Publications

1. 2026
   Lim J, Larimer-Picciani AM, Moshiri A, Wang JK, Takahashi N, Raposo ACS, Motta MJ, Byrne LC, Thomasy SM Ocular safety and efficacy of AAV-mediated tyrosinase gene augmentation in a nonhuman primate model bioRxiv 2026.07.13.738268 · Preprint

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2. 2026
   Johnson ME, Ozturk BE, Tugwell TH, Lambros M, Janowitz HN, Flohr K, Sedorovitz M, Campello L, Hartung JE, Hogle B, Gillespie M, Schriever H, Aweidah H, Koester J, Clausen I, Seeber S, Revelant F, Schreurs R, Koechl F, Sieving PA, Sahel JA, Stauffer WR, Peixoto RT, Fauser S, Lin R, Conway JF, da Silva S, Krol J, Betegon M, Byrne LC Structural dynamics insights into principles underlying the fitness of new broadly potent AAVs bioRxiv 2026.03.24.713814 · Preprint

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3. 2026
   Kriebel WG, Larimer-Picciani AM, Nukala M, Sahel JA, Byrne LC Quantifying functional vision in a mouse model of oculocutaneous albinism type 1 Scientific Reports 16(1):14563

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4. 2026
   Westbay JH, Bigley DP, Sappa S, Ayalon A, Aweidah H, Dignam LD, Martel JN, Beltran WA, Sahel JA, Byrne LC, DiLeo MV Synthesis and characterization of an injectable telechelic material for the epiretinal delivery of retinal gene therapies Journal of Materials Chemistry B 14(12):3703-3718

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5. 2026
   Lohss M, Samski KS, Liasis A, Aweidah H, Komatsu C, Beale O, Lee DMW, Rossi EA, Shroff SG, Sahel JA, Stefko ST, Byrne LC A mobile system for whole eye perfusion supporting retinal function and surgery Frontiers in Bioengineering and Biotechnology 13:1699876

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6. 2025
   Sudharsan R, Murgiano L, Ahuja A, Sato Y, Kwok J, Dolgova N, Savina S, Sedorovitz M, Dufour VL, Aguirre GD, Byrne LC, Beltran WA Novel photoreceptor-specific promoters for gene therapy in mid- to late-stage retinal degeneration Molecular Therapy 33(9):4320-4337

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7. 2025
   He J, Phan BN, Kerkhoff WG, Alikaya A, Hong T, Brull OR, Fredericks JM, Sedorovitz M, Srinivasan C, Leone MJ, Wirfel OM, Brown A, Dauby S, Tittle RK, Lin MK, Hooks BM, Bostan AC, Gharbawie OA, Byrne LC, Pfenning AR, Stauffer WR Machine learning identification of enhancers in the rhesus macaque genome Neuron 113(10):1548-1561.e8

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8. 2025
   Sedorovitz M, Byrne LC, Betegon M Chromatographic purification and polishing of AAV particles Methods in Molecular Biology 2848:249-257

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9. 2025
   Vats A, Xi Z, Byrne LC, Chen Y Retinal explant culture from mouse, human, and nonhuman primates and its applications in vision research Methods in Molecular Biology 2848:169-186

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10. 2025
    Beryozkin A, Byrne LC In vivo imaging of rodent retina in retinal disease Methods in Molecular Biology 2848:151-167

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11. 2023
    Levochkina M, Beryozkin A, Aweidah H, Beale O, Byrne L Can Viruses Be Used to Make People Healthy? Frontiers for Young Minds 11:1021056

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12. 2023
    Hamzah Aweidah, Zhouhuan Xi, José-Alain Sahel, Leah C. Byrne PRPF31-retinitis pigmentosa: Challenges and opportunities for clinical translation Vision Research, Volume 213, 108315

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13. 2023
    Lohss MB, Ho J, Naylor N, Cashman S, Fu R, Stefko ST, Byrne LC Adaptable Three-Pin Skull Clamp for Large Animal Research HardwareX, e00472

