In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery R Bahal, N Ali McNeer, E Quijano, Y Liu, P Sulkowski, A Turchick, YC Lu, ... Nature communications 7 (1), 13304, 2016 | 159 | 2016 |
Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium NA McNeer, K Anandalingam, RJ Fields, C Caputo, S Kopic, A Gupta, ... Nature communications 6 (1), 6952, 2015 | 152 | 2015 |
Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors NA McNeer, JY Chin, EB Schleifman, RJ Fields, PM Glazer, WM Saltzman Molecular Therapy 19 (1), 172-180, 2011 | 108 | 2011 |
Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo NA McNeer, EB Schleifman, A Cuthbert, M Brehm, A Jackson, C Cheng, ... Gene therapy 20 (6), 658-669, 2013 | 93 | 2013 |
Genetic mechanisms of primary chemotherapy resistance in pediatric acute myeloid leukemia NA McNeer, J Philip, H Geiger, RE Ries, VP Lavallee, M Walsh, M Shah, ... Leukemia 33 (8), 1934-1943, 2019 | 88 | 2019 |
Site-specific genome editing in PBMCs with PLGA nanoparticle-delivered PNAs confers HIV-1 resistance in humanized mice EB Schleifman, NA McNeer, A Jackson, J Yamtich, MA Brehm, LD Shultz, ... Molecular Therapy-Nucleic Acids 2, 2013 | 44 | 2013 |
Modified poly (lactic-co-glycolic acid) nanoparticles for enhanced cellular uptake and gene editing in the lung RJ Fields, E Quijano, NA McNeer, C Caputo, R Bahal, K Anandalingam, ... Advanced healthcare materials 4 (3), 361, 2015 | 39 | 2015 |
Forward RNAi screens in primary human hematopoietic stem/progenitor cells N Ali, C Karlsson, M Aspling, G Hu, N Hacohen, DT Scadden, J Larsson Blood, The Journal of the American Society of Hematology 113 (16), 3690-3695, 2009 | 39 | 2009 |
Nanoparticle for delivery of antisense γPNA oligomers targeting CCR5 R Bahal, NA McNeer, DH Ly, WM Saltzman, PM Glazer Artificial DNA: PNA & XNA 4 (2), 49-57, 2013 | 38 | 2013 |
Targeted genome modification via triple helix formation AS Ricciardi, NA McNeer, KK Anandalingam, WM Saltzman, PM Glazer Cancer Genomics and Proteomics: Methods and Protocols, 89-106, 2014 | 30 | 2014 |
Polymer delivery systems for site-specific genome editing NA McNeer, EB Schleifman, PM Glazer, WM Saltzman Journal of controlled release 155 (2), 312-316, 2011 | 16 | 2011 |
Compositions for enhancing targeted gene editing and methods of use thereof WM Saltzman, P Glazer, R Bahal, NA McNeer, E Quijano US Patent 11,136,597, 2021 | 14 | 2021 |
Polymeric materials loaded with mutagenic and recombinagenic nucleic acids WM Saltzman, PM Glazer, J Chin, N McNeer US Patent App. 12/938,177, 2011 | 12 | 2011 |
Correction of F508del CFTR in airway epithelium using nanoparticles delivering triplex-forming PNAs NA McNeer, K Anandalingam, RJ Fields, C Caputo, S Kopic, A Gupta, ... Nature communications 6, 6952, 2015 | 7 | 2015 |
Revumenib monotherapy in patients with relapsed/refractory kmt2ar acute leukemia: Topline efficacy and safety results from the pivotal augment-101 Phase 2 study I Aldoss, GC Issa, M Thirman, J DiPersio, M Arellano, JS Blachly, ... Blood 142, LBA-5, 2023 | 6 | 2023 |
Compositions and methods for treatment of cystic fibrosis PM Glazer, WM Saltzman, M Egan, NA McNeer US Patent App. 15/998,613, 2020 | 6 | 2020 |
NIMBLE: a phase I/II study of AZD0466 monotherapy or in combination in patients with advanced hematological malignancies M Konopleva, N Jain, CL Andersen, NC Francisco, N Elgeioushi, ... Blood 138, 2353, 2021 | 5 | 2021 |
Revumenib monotherapy in patients with relapsed/refractory KMT2Ar acute leukemias: efficacy and safety results from the Augment-101 phase 1/2 study I Aldoss, GC Issa, MJ Thirman, J DiPersio, M Arellano, JS Blachly, ... Blood 142, 2907, 2023 | 3 | 2023 |
Outcomes after transplant in relapsed/refractory KMT2Ar (MLLr) and mNPM1 (NPM1c) leukemia patients achieving remissions after menin inhibition: SNDX-5613 (revumenib) Ph1 experience GC Issa, B Cuglievan, E Stein, ML Arellano, A Žucenka, N Khera, ... Blood 140 (Supplement 1), 914-916, 2022 | 3 | 2022 |
Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium. Nat Commun. 2015; 6: 6952 NA McNeer, K Anandalingam, RJ Fields, C Caputo, S Kopic, A Gupta | 3 | |