Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque KT McCullough, SL Boye, D Fajardo, K Calabro, JJ Peterson, CE Strang, ... Human gene therapy 30 (5), 571-589, 2019 | 85 | 2019 |
Impact of heparan sulfate binding on transduction of retina by recombinant adeno-associated virus vectors SL Boye, A Bennett, ML Scalabrino, KT McCullough, K Van Vliet, ... Journal of virology 90 (8), 4215-4231, 2016 | 80 | 2016 |
Gene Therapy Fully Restores Vision to the All-Cone Nrl−/−Gucy2e−/− Mouse Model of Leber Congenital Amaurosis-1 SL Boye, JJ Peterson, S Choudhury, SH Min, Q Ruan, KT McCullough, ... Human Gene Therapy 26 (9), 575-592, 2015 | 38 | 2015 |
Postchallenge administration of brincidofovir protects healthy and immune-deficient mice reconstituted with limited numbers of T cells from lethal challenge with IHD-J-Luc … M Zaitseva, KT McCullough, S Cruz, A Thomas, CG Diaz, L Keilholz, ... Journal of Virology 89 (6), 3295-3307, 2015 | 14 | 2015 |
Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis SL Boye, C O’Riordan, J Morris, M Lukason, D Compton, R Baek, ... Molecular Therapy Methods & Clinical Development 28, 129-145, 2023 | 3 | 2023 |
Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion PC Kellish, D Marsic, SM Crosson, S Choudhury, ML Scalabrino, ... Molecular Therapy 31 (12), 3441-3456, 2023 | 1 | 2023 |
Investigating Dual AAV-Based Treatments for MYO7A Usher Syndrome in Myo7a-/-Mice and Macaque KR Calabro, S Boye, S Choudhury, KT McCullough, D Fajardo, CE Strang, ... MOLECULAR THERAPY 27 (4), 316-317, 2019 | 1 | 2019 |
AAV5 made by rHSV complementation displays increased retinal transduction relative to AAV5 made by plasmid transfection. KT Mccullough, L Adamson-Small, J Peterson, S Boye, N Clément, ... Investigative Ophthalmology & Visual Science 58 (8), 4084-4084, 2017 | 1 | 2017 |
Efficient In Vivo Gene Editing of Inherited Retinal Disease Genes in Mice and Non-Human Primates S Boye, M Stefanidakis, R Mepani, MN Skor, SW Gloskowski, J Horng, ... MOLECULAR THERAPY 25 (5), 353-354, 2017 | 1 | 2017 |
Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas SL Boye, EV Olshevskaya, IV Peshenko, KT McCullough, SE Boye, ... Molecular Vision 22, 1342, 2016 | 1 | 2016 |
Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6 RW Mellen, KR Calabro, KT McCullough, SM Crosson, A de la Cova, ... Molecular Therapy-Methods & Clinical Development 30, 48-64, 2023 | | 2023 |
AAV-CRISPR/Cas9 Gene Editing is Therapeutic in a Novel, Humanized Mouse Model of GUCY2D-Associated Cone Rod Dystrophy (CORD6) RW Mellen, KT McCullough, D Fajardo, K Calabro, S Crosson, E Xu, ... MOLECULAR THERAPY 30 (4), 566-566, 2022 | | 2022 |
CRISPR Genome Editing in Microphysiological Human Tissue System Models of Duchenne Muscular Dystrophy MJ Sitton, A Khodabukus, KT McCullough, N Bursac, CA Gersbach MOLECULAR THERAPY 30 (4), 500-500, 2022 | | 2022 |
A Novel GUCY2D (R838S) Knock-In Mouse Model of Autosomal Dominant Cone Rod Dystrophy (CORD6) Displays Progressive Photoreceptor Degeneration/Dysfunction RW Mellen, SL Boye, SM Crosson, KR Calabro, DS Fajardo, ... MOLECULAR THERAPY 29 (4), 261-261, 2021 | | 2021 |
Development of a Novel Cone-Rod Dystrophy 6 (CORD6) Mouse Model to Assess AAV-CRISPR/Cas9-Based Therapies RW Mellen, SL Boye, KT McCullough, D Fajardo, S Crosson, K Calabro, ... MOLECULAR THERAPY 28 (4), 267-267, 2020 | | 2020 |
An AAV-CRISPR/Cas9 Gene Editing Approach for GUCY2D-Associated Cone Rod Dystrophy (CORD6) RW Mellen, KT McCullough, SL Boye, D Fajardo, CE Strang, ... MOLECULAR THERAPY 27 (4), 429-429, 2019 | | 2019 |
AAV 44.9-a novel capsid that efficiently transduces photoreceptors and retinal pigment epithelium S Choudhury, R Mellen, K Mccullough, J Peterson, D Fajardo, J Chiorini, ... Investigative Ophthalmology & Visual Science 59 (9), 4536-4536, 2018 | | 2018 |
Investigating tolerability of subretinally delivered dual AAV-MYO7A vectors in non-human primate (NHP) K Calabro, SL Boye, K Mccullough, D Fajardo, WC Smith, CE Strang, ... Investigative Ophthalmology & Visual Science 59 (9), 4535-4535, 2018 | | 2018 |
An AAV-CRISPR/Cas9 gene editing approach for GUCY2D-associated cone rod dystrophy (CORD6) K Mccullough, SL Boye, D Fajardo, CE Strang, DC Witherspoon, ... Investigative Ophthalmology & Visual Science 59 (9), 6020-6020, 2018 | | 2018 |
Investigating the utility of mice as a model for developing clinically relevant dual AAV vectors to treat USH1B K Calabro, S Boye, KT Mccullough, S Choudhury, PD Gamlin, SE Boye Investigative Ophthalmology & Visual Science 58 (8), 4098-4098, 2017 | | 2017 |