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14. 2023
    Emilia A Zin, Bilge E Ozturk, Deniz Dalkara, Leah C Byrne Developing New Vectors for Retinal Gene Therapy Cold Spring Harbor Perspectives in Medicine, a041291

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15. 2022
    Xi Z, Vats A, Sahel JA, Chen Y, Byrne LC Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa Nature Communications 13(1):7695

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16. 2022
    Rodrigues A, Slembrouck-Brec A, Nanteau C, Terray A, Tymoshenko Y, Zagar Y, Reichman S, Xi Z, Sahel JA, Fouquet S, Orieux G, Nandrot EF, Byrne LC, Audo I, Roger JE, Goureau O Modeling PRPF31 retinitis pigmentosa using retinal pigment epithelium and organoids combined with gene augmentation rescue npj Regenerative Medicine 7(1):39

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17. 2022
    Zhouhuan Xi, Bilge E. Öztürk, Molly E. Johnson, Serhan Turunç, William R. Stauffer, Leah C. Byrne Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants Molecular Therapy — Methods & Clinical Development

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18. 2022
    Alyssa J Lawler, Easwaran Ramamurthy, Ashley R Brown, Naomi Shin, Yeonju Kim, Noelle Toong, Irene M Kaplow, Morgan Wirthlin, Xiaoyu Zhang, BaDoi N Phan, Grant A Fox, Kirsten Wade, Jing He, Bilge Esin Ozturk, Leah C Byrne, William R Stauffer, Kenneth N Fish, Andreas R Pfenning Machine learning sequence prioritization for cell type-specific enhancer design eLife 11:e69571

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19. 2022
    Miyadera K, Santana E, Roszak K, Iffrig S, Visel M, Iwabe S, Boyd RF, Bartoe JT, Sato Y, Gray A, Ripolles-Garcia A, Dufour VL, Byrne LC, Flannery JG, Beltran WA, Aguirre GD Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness PNAS 119(13):e2117038119

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20. 2021
    Bilge E. Ozturk, Molly E. Johnson, Michael Kleyman, Serhan Turunc, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valerie L. Dufour, Simone Iwabe, Felipe Pompeo Marinho, Gustavo D. Aguirre, Jose-Alain Sahel, David V. Schaffer, Andreas R. Pfenning, John G. Flannery, William A. Beltran, William R. Stauffer, Leah C. Byrne scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution eLife 10:e64175

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21. 2021
    Jing He, Michael Kleyman, Jianjiao Chen, Aydin Alikaya, Kathryn M. Rothenhoefer, Bilge Esin Ozturk, Morgan Wirthlin, Kenneth Fish, Leah C. Byrne, Andreas R. Pfenning, William R. Stauffer Transcriptional Diversity of Medium Spiny Neurons in the Primate Striatum Current Biology

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22. 2021
    Gemayel M, Byrne LC, Ciulla T, Sahel JA Delivery of Genetic Information: Viral Vector and Nonviral Vector Gene Therapies International Ophthalmology Clinics 61(3):35-57

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23. 2021
    Lambert JT, Su-Feher L, Cichewicz K, Warren TL, Zdilar I, Wang Y, Lim KJ, Haigh JL, Morse SJ, Canales CP, Stradleigh TW, Castillo Palacios E, Haghani V, Moss SD, Parolini H, Quintero D, Shrestha D, Vogt D, Byrne LC, Nord AS Parallel functional testing identifies enhancers active in early postnatal mouse brain eLife 10:e69479

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24. 2020
    Byrne LC, Day TP, Visel M, Strazzeri JA, Fortuny C, Dalkara D, Merigan WH, Schaffer DV, Flannery JG In vivo-directed evolution of adeno-associated virus in the primate retina JCI Insight 5(10):e135112

