| Rationally designed base editors for precise editing of the sickle cell disease mutation SH Chu, M Packer, H Rees, D Lam, Y Yu, J Marshall, LI Cheng, D Lam, ... The CRISPR Journal 4 (2), 169-177, 2021 | 71 | 2021 |
| 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 | 22 | 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 |
| Compositions and methods for treating glycogen storage disease type 1a B Cafferty, T Bohnuud, LI Cheng, M Packer, Y Aratyn-schaus US Patent App. 18/031,547, 2023 | 1 | 2023 |
| 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 |
| 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 treating transthyretin amyloidosis M Packer, LI Cheng, T Bohnuud US Patent App. 18/507,980, 2024 | | 2024 |
| Optimized Base Editing Reagents Yield More Potent Genetic Correction in a Mouse Model of Alpha-1 Antitrypsin Deficiency MS Packer, D Leboeuf, LI Cheng, R Yang, J Decker, M Otero, LA Barrera, ... MOLECULAR THERAPY 30 (4), 117-118, 2022 | | 2022 |
| 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 |
| 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, ... The Liver Meeting Digital Experience™, 2020 | | 2020 |
| 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 |
| Base Editing of an Allosteric Site within Serpina1 Yields Improved Phenotypes in Models of Alpha-1 Antitrypsin Deficiency V Chowdhary, M Packer, LI Cheng, Y Aratyn-Schaus, T Fernandez, ... MOLECULAR THERAPY 28 (4), 580-580, 2020 | | 2020 |
