, El Hajji S, Bell K, Di Polo A, Williams PA. Neuroprotection in glaucoma: mechanisms beyond intraocular pressure lowering. Mol Aspects Med. 92:101193. (2023). PMID: 37331129
  • Tribble JR, Joe M, Varricchio C, … Brancale A, Williams PA. NMNAT2 is a druggable target to drive neuronal NAD production. Nat Commun. 15(1):6256. (2024). PMID: 39048544
  • Tribble JR, Wong VHY, Stuart KV, … Bui BV, Williams PA. Dysfunctional one-carbon metabolism identifies vitamins B6, B9, B12, and choline as neuroprotective in glaucoma. Cell Rep Med. 6(5):102127. (2025). PMID: 40345183
  • Whitley JB, Masterson SP, Gordon T, … Guido W, Bickford ME. GABAergic projections from the pretectum boost retinogeniculate signal transfer via disinhibition. J Neurosci. 45(22):e2325242025. (2025). PMID: 40228897
  • Wohlschlegel J, Kierney F, Arakelian KL, … Moshiri A, Reh TA. Stimulating the regenerative capacity of the human retina with proneural transcription factors in 3D cultures. Proc Natl Acad Sci U S A. 122(3):e2417228122. (2025). PMID: 39823300
  • Yu A, Fang C, Tan LX, Lakkaraju A, Della Santina L, Ou Y. Microglia target synaptic sites early during excitatory circuit disassembly in neurodegeneration. iScience. 28(4):112201. (2025). PMID: 40212592
  • Zhang KY, Nagalingam A, Mary S, … Zack DJ, Johnson TV. Rare intercellular material transfer as a confound to interpreting inner retinal neuronal transplantation following internal limiting membrane disruption. Stem Cell Reports. 18(11):2203. (2023). PMID: 37802075

Recent papers nominated by RReSTORe Members as having especially high impact on the field of vision restoration in optic neuropathy:

  • Guttenplan KA, Weigel MK, Prakash P, … Liddelow SA, Barres BA. Neurotoxic reactive astrocytes induce cell death via saturated lipids. Nature. 599(7883):102. (2021). PMID: 34616039
  • Tian F, Cheng Y, Zhou S, … Geschwind DH, He Z. Core transcription programs controlling injury-induced neurodegeneration of retinal ganglion cells. Neuron. 110(16):2607. (2022). PMID: 35767995
  • Welsbie DS, Mitchell KL, Jaskula-Ranga V, … Berlinicke CA, Zack DJ. Enhanced functional genomic screening identifies novel mediators of dual leucine zipper kinase-dependent injury signaling in neurons. Neuron. 94(6):1142. (2017). PMID: 28641113
  • Zhao M, Toma K, Kinde B, … Han Y, Duan X. Osteopontin drives retinal ganglion cell resiliency in glaucomatous optic neuropathy. Cell Rep. 42(9):113038. (2023). PMID: 37624696
  • Jacobi A, Tran NM, Yan W, … He Z, Sanes JR. Overlapping transcriptional programs promote survival and axonal regeneration of injured retinal ganglion cells. Neuron. 110(16):2625. (2022). PMID: 35767994
  • Petrova V, Pearson CS, Ching J, … Fawcett JW, Eva R. Protrudin functions from the endoplasmic reticulum to support axon regeneration in the adult CNS. Nat Commun. 11(1):5614. (2020). PMID: 33154382
  • Cheng Y, Yin Y, Zhang A, … Geschwind DH, Benowitz LI. Transcription factor network analysis identifies REST/NRSF as an intrinsic regulator of CNS regeneration in mice. Nat Commun. 13(1):4418. (2022). PMID: 35906210
  • Passino R, Finneran MC, Hafner H, … Benowitz LI, Giger RJ. Neutrophil-inflicted vasculature damage suppresses immune-mediated optic nerve regeneration. Cell Rep. 43(3):113931. (2024). PMID: 38492223
  • Varadarajan SG, Wang F, Dhande OS, Le P, Duan X, Huberman AD. Postsynaptic neuronal activity promotes regeneration of retinal axons. Cell Rep. 42(5):112476. (2023). PMID: 37141093
  • Li L, Fang F, Feng X, … Sun Y, Hu Y. Single-cell transcriptome analysis of regenerating RGCs reveals potent glaucoma neural repair genes. Neuron. 110(16):2646. (2022). PMID: 35952672
  • Soucy JR, Todd L, Kriukov E, … Reh TA, Baranov P. Controlling donor and newborn neuron migration and maturation in the eye through microenvironment engineering. Proc Natl Acad Sci U S A. 120(46):e2302089120. (2023). PMID: 37931105
  • Rheaume BA, Jereen A, Bolisetty M, … Robson P, Trakhtenberg EF. Single cell transcriptome profiling of retinal ganglion cells identifies cellular subtypes. Nat Commun. 9(1):2759. (2018). PMID: 30018341
  • Agarwal D, Dash N, Mazo KW, … Welsbie DS, Wahlin KJ. Human retinal ganglion cell neurons generated by synchronous BMP inhibition and transcription factor mediated reprogramming. NPJ Regen Med. 8(1):55. (2023). PMID: 37773257
  • Subramani M, Van Hook MJ, Ahmad I. Reproducible generation of human retinal ganglion cells from banked retinal progenitor cells: analysis of target recognition and IGF-1-mediated axon regeneration. Front Cell Dev Biol. 11:1214104. (2023). PMID: 37519299
  • Cameron EG, Nahmou M, Toth AB, … Kapiloff MS, Goldberg JL. A molecular switch for neuroprotective astrocyte reactivity. Nature. 626(8000):574. (2024). PMID: 38086421
  • Ludwig AL, Mayerl SJ, Gao Y, … Zhao X, Gamm DM. Re-formation of synaptic connectivity in dissociated human stem cell-derived retinal organoid cultures. Proc Natl Acad Sci U S A. 120(2):e2213418120. (2023). PMID: 36598946
  • Xing J, Theune WC, Lukomska A, Frost MP, Damania A, Trakhtenberg EF. Experimental upregulation of developmentally downregulated ribosomal protein large subunits 7 and 7A promotes axon regeneration after injury in vivo. Exp Neurol. 368:114510. (2023). PMID: 37633482

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