| CHANGE-seq reveals genetic and epigenetic effects on CRISPR–Cas9 genome-wide activity CR Lazzarotto, NL Malinin, Y Li, R Zhang, Y Yang, GH Lee, E Cowley, ... Nature biotechnology 38 (11), 1317-1327, 2020 | 181 | 2020 |
| Genome editing of HBG1 and HBG2 to induce fetal hemoglobin JY Métais, PA Doerfler, T Mayuranathan, DE Bauer, SC Fowler, MM Hsieh, ... Blood advances 3 (21), 3379-3392, 2019 | 141 | 2019 |
| Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase: RTB lectin fusion J Condori, W Acosta, J Ayala, V Katta, A Flory, R Martin, J Radin, ... Molecular genetics and metabolism 117 (2), 199-209, 2016 | 63 | 2016 |
| Human genetic diversity alters off-target outcomes of therapeutic gene editing S Cancellieri, J Zeng, LY Lin, M Tognon, MA Nguyen, J Lin, N Bombieri, ... Nature genetics 55 (1), 34-43, 2023 | 49 | 2023 |
| Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv 3: 3379–3392 JY Métais, PA Doerfler, T Mayuranathan, DE Bauer, SC Fowler, MM Hsieh, ... | 7 | 2019 |
| Lectin-mediated delivery of α-L-iduronidase: A novel approach for MPS I enzyme replacement therapy WL Acosta, L Ou, J Ayala, J Condori, V Katta, A Flory, R Martin, J Radin, ... Molecular Genetics and Metabolism 2 (117), S14-S15, 2016 | 6 | 2016 |
| Novel bioproduction and delivery strategies for MPS IIIA enzyme replacement therapeutics J Condori, V Katta, W Acosta, J Ayala, A Flory, J Radin, S Davis, ... Molecular Genetics and Metabolism 2 (117), S36, 2016 | 3 | 2016 |
| Combined+ 58 and+ 55 BCL11A enhancer editing Yields exceptional efficiency, specificity and HbF induction in human and NHP preclinical models J Zeng, S Demirci, MA Nguyen, LY Lin, SA Maitland, E Mintzer, Y Wu, ... Blood 138, 1852, 2021 | 2 | 2021 |
| Human genetic diversity modifies therapeutic gene editing off-target potential S Cancellieri, J Zeng, LY Lin, F Masillo, A Nguyen, N Bombieri, ... bioRxiv, 2021 | 2 | 2021 |
| Uptake, lysosomal activation, and disease correction in GM1 gangliosidosis cells by plant-made β-galactosidase: Lectin fusions J Ayala, W Acosta, J Condori, I Annunziata, V Katta, A Flory, R Martin, ... Molecular Genetics and Metabolism 2 (117), S23, 2016 | 2 | 2016 |
| CHANGE-seq-BE enables simultaneously sensitive and unbiased in vitro profiling of base editor genome-wide activity C Lazzarotto, V Katta, Y Li, E Urbina, GH Lee, SQ Tsai bioRxiv, 2024.03. 28.586621, 2024 | 1 | 2024 |
| Human genetic diversity alters therapeutic gene editing off-target outcomes S Cancellieri, J Zeng, LY Lin, M Tognon, MA Nguyen, J Lin, N Bombieri, ... bioRxiv, 2021.05. 20.445054, 2021 | 1 | 2021 |
| Receptor-independent mechanisms of RTB lectin-mediated ERT delivery provide unique advantages in enzyme uptake capacity, transcytosis, and lysosomal correction R Martin, W Acosta, J Ayala, V Katta, A Flory, J Radin, S Devaiah, ... Molecular Genetics and Metabolism 1 (120), S91, 2017 | 1 | 2017 |
| Selective haematological cancer eradication with preserved haematopoiesis S Garaudé, R Marone, R Lepore, A Devaux, A Beerlage, D Seyres, ... Nature, 1-8, 2024 | | 2024 |
| Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease V Katta, K O'Keefe, Y Li, T Mayuranathan, RM Levine, CR Lazzarotto, ... bioRxiv, 2024.04. 30.591737, 2024 | | 2024 |
| Virus-free CRISPR knock-in of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells K Shankar, I Zingler-Hoslet, L Shi, V Katta, BE Russell, SQ Tsai, ... bioRxiv, 2024.02. 14.580371, 2024 | | 2024 |
| Human genetic diversity alters therapeutic gene editing off-target outcomes [preprint] S Cancellieri, J Zeng, LY Lin, M Tognon, MA Nguyen, J Lin, N Bombieri, ... bioRxiv, 2022 | | 2022 |
| Genome Editing of Human Hematopoietic Stem Cells to Induce Fetal Hemoglobin for Autologous Cellular Therapy of Sickle Cell Disease V Katta, K O'Keefe, R Wood, CR Lazzarotto, T Mayuranathan, J Yen, ... MOLECULAR THERAPY 30 (5), 3-3, 2022 | | 2022 |
| Human Genetic Diversity Alters Therapeutic Gene Editing Off-Target Outcomes LY Lin, S Cancellieri, J Zeng, F Masillo, MA Nguyen, N Bombieri, ... Blood 138 (Supplement 1), 3993-3993, 2021 | | 2021 |
| CRISPR-Cas9 Genome Editing of Human CD34+ Cells at Gamma-globin Promoter to Induce Fetal Hemoglobin as Sickle Cell Disease Therapy V Katta, K O'Keefe, CR Lazzarotto, T Mayuranathan, J Yen, GH Lee, Y Li, ... MOLECULAR THERAPY 29 (4), 9-9, 2021 | | 2021 |
Shivakumar P. DevaiahVerified email at biostrategies-lc.com
