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6404 results sorted by

Non-viral expression of chimeric antigen receptors with multiplex gene editing in primary T cells

Efficient engineering of T cells to express exogenous tumor-targeting receptors such as chimeric antigen receptors (CARs) or T-cell receptors (TCRs) is a key requirement of effective adoptive cell therapy for cancer. Genome editing technologies, such...
Authors: Cappabianca Dan, Li Jingling, Zheng Yueting, Tran Cac, Kasparek Kassandra, Mendez Pedro, Thu Ricky,...
SST Peer Reviewed

Therapeutic adenine base editing of human hematopoietic stem cells

In ß-thalassemia, either -globin induction to form fetal hemoglobin (a22) or ß-globin repair to restore adult hemoglobin (a2ß2) could be therapeutic. ABE8e, a recently evolved adenine base editor variant, can achieve efficient adenine conversion, yet...
Authors: Liao J, Chen S, Hsiao S, Jiang Y, Yang Y, Zhang Y, Wang X, Lai Y, Bauer DE, Wu Y
SST Peer Reviewed

The PP2A-Integrator-CDK9 axis fine-tunes transcription and can be targeted therapeutically in cancer

Gene expression by RNA polymerase II (RNAPII) is tightly controlled by cyclin-dependent kinases (CDKs) at discrete checkpoints during the transcription cycle. The pausing checkpoint following transcription initiation is primarily controlled by CDK9....
Authors: Vervoort SJ, Welsh SA, Devlin JR, Barbieri E, Knight DA, Offley S, Bjelosevic S, Costacurta M,...
SST Peer Reviewed

Integration of ?-deficient CARs into the CD3-zeta gene conveys potent cytotoxicity in T and NK cells

Chimeric antigen receptor (CAR)-redirected immune cells hold significant therapeutic potential for oncology, autoimmune diseases, transplant medicine, and infections. All  approved CAR-T therapies rely on personalized manufacturing using...
Authors: Kath J, Franke C, Drosdek V, Du W, Glaser V, Fuster-Garcia C, Stein M, Zittel T, Schulenberg S,...
SST Peer Reviewed

Improved cytosine base editors generated from TadA variants

Cytosine base editors (CBEs) enable programmable genomic C·G-to-T·A transition mutations and typically comprise a modifed CRISPR–Cas enzyme, a naturally occurring cytidine deaminase, and an inhibitor of uracil repair. Previous studies have shown that...
Authors: Lam DK, Feliciano PR, Arif A, Bohnuud T, Fernandez TP, Gehrke JM, Grayson P, Lee KD, Ortega MA,...
SST Peer Reviewed

Highly scalable arrayed CRISPR mediated gene silencing in primary lungsmall airway epithelial cells

Small airway epithelial cells (SAECs) play a central role in the pathogenesis of lung diseases and are now becoming a crucial cellular model for target identification and validation in drug discovery. However, primary cell lines such as SAECs are...
Authors: Dickson A, Mullooly N, Serrano A, Escudero-Ibarz L, Wiggins C, Gianni D
SST Peer Reviewed

THP-1 reference data: Proposal of an in vitro branched evolution model for cancer cell lines

THP-1 is a representative leukemia cell line and is registered with four different numbers in JCRB and RIKEN BRC cell banks. However, differences between these four lines remain unclear. In our study, these four THP-1 cell lines, JCRB0112, JCRB0112.1...
Authors: Fumio Kasai1,2 | Noriko Hirayama2 | Makoto Fukushima1 | Arihiro Kohara2 |Yukio Nakamura1

Neoantigen-targeted CD8+ T cell responses with PD-1 blockade therapy

Neoantigens are peptides derived from non-synonymous mutations presented by human leukocyte antigens (HLAs), which are recognized by antitumour T cells1-14. The large HLA allele diversity and limiting clinical samples have restricted the study of the...
Authors: Cristina Puig-Saus 1 2 3 4, Barbara Sennino 5, Songming Peng 5, Clifford L Wang 5, Zheng...
SST Peer Reviewed

Variable characteristics overlooked in human K-562 leukemia cell lines with a common signature

K-562 is a well-known in vitro cellular model that represents human leukemia cell lines. Although the K-562 cells have been extensively characterized, there are inconsistencies in the data across publications, showing the presence of multiple K-562...
Authors: Fumio Kasai *, Kumiko Mizukoshi & Yukio Nakamura

Ultra-deep sequencing validates safety of CRISPR/Cas9 genome editing in human hematopoietic stem and progenitor cells

As CRISPR-based therapies enter the clinic, evaluation of safety remains a critical and active area of study. Here, we employ a clinical next generation sequencing (NGS) workflow to achieve high sequencing depth and detect ultra-low frequency...
Authors: Cromer MK, Barsan VV, Jaeger E, Wang M, Hampton JP, Chen F, Kennedy D, Xiao J, Khrebtukova I, Granat...
SST Peer Reviewed
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