A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing JD Finn, AR Smith, MC Patel, L Shaw, MR Youniss, J van Heteren, ... Cell reports 22 (9), 2227-2235, 2018 | 668 | 2018 |
Blood-based identification of non-responders to anti-TNF therapy in rheumatoid arthritis TM Thomson, RM Lescarbeau, DA Drubin, D Laifenfeld, D de Graaf, ... BMC medical genomics 8, 1-12, 2015 | 39 | 2015 |
In vitro model of metastasis to bone marrow mediates prostate cancer castration resistant growth through paracrine and extracellular matrix factors RM Lescarbeau, FP Seib, M Prewitz, C Werner, DL Kaplan Public Library of Science 7 (8), e40372, 2012 | 32 | 2012 |
Response to “Unexpected mutations after CRISPR–Cas9 editing in vivo” RM Lescarbeau, B Murray, TM Barnes, N Bermingham Nature methods 15 (4), 237-237, 2018 | 25 | 2018 |
A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent JD Finn, AR Smith, MC Patel, L Shaw, MR Youniss, J Van Heteren, ... in vivo genome editing. Cell Rep 22, 2227-2235, 2018 | 15 | 2018 |
A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing, Cell Rep. 22 (2018) 2227–2235 JD Finn, AR Smith, MC Patel, L Shaw, MR Youniss, J van Heteren, ... | 13 | |
Quantitative analysis of castration resistant prostate cancer progression through phosphoproteome signaling RM Lescarbeau, DL Kaplan BMC cancer 14, 1-12, 2014 | 12 | 2014 |
Compositions and methods for the treatment of hemoglobinopathies AE Boitano, M Cooke, LB Klickstein, R Lescarbeau, CS Mickanin, ... | 11 | 2022 |
Compositions and methods for immunooncology MW Chen, D Melissa, G Dranoff, CS Mackanin, R Lescarbeau, ... US Patent App. 15/780,751, 2018 | 9 | 2018 |
CRISPR/Cas9 gene-edited hematopoietic stem cell therapy for sickle cell disease WC Vionnie, Y Liu, M Curran, P Zhang, J Snead, C Schmedt, Y Yang, ... Blood 130, 535, 2017 | 8 | 2017 |
A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep 2018; 22: 2227-35 JD Finn, AR Smith, MC Patel, L Shaw, MR Youniss, J van Heteren, ... | 6 | |
A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing. Cell Rep 22 (9): 2227–2235 JD Finn, AR Smith, MC Patel, L Shaw, MR Youniss, J van Heteren, ... | 5 | 2018 |
Development of NTLA-2001, a CRISPR/Cas9 genome editing therapeutic for the treatment of ATTR. K Wood, M Pink, J Seitzer, N Gardner, S Alexander, T DiMezzo, A Amaral, ... Liver 17 (19), 24, 2019 | 3 | 2019 |
A Novel Strategy for Off-the-Shelf T Cell Therapy Which Evades Allogeneic T Cell and NK Cell Rejection Y Zhang, S Goel, A Prodeus, U Jetley, Y Tan, J Averill, U Ranade, ... Blood 138, 1711, 2021 | 2 | 2021 |
Validation of CRISPR/Cas9 off-target discovery profiles from in silico prediction, cell-based and biochemical-based assays with targeted off-target sequencing N Patel, B Han, B Murray, J Growe, P Salerno, R Lescarbeau, J Seitzer, ... Mol Ther 28, 99, 2020 | 2 | 2020 |
Rescue of Amyloid Deposition Phenotype after Single-Treatment CRISPR/Cas9 Gene Editing in a Humanized Mouse Model of TTR Amyloidosis MJ Saraiva, S Costelha, P Goncalves, A Teixeira, H Martins, T Dirstine, ... MOLECULAR THERAPY 26 (5), 130-130, 2018 | 2 | 2018 |
A reanalysis of Schaefer et al. does not indicate extensive CRISPR/Cas9 mediated off-target editing events RM Lescarbeau, B Murray, TM Barnes, N Bermingham bioRxiv, 159608, 2017 | 2 | 2017 |
Compositions and methods for treating alpha-1 antitrypsin deficiency S Odate, W Strapps, RM Lescarbeau US Patent 11,549,107, 2023 | 1 | 2023 |
Compositions and Methods for Hydroxyacid Oxidase 1 (HAO1) Gene Editing for Treating Primary Hyperoxaluria Type 1 (PH1) ZW Dymek, S Odate, BA Murray, RM Lescarbeau, A Huebner, W Strapps, ... US Patent App. 17/162,377, 2021 | 1 | 2021 |
Compositions and Methods for TTR Gene Editing and Treating ATTR Amyloidosis AMP Kanjolia, S Odate, JL Seitzer, RM Lescarbeau, W Strapps US Patent App. 16/828,573, 2020 | 1 | 2020 |