Publications
Levochkina M, Beryozkin A, Aweidah H, Beale O and Byrne L (2023) Can Viruses Be Used to Make People Healthy?. Front. Young Minds. 11:1021056. doi: 10.3389/frym.2023.1021056
Hamzah Aweidah, Zhouhuan Xi, José-Alain Sahel, Leah C. Byrne. PRPF31-retinitis pigmentosa: Challenges and opportunities for clinical translation. Vision Research. Volume 213, 2023,108315, ISSN 0042-6989,https://doi.org/10.1016/j.visres.2023.108315.
Lohss, Maxwell Benjamin, Ho, Jonathan, Naylor, Nathan, Cashman, Stacy, Fu, Roxana, Stefko, S. Tonya, Byrne, Leah C. Adaptable Three-Pin Skull Clamp for Large Animal Research. HardwareX. 2023, e00472, ISSN 2468-0672. https://doi.org/10.1016/j.ohx.2023.e00472.
Emilia A Zin, Bilge E Ozturk, Deniz Dalkara, Leah C Byrne. Developing New Vectors for Retinal Gene Therapy. Cold Spring Harb Perspect Med. 2023 Mar 27;a041291. doi: 10.1101/cshperspect.a041291. PMID: 36987583.
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. Nat Commun. 2022 Dec 13;13(1):7695. doi: 10.1038/s41467-022-35361-8.PMID: 36509783.
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 Regen Med. 2022 Aug 16;7(1):39. doi: 10.1038/s41536-022-00235-6. PubMed PMID: 35974011.
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, 2022. https://doi.org/10.1016/j.omtm.2022.04.014.
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 2022;11:e69571 DOI: 10.7554/eLife.69571.
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. Proc Natl Acad Sci U S A. 2022 Mar 29;119(13):e2117038119. doi: 10.1073/pnas.2117038119.
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. 2021;10:e64175 DOI: 10.7554/eLife.64175
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. Available online 1 November 2021. https://doi.org/10.1016/j.cub.2021.10.015.
Gemayel M, Byrne LC, Ciulla T, Sahel JA. Delivery of Genetic Information: Viral Vector and Nonviral Vector Gene Therapies. Int Ophthalmol Clin. 2021 Jul 1;61(3):35-57. doi: 10.1097/IIO.0000000000000360.
Byrne LC, Day TP, Visel M, Fortuny C, Dalkara D, Merigan WH, Schaffer DV, Flannery JG. In vivo directed evolution of AAV in the primate retina. JCI Insight. 2020 Apr 9;. doi: 10.1172/jci.insight.135112. https://insight.jci.org/articles/view/135112.
Day TP, Byrne LC, Flannery JG, Schaffer DV. Screening for Neutralizing Antibodies Against Natural and Engineered AAV Capsids in Nonhuman Primate Retinas. Methods Mol Biol. 2018;1715:239-249.
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. 2015 May 7;161(4):817-32.
Byrne LC, Lee T, Flannery JG, Dalkara D. The expression pattern of systemically injected AAV9 in the developing mouse retina is determined by age. Mol Ther. 2015 Feb;23(2):290-6.
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. J Clin Invest. 2015 Jan;1 25(1):105-16.
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 Ther. 2014 Jun;21(6):585-92.
Day TP/Byrne LC, Schaffer DV, Flannery JG. Advances in AAV Vector Development for Gene Therapy in the Retina. Adv Exp Med Biol. 2014;801:687-93.
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. Sci Transl Med. 2013 Jun 12;5(189):189ra76.
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