Search Results

A High-Throughput Platform to Identify Small-Molecule Inhibitors of CRISPR-Cas9

The precise control of CRISPR-Cas9 activity is required for a number of genome engineering technologies. Here, we report a generalizable platform that provided the first synthetic small-molecule inhibitors of Streptococcus pyogenes Cas9 (SpCas9) that...

Authors: Maji B, Gangopadhyay SA, Lee M, Shi M, Wu P, Heler R, Mok B, Lim D, Siriwardena SU, Paul B, Dancík...

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Targeted genome editing in hematopoietic stem/progenitor cells (HSPCs) is an attractive strategy for treating immunohematological diseases. However, the limited efficiency of homology-directed editing in primitive HSPCs constrains the yield of...

Authors: Schiroli G, Ferrari S, Conway A, Jacob A, Capo V, Albano L, Plati T, Castiello MC, Sanvito F,...

Setup of human liver-chips integrating 3D models, microwells and a standardized microfluidic platform as proof-of-concept study to support drug evaluation

Human 3D liver microtissues/spheroids are powerful in vitro models to study drug-induced liver injury (DILI) but the small number of cells per spheroid limits the models’ usefulness to study drug metabolism. In this work, we scale up the number of...

Authors: Benoit Cox, Patrick Barton , Reiner Class, Hannah Coxhead, Claude Delatour , Eric Gillent , Jamie...

An organ-on-chip model of pulmonary arterial hypertension identifies a BMPR2-SOX17-prostacyclin signaling axis

Pulmonary arterial hypertension (PAH) is an unmet clinical need. The lack of models of human disease is a key obstacle to drug development. We present a biomimetic model of pulmonary arterial endothelial-smooth muscle cell interactions in PAH,...

Authors: Alexander J Ainscough , Timothy J Smith , Maike Haensel , Christopher J Rhodes , Adam...

Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV

Human primary natural killer (NK) cells are being widely advanced for cancer immunotherapy. However, methods for gene editing of these cells have suffered low transduction rates, high cell death, and loss of transgene expression after expansion....

Authors: Naeimi Kararoudi M, Likhite S, Elmas E, Yamamoto K, Schwartz M, Sorathia K, de Souza Fernandes...

SST Peer Reviewed

Helios enhances the preferential differentiation of human fetal CD4+ naïve T cells into regulatory T cells

T cell receptor (TCR) stimulation and cytokine cues drive the differentiation of CD4+ naïve T cells into effector T cell populations with distinct proinflammatory or regulatory functions. Unlike adult naïve T cells, human fetal naïve CD4+ T...

Authors: Ng MSF, Roth TL, Mendoza VF, Marson A, Burt TD

Highly efficient CRISPR-Cas9-mediated geneknockout in primary human B cells forfunctional genetic studies of Epstein-Barrvirus infection

Gene editing is now routine in all prokaryotic and metazoan cells but has not received much attention in immune cells when the CRISPR-Cas9 technology was introduced in the field of mammalian cell biology less than ten years ago. This versatile...

Authors: Akidil E, Albanese M, Buschle A, Ruhle A, Pich D, Keppler OT, Hammerschmidt W.

A BAFF ligand-based CAR-T cell targeting three receptors and multiple B cell cancers

B cell-activating factor (BAFF) binds the three receptors BAFF-R, BCMA, and TACI, predominantly expressed on mature B cells. Almost all B cell cancers are reported to express at least one of these receptors. Here we develop a BAFF ligand-based...

Authors: Derek P Wong , Nand K Roy , Keman Zhang , Anusha Anukanth, Abhishek Asthana , Nicole J...

Optimized design parameters for CRISPR Cas9 and Cas12a homology-directed repair

CRISPR-Cas proteins are RNA-guided nucleases used to introduce double-stranded breaks (DSBs) at targeted genomic loci. DSBs are repaired by endogenous cellular pathways such as non-homologous end joining (NHEJ) and homology-directed repair (HDR)....

Authors: Schubert MS, Thommandru B, Woodley J, Turk R, Yan S, Kurgan G, McNeill MS, Rettig GR.

SHP2 blockade enhances anti-tumor immunity via tumor cell intrinsic and extrinsic mechanisms

SHP2 is a ubiquitous tyrosine phosphatase involved in regulating both tumor and immune cell signaling. In this study, we discovered a novel immune modulatory function of SHP2. Targeting this protein with allosteric SHP2 inhibitors promoted anti-tumor...

Authors: Ye Wang , Morvarid Mohseni , Angelo Grauel , Javier Estrada Diez , Wei Guan , Simon Liang , Jiyoung...

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