Search Results

Efficient C• G-to-G• C base editors developed using CRISPRi screens, target-library analysis, and machine learning

Programmable C•G-to-G•C base editors (CGBEs) have broad scientific and therapeutic potential, but their editing outcomes have proved difficult to predict and their editing efficiency and product purity are often low. We describe a suite of engineered...

Authors: Koblan LW, Arbab M, Shen MW, Hussmann JA, Anzalone AV, Doman JL, Newby GA, Yang D, Mok B, Replogle...

Establishment of induced pluripotent stem cell line CSUASOi004-A by reprogramming peripheral blood mononuclear cells of a PRPF6-related dominant retinitis pigmentosa patient

We have established the patient-specific induced pluripotent stem (iPS) cell line CSUASOi004-A by using peripheral blood mononuclear cells (PBMCs) of a retinitis pigmentosa (RP) patient with a PRPF6 gene mutation (c.G2699A:p.R900H). CSUASOi004-A was...

Authors: Yalan Zhou, Yutong Jing, Shengru Mao, Jian Liu, Zekai Cui, Yini Wang, Juan Chen, Hon Fai Chan, Shibo...

A 3D Human Liver Model of Nonalcoholic Steatohepatitis

Background and Aims: To better understand nonalcoholicsteatohepatitis (NASH) disease progression and to evaluatedrug targets and compound activity, we undertook the development of an in vitro 3D model to mimic liver architecture andthe NASH...

Authors: Marion Duriez , Agnes Jacquet , Lucile Hoet , Sandrine Roche , Marie-Dominique Bock , Corinne Rocher...

SST Peer Reviewed

CRISPR-Cas9-mediated nuclear transport and genomic integration of nanostructured genes in human primary cells

DNA nanostructures are a promising tool to deliver molecular payloads to cells. DNA origami structures, where long single-stranded DNA is folded into a compact nanostructure, present an attractive approach to package genes; however, effective...

Authors: Lin-Shiao E, Pfeifer WG, Shy BR, Saffari Doost M, Chen E, Vykunta VS, Hamilton JR, Stahl EC, Lopez...

CRISPR-based functional genomics in human dendritic cells

Dendritic cells (DCs) regulate processes ranging from antitumor and antiviral immunity to host-microbe communication at mucosal surfaces. It remains difficult, however, to genetically manipulate human DCs, limiting our ability to probe how DCs elicit...

Authors: Marco Jost, Amy N. Jacobson, Jeffrey A. Hussmann, Giana Cirolia, Michael A. Fischbach, Jonathan S....

CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells

Prime editors have been delivered using DNA or RNA vectors. Here we demonstrate prime editing with purified ribonucleoprotein complexes. We introduced somatic mutations in zebrafish embryos with frequencies as high as 30% and demonstrate germline...

Authors: Petri K, Zhang W, Ma J, Schmidts A, Lee H, Horng JE, Kim DY, Kurt IC, Clement K, Hsu JY, Pinello L,...

SST Peer Reviewed

Closed-system transposon-mediated manufacture of GMP grade CAR T-cells via the Lonza Nucleofector LV XL

Background & Aim A method for the non-viral transposon based manufacture of CAR19.41BBz T-cells for the Phase I CARTELL (UTN: U1111- 1204-6974) and AUTOCAR19 (U1111-1226-8682) clinical trials has previously been described. The original method was...

Authors: L.M. Brownrigg, S. Nichols, E. Bosio, B. Carnley, M. Sturm

SST Peer Reviewed

c-Jun overexpression in CAR T cells induces exhaustion resistance

Chimeric antigen receptor (CAR) T cells mediate anti-tumour efects in a small subset of patients with cancer , but dysfunction due to T cell exhaustion is an important barrier to progress. To investigate the biology of exhaustion in human...

Authors: Lynn RC, Weber EW, Sotillo E, Gennert D, Xu P, Good Z, Anbunathan H, Lattin J, Jones R, Tieu V,...

Luminescent peptide tagging enables efficient screening for CRISPR-mediated knock-in in human induced pluripotent stem cells

Human pluripotent stem cells are increasingly used for CRISPR-mediated gene targeting in efforts to generate models of human diseases. This is a challenging task because of the high sensitivity of these cells to suboptimal conditions, including...

Authors: Madsen RR, Semple RK

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

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Efficient C• G-to-G• C base editors developed using CRISPRi screens, target-library analysis, and machine learning

Establishment of induced pluripotent stem cell line CSUASOi004-A by reprogramming peripheral blood mononuclear cells of a PRPF6-related dominant retinitis pigmentosa patient

A 3D Human Liver Model of Nonalcoholic Steatohepatitis

CRISPR-Cas9-mediated nuclear transport and genomic integration of nanostructured genes in human primary cells

CRISPR-based functional genomics in human dendritic cells

CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells

Closed-system transposon-mediated manufacture of GMP grade CAR T-cells via the Lonza Nucleofector LV XL

c-Jun overexpression in CAR T cells induces exhaustion resistance

Luminescent peptide tagging enables efficient screening for CRISPR-mediated knock-in in human induced pluripotent stem cells

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