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Functional CRISPR dissection of gene networks controlling human regulatory T cell identity

Human regulatory T (Treg) cells are essential for immune homeostasis. The transcription factor FOXP3 maintains Treg cell identity, yet the complete set of key transcription factors that control Treg cell gene expression remains unknown....

Authors: Schumann K, Raju SS, Lauber M, Kolb S, Shifrut E, Cortez JT, Skartsis N, Nguyen VQ, Woo JM, Roth TL,...

Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

Sickle cell disease (SCD) is a monogenