| Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency MS Packer, V Chowdhary, G Lung, LI Cheng, Y Aratyn-Schaus, ... Molecular Therapy 30 (4), 1396-1406, 2022 | 19 | 2022 |
| A novel base editing approach to directly edit the causative mutation in sickle cell disease SH Chu, MS Packer, JR Olins, G Lung, LI Cheng, A Liquori, C Lee, ... Mol. Ther 28 (808), 10.1016, 2020 | 3 | 2020 |
| Precise Correction of A1AT E342K by Modified NGA PAM Prime Editing and Determination of Prime Editing Inhibition by TREX2 G Lung Harvard University, 2021 | 2 | 2021 |
| Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsine Deficiency M Packer, V Chowdhary, G Lung, LI Cheng, Y Aratyn-Schaus, S Smith, ... MOLECULAR THERAPY 28 (4), 311-312, 2020 | 1 | 2020 |
| COMPOSITIONS AND METHODS FOR EDITING A TRANSTHYRETIN GENE G LUNG, LI CHENG, T BOHNUUD, CD SAGO US Patent WO2,024,112,775, 2024 | | 2024 |
| Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia J Chou, I Arnaoutova, Y Aratyn-Schaus, L Zhang, M Packer, HD Chen, ... | | 2024 |
| COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA T Bohnuud, G Lung WO Patent WO/2023/086,953, 2023 | | 2023 |
| Open-Source Four-Parameter Logistic Curve Fit For ELISA G Lung, ChatGPT https://github.com/Zhandragon/Open-Source-Four-Parameter-Logistic-Curve-Fit …, 2023 | | 2023 |
| Single, Systemic Administration of BEAM-301 Mitigates Fasting Hypoglycemia and Restores Metabolic Function in a Transgenic Mouse Model of Glycogen Storage Disease Type Ia Y Aratyn-Schaus, I Arnaoutova, D Leboeuf, TP Fernandez, T Leete, ... MOLECULAR THERAPY 30 (4), 42-43, 2022 | | 2022 |
| In vivo Base-Editing Corrects Metabolic Defects in Glycogen Storage Disease Type-Ia Y Aratyn-Schaus, I Arnaoutova, D LeBoeuf, TP Fernandez, M Packer, ... HUMAN GENE THERAPY 32 (19-20), A9-A9, 2021 | | 2021 |
| BASE-EDITING AS A THERAPEUTIC APPROACH FOR THE DIRECT CORRECTION OF DISEASE-CAUSING MUTATIONS UNDERLYING GLYCOGEN STORAGE DISEASE TYPE IA Y Aratyn-Schaus, TP Fernandez, MS Packer, LI Cheng, G Lung, SE Smith, ... The Liver Meeting Digital Experience™, 2020 | | 2020 |
| EVALUATION OF ADENINE BASE EDITING AS A POTENTIAL TREATMENT FOR ALPHA-1 ANTITRYPSIN DEFICIENCY MS Packer, V Chowdhary, G Lung, LI Cheng, Y Aratyn-Schaus, SE Smith, ... HEPATOLOGY 72, 25A-26A, 2020 | | 2020 |
| Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence N GAUDELLI, M Packer, I SLAYMAKER, Y Yu, B ZETSCHE, JM GEHRKE, ... | | 2020 |
| COMPOSITIONS AND METHODS FOR TREATING GLYCOGEN STORAGE DISEASE TYPE 1A N GAUDELLI, M PACKER, I SLAYMAKER, Y YU, B ZETSCHE, Y ARATYN, ... | | 2020 |
| ADENOSINE DEAMINASE BASE EDITORS AND METHODS OF USING SAME TO MODIFY A NUCLEOBASE IN A TARGET SEQUENCE N GAUDELLI, M PACKER, I SLAYMAKER, Y YU, B ZETSCHE, ... WO Patent WO/2020/168,132, 2020 | | 2020 |
Giuseppe CiaramellaVerified email at modernatx.com
