Search Results

Targeted T cell receptor gene editing provides predictable T cell product function for immunotherapy

Adoptive transfer of T cells expressing a transgenic T cell receptor (TCR) has the potential to revolutionize immunotherapy of infectious diseases and cancer. However, the generation of defined TCR-transgenic T cell medicinal products with...

Authors: Thomas R Müller , Sebastian Jarosch 1, Monika Hammel , Justin Leube , Simon Grassmann , Bettina...

SST Peer Reviewed

RNAi Transfection Optimized in Primary Naïve B Cells for the Targeted Analysis of Human Plasma Cell Differentiation

Upon antigen recognition, naïve B cells undergo rapid proliferation followed by differentiation to specialized antibody secreting cells (ASCs), called plasma cells. Increased circulating plasma cells are reported in patients with B cell-associated...

Authors: Shih T, De S, Barnes BJ

Modeling retinitis pigmentosa through patient-derived retinal organoids

Human-induced pluripotent stem cells (hiPSCs) can be differentiated into well-structured retinal organoids. In this protocol, we successfully established 3D retinae from patient-derived hiPSCs and built the retinitis pigmentosa model in vitro....

Authors: Yan-Ping Li , Wen-Li Deng , Zi-Bing Jin

SST Peer Reviewed

A functional genetic toolbox for human tissue-derived organoids

Human organoid systems recapitulate key features of organs offering platforms for modelling developmental biology and disease. Tissue-derived organoids have been widely used to study the impact of extrinsic niche factors on stem cells. However, they...

Authors: Dawei Sun , Lewis Evans , Francesca Perrone , Vanesa Sokleva , Kyungtae Lim , Saba Rezakhani ,...

Rapid, efficient and activation-neutral gene editing of polyclonal primary human resting CD4 + T cells allows complex functional analyses

CD4+ T cells are central mediators of adaptive and innate immune responses and constitute a major reservoir for human immunodeficiency virus (HIV) in vivo. Detailed investigations of resting human CD4+ T cells have been precluded by the absence of...

Authors: Manuel Albanese, Adrian Ruhle, Jennifer Mittermaier, Ernesto Mejías-Pérez, Madeleine Gapp, Andreas...

SST Peer Reviewed

Generation of mesenchyme free intestinal organoids from human induced pluripotent stem cells

Efficient generation of human induced pluripotent stem cell (hiPSC)-derived human intestinal organoids (HIOs) would facilitate the development of in vitro models for a variety of diseases that affect the gastrointestinal tract, such as inflammatory...

Authors: Aditya Mithal, Amalia Capilla,Dar Heinze, Andrew Berical, Carlos Villacorta-Martin, Marall Vedaie,...

High temporal resolution proteome and phosphoproteome profiling of stem cell-derived hepatocyte development

Primary human hepatocytes are widely used to evaluate liver toxicity of drugs, but they are scarce and demanding to culture. Stem cell-derived hepatocytes are increasingly discussed as alternatives. To obtain a better appreciation of the molecular...

Authors: Johannes Krumm, Keisuke Sekine , Patroklos Samaras, Agnieska Brazovskaja , Markus Breunig , Ryota...

Pooled Knockin Targeting for Genome Engineering of Cellular Immunotherapies

Adoptive transfer of genetically modified immune cells holds great promise for cancer immunotherapy. CRISPR knockin targeting can improve cell therapies, but more high-throughput methods are needed to test which knockin gene constructs most potently...

Authors: Roth TL, Li PJ, Blaeschke F, Nies JF, Apathy R, Mowery C, Yu R, Nguyen MLT, Lee Y, Truong A, Hiatt...

SST Peer Reviewed

Reprogramming of human Peripheral Blood Mononuclear Cell (PBMC) from a Chinese patient suffering Duchenne muscular dystrophy to iPSC line (SDQLCHi007-A) carrying deletion of 49-50 exons in the DMD gene

Duchenne muscular dystrophy (DMD), an X-linked genetic disorder characterized by progressive muscle weakness and atrophies affecting skeletal and cardiac muscles, is caused by mutations in dystrophin (DMD) gene that spans 79 exons. Here, we generated...

Authors: Jingyun Guan, Xinnong Liu, Haiyan Zhang, Xiaomeng Yang, Yanyan Ma, Yue Li, Zhongtao Gai, Yi Liu

SST Peer Reviewed

Functional Genomics: Arrayed CRISPR KO Screens

Functional genomics is the discipline in molecular and cellular biology field that studies how genes and pathways contribute to a particular disease phenotype. Functional genomic screens are key in target discovery and hold the promise to enable the...

Authors: Davide Gianni and Leire Escudero-Ibarz

SST Peer Reviewed

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Targeted T cell receptor gene editing provides predictable T cell product function for immunotherapy

RNAi Transfection Optimized in Primary Naïve B Cells for the Targeted Analysis of Human Plasma Cell Differentiation

Modeling retinitis pigmentosa through patient-derived retinal organoids

A functional genetic toolbox for human tissue-derived organoids

Rapid, efficient and activation-neutral gene editing of polyclonal primary human resting CD4 + T cells allows complex functional analyses

Generation of mesenchyme free intestinal organoids from human induced pluripotent stem cells

High temporal resolution proteome and phosphoproteome profiling of stem cell-derived hepatocyte development

Pooled Knockin Targeting for Genome Engineering of Cellular Immunotherapies

Reprogramming of human Peripheral Blood Mononuclear Cell (PBMC) from a Chinese patient suffering Duchenne muscular dystrophy to iPSC line (SDQLCHi007-A) carrying deletion of 49-50 exons in the DMD gene

Functional Genomics: Arrayed CRISPR KO Screens