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Leah Byrne

  • Assistant Professor, Department of Ophthalmology

Current Research Interests

Gene therapy, viral vector engineering, genome editing, inherited retinal degeneration, genetics of eye disease

Selected Research Publications

 
He J, Kleyman M, Chen J, Alikaya A, Rothenhoefer KM, Ozturk BE, Wirthlin M, Bostan AC, Fish K, Byrne LC, Pfenning AR, Stauffer WR. Transcriptional and anatomical diversity of medium spiny neurons in the primate striatum. Curr Biol. 2021 Dec 20;31(24):5473-5486.e6. doi: 10.1016/j.cub.2021.10.015. Epub 2021 Nov 1. PubMed PMID: 34727523; PubMed Central PMCID: PMC9359438.
 
 
Öztürk BE, Johnson ME, Kleyman M, Turunç S, He J, Jabalameli S, Xi Z, Visel M, Dufour VL, Iwabe S, Pompeo Marinho LFL, Aguirre GD, Sahel JA, Schaffer DV, Pfenning AR, Flannery JG, Beltran WA, Stauffer WR, Byrne LC. scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution. Elife. 2021 Oct 19;10. doi: 10.7554/eLife.64175. PubMed PMID: 34664552; PubMed Central PMCID: PMC8612735.
 
 
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. 2021 Oct 4;10. doi: 10.7554/eLife.69479. PubMed PMID: 34605404; PubMed Central PMCID: PMC8577842.
 
 
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. PubMed PMID: 34196317.
 
 
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 J. 2020 Aug;34(8):10146-10167. doi: 10.1096/fj.202000282R. Epub 2020 Jun 14. PubMed PMID: 32536017; PubMed Central PMCID: PMC7688577.
 
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. 2020 May 21;5(10). doi: 10.1172/jci.insight.135112. PubMed PMID: 32271719; PubMed Central PMCID: PMC7259523.
 
 
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. Sci Adv. 2017 Dec;3(12):eaar3952. doi: 10.1126/sciadv.aar3952. eCollection 2017 Dec. PubMed PMID: 29279867; PubMed Central PMCID: PMC5738228.
 
 
Byrne LC. Anti-VEGF AAV2 injections: The fewer the better. Sci Transl Med. 2017 Jun 7;9(393). doi: 10.1126/scitranslmed.aan4921. PubMed PMID: 28592565.
 
 
Byrne LC. What's old is new again: Autologous stem cell transplant for AMD. Sci Transl Med. 2017 Apr 26;9(387). doi: 10.1126/scitranslmed.aan2783. PubMed PMID: 28446683.
 
 
Byrne L. Rounding up sickle cells with gene therapy. Sci Transl Med. 2017 Mar 15;9(381). doi: 10.1126/scitranslmed.aam9864. PubMed PMID: 28298423.
 
 
Shen SQ, Myers CA, Hughes AE, Byrne LC, Flannery JG, Corbo JC. Massively parallel cis-regulatory analysis in the mammalian central nervous system. Genome Res. 2016 Feb;26(2):238-55. doi: 10.1101/gr.193789.115. Epub 2015 Nov 17. PubMed PMID: 26576614; PubMed Central PMCID: PMC4728376.