Gene augmentation prevents retinal degeneration in a CRISPR/Cas9-based mouse model of PRPF31 retinitis pigmentosa nork Zhouhuan Xi, Abhishek Vats, José-Alain Sahel, Yuanyuan Chen, Leah C. Byrne

Testu osoa

Adaptable three-pin skull clamp for large animal research nork Maxwell Lohss, Jonathan Ho, Nathan Naylor, Stacy Cashman, Roxana Fu, S. Tonya Stefko, Leah C. Byrne

Testu osoa

Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants nork Zhouhuan Xi, Bilge E. Öztürk, Molly E. Johnson, Serhan Turunç, William R. Stauffer, Leah C. Byrne

Testu osoa

AAV-mediated, optogenetic ablation of Müller Glia leads to structural and functional changes in the mouse retina. nork Leah C Byrne, Fakhra Khalid, Trevor Lee, Emilia A Zin, Kenneth P Greenberg, Meike Visel, David V Schaffer, John G Flannery

Testu osoa

Modeling PRPF31 retinitis pigmentosa using retinal pigment epithelium and organoids combined with gene augmentation rescue nork Amélie Rodrigues, Amélie Slembrouck-Brec, Céline Nanteau, Angélique Terray, Yelyzaveta Tymoshenko, Yvrick Zagar, Sacha Reichman, Zhouhuan Xi, José-Alain Sahel, Stéphane Fouquet, Gael Orieux, Emeline F. Nandrot, Leah C. Byrne, Isabelle Audo, Jérôme E. Roger, Olivier Goureau

Testu osoa

Machine learning sequence prioritization for cell type-specific enhancer design nork 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

Testu osoa

Parallel functional testing identifies enhancers active in early postnatal mouse brain nork Jason T Lambert, Linda Su-Feher, Karol Cichewicz, Tracy L Warren, Iva Zdilar, Yurong Wang, Kenneth J Lim, Jessica L Haigh, Sarah J Morse, Cesar P Canales, Tyler W Stradleigh, Erika Castillo Palacios, Viktoria Haghani, Spencer D Moss, Hannah Parolini, Diana Quintero, Diwash Shrestha, Daniel Vogt, Leah C Byrne, Alex S Nord

Testu osoa

scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution nork Bilge E Öztürk, Molly E Johnson, Michael Kleyman, Serhan Turunç, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valérie L Dufour, Simone Iwabe, Luis Felipe L Pompeo Marinho, Gustavo D Aguirre, José-Alain Sahel, David V Schaffer, Andreas R Pfenning, John G Flannery, William A Beltran, William R Stauffer, Leah C Byrne

Testu osoa