High-yield genome engineering in primary cells using a hybrid ssDNA repair template and small-molecule cocktails

Author:

Shy Brian R.ORCID,Vykunta Vivasvan S.ORCID,Ha AlvinORCID,Talbot AlexisORCID,Roth Theodore L.ORCID,Nguyen David N.ORCID,Pfeifer Wolfgang G.ORCID,Chen Yan YiORCID,Blaeschke FranziskaORCID,Shifrut EricORCID,Vedova Shane,Mamedov Murad R.ORCID,Chung Jing-Yi Jing,Li Hong,Yu Ruby,Wu DavidORCID,Wolf Jeffrey,Martin Thomas G.,Castro Carlos E.ORCID,Ye Lumeng,Esensten Jonathan H.,Eyquem JustinORCID,Marson AlexanderORCID

Funder

U.S. Department of Health & Human Services | National Institutes of Health

U.S. Department of Health & Human Services | NIH | National Center for Advancing Translational Sciences

Deutsche Forschungsgemeinschaft

Michelson Prize and Grants

Parker Institute for Cancer Immunotherapy

National Science Foundation

Publisher

Springer Science and Business Media LLC

Subject

Biomedical Engineering,Molecular Medicine,Applied Microbiology and Biotechnology,Bioengineering,Biotechnology

Reference51 articles.

1. Frangoul, H. et al. CRISPR-Cas9 gene editing for sickle cell disease and beta-thalassemia. New Engl. J. Med. 384, 252–260 (2021).

2. Stadtmauer, E. A. et al. CRISPR-engineered T cells in patients with refractory cancer. Science https://doi.org/10.1126/science.aba7365 (2020).

3. US National Laboratory of Medicine. A safety and efficacy study evaluating ctx110 in subjects with relapsed or refractory B-cell malignancies (CARBON). ClinicalTrials.gov https://ClinicalTrials.gov/show/NCT04035434 (2019).

4. US National Laboratory of Medicine. CRISPR-edited allogeneic anti-CD19 CAR-T cell therapy for relapsed/refractory B cell non-Hodgkin lymphoma. ClinicalTrials.gov https://ClinicalTrials.gov/show/NCT04637763 (2020).

5. US National Laboratory of Medicine. Transplantation of clustered regularly interspaced short palindromic repeats modified hematopoietic progenitor stem cells (CRISPR_SCD001) in patients with severe sickle cell disease. ClinicalTrials.gov https://ClinicalTrials.gov/show/NCT04774536 (2021).

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