AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines L Zhang, JA Zuris, R Viswanathan, JN Edelstein, R Turk, B Thommandru, ... Nature communications 12 (1), 3908, 2021 | 102 | 2021 |
Detection and modulation of DNA translocations during multi-gene genome editing in T cells A Bothmer, KW Gareau, HS Abdulkerim, F Buquicchio, L Cohen, ... The CRISPR journal 3 (3), 177-187, 2020 | 48 | 2020 |
RNA interference-based therapeutics: molecular platforms for infectious diseases RJ Sathish Dyawanapelly, Sharwari Bhagwat Ghodke, Ramya Vishwanathan ... Journal of Biomedical Nanotechnology 10 (Number 9), 2014 | 41 | 2014 |
EDIT-301: an experimental autologous cell therapy comprising Cas12a-RNP modified mPB-CD34+ cells for the potential treatment of SCD E De Dreuzy, J Heath, JA Zuris, P Sousa, R Viswanathan, S Scott, ... Blood 134, 4636, 2019 | 27 | 2019 |
A highly efficient transgene knock-in technology in clinically relevant cell types AG Allen, SQ Khan, CM Margulies, R Viswanathan, S Lele, L Blaha, ... Nature biotechnology 42 (3), 458-469, 2024 | 19 | 2024 |
PS1518 Genome Editing Of HBG1/2 Promoter Leads to Robust Hbf Induction In Vivo, While Editing of BCL11A Erythroid Enhancer Results In Erythroid Defects J Heath, E de Dreuzy, M Sanchez, S Haskett, T Wang, P Sousa, G Gotta, ... HemaSphere 3 (S1), 699-700, 2019 | 6 | 2019 |
Comparative studies reveal robust HbF induction by editing of HBG1/2 promoters or BCL11A erythroid-enhancer in human CD34+ Cells but that BCL11A erythroid-enhancer editing is … KH Chang, M Sanchez, J Heath, E deDreuzy, S Haskett, A Vogelaar, ... Blood 132 (Supplement 1), 409-409, 2018 | 6 | 2018 |
Expanding CRISPR genome editing strategies in hematopoietic stem and progenitor cells for the treatment of hematologic diseases E deDreuzy, A Chalishazar, J Heath, CM Margulies, J Labella, ... Blood, The Journal of the American Society of Hematology 130 (Suppl_1), 4619 …, 2017 | 2 | 2017 |
Highly Efficient Multi-Gene Knockout and Transgene Knock-in Using CRISPR-Cas12a in Induced Pluripotent Stem Cells for the Generation of Engineered Cell Immunotherapies J Zuris, CM Margulies, R Viswanathan, JN Edelstein, R Pattali, ... MOLECULAR THERAPY 28 (4), 102-102, 2020 | 1 | 2020 |
SLEEK: A Method for Highly Efficient Knock-In and Expression of Transgene Cargos for Next-Generation Cell-Based Medicines R Viswanathan, AG Allen, SQ Khan, CM Margulies, S Lele, SN Scott, ... MOLECULAR THERAPY 30 (4), 392-392, 2022 | | 2022 |
Strategies for multi-gene editing and reduction of translocations with CRISPR-Cpf1 in T cells for the development of improved cell therapies JA Zuris, A Bothmer, R Viswanathan, HS Abdulkerim, K Gareau, ... MOLECULAR THERAPY 27 (4), 105-106, 2019 | | 2019 |
Highly Efficient Single and Multi-gene Knockout with CRISPR-Cpf1 in T Cells for the Development of Improved Cell Therapies JA Zuris, R Viswanathan, KI Loveluck, TM Swartjes, MS Chin, JW Fang, ... HUMAN GENE THERAPY 29 (12), A103-A103, 2018 | | 2018 |