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...

Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia

Genome editing of allogeneic T cells can provide "off-the-shelf" alternatives to autologous chimeric antigen receptor (CAR) T cell therapies. Disruption of T cell receptor a chain (TRAC) to prevent graft-versus-host disease (GVHD) and removal of CD52...

Authors: Ottaviano G, Georgiadis C, Gkazi SA, Syed F, Zhan H, Etuk A, Preece R, Chu J, Kubat A, Adams S, Veys...

SST Peer Reviewed

Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins

Many genetic diseases are caused by single-nucleotide polymorphisms. Base editors can correct these mutations at single-nucleotide resolution, but until recently, only allowed for transition edits, addressing four out of twelve possible DNA base...

Authors: Chen L, Park JE, Paa P, Rajakumar PD, Prekop HT, Chew YT, Manivannan SN, Chew WL.

SST Peer Reviewed

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

Generation of locus-specific degradable tag knock-ins in mouse and human cell lines

Protein degradation technologies represent a powerful functional genomics tool, allowing fast and controllable target protein depletion. Establishing these systems requires a knock-in of the degradation tag into both endogenous target gene alleles....

Authors: Damhofer H, Radzisheuskaya A, Helin K.

SST Peer Reviewed

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...

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...

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

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

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

Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia

Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins

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

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

Generation of locus-specific degradable tag knock-ins in mouse and human cell lines

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

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