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25. 2020
    Liu X, Feng B, Vats A, Tang H, Seibel W, Swaroop M, Tawa G, Zheng W, Byrne L, Schurdak M, Chen Y Pharmacological clearance of misfolded rhodopsin for the treatment of RHO-associated retinitis pigmentosa FASEB Journal 34(8):10146-10167

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26. 2018
    Day TP, Byrne LC, Flannery JG, Schaffer DV Screening for Neutralizing Antibodies Against Natural and Engineered AAV Capsids in Nonhuman Primate Retinas Methods in Molecular Biology 1715:239-249

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27. 2017
    Gaj T, Ojala DS, Ekman FK, Byrne LC, Limsirichai P, Schaffer DV In vivo genome editing improves motor function and extends survival in a mouse model of ALS Science Advances 3(12):eaar3952

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28. 2017
    Byrne LC Anti-VEGF AAV2 injections: the fewer the better Science Translational Medicine 9(393):eaan4921 · Commentary

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29. 2017
    Byrne LC What's old is new again: autologous stem cell transplant for AMD Science Translational Medicine 9(387):eaan2783 · Commentary

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30. 2017
    Byrne L Rounding up sickle cells with gene therapy Science Translational Medicine 9(381):eaam9864 · Commentary

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31. 2016
    Shen SQ, Myers CA, Hughes AE, Byrne LC, Flannery JG, Corbo JC Massively parallel cis-regulatory analysis in the mammalian central nervous system Genome Research 26(2):238-55

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32. 2015
    Aït-Ali N, Fridlich R, Millet-Puel G, Clérin E, Delalande F, Jaillard C, Blond F, Perrocheau L, Reichman S, Byrne LC, Olivier-Bandini A, Bellalou J, Moyse E, Bouillaud F, Nicol X, Dalkara D, van Dorsselaer A, Sahel JA, Léveillard T Rod-derived cone viability factor promotes cone survival by stimulating aerobic glycolysis Cell 161(4):817-32

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33. 2015
    Byrne LC, Lin YJ, Lee T, Schaffer DV, Flannery JG The expression pattern of systemically injected AAV9 in the developing mouse retina is determined by age Molecular Therapy 23(2):290-6

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34. 2015
    Xue Y, Shen SQ, Jui J, Rupp AC, Byrne LC, Hattar S, Flannery JG, Corbo JC, Kefalov VJ CRALBP supports the mammalian retinal visual cycle and cone vision Journal of Clinical Investigation 125(2):727-38

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35. 2015
    Byrne LC, Dalkara D, Luna G, Fisher SK, Clérin E, Sahel JA, Léveillard T, Flannery JG Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration Journal of Clinical Investigation 125(1):105-16

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36. 2014
    Byrne LC, Oztürk BE, Lee T, Fortuny C, Visel M, Dalkara D, Schaffer DV, Flannery JG Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h-/- mouse Gene Therapy 21(6):585-92

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37. 2014
    Day TP, Byrne LC, Schaffer DV, Flannery JG Advances in AAV Vector Development for Gene Therapy in the Retina Advances in Experimental Medicine and Biology 801:687-93 · TP Day and LC Byrne contributed equally

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38. 2013
    Dalkara D, Byrne LC, Klimczak RR, Visel M, Yin L, Merigan WH, Flannery JG, Schaffer DV In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous Science Translational Medicine 5(189):189ra76 · D Dalkara and LC Byrne contributed equally

    PubMed
    
39. 2013
    Byrne LC, Khalid F, Lee T, Zin EA, Greenberg KP, Visel M, Schaffer DV, Flannery JG AAV-mediated, optogenetic ablation of Müller glia leads to structural and functional changes in the mouse retina PLoS One 8(9):e76075

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40. 2012
    Dalkara D, Byrne LC, Lee T, Hoffmann NV, Schaffer DV, Flannery JG Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9 Gene Therapy 19(2):176-81

    DOI PubMed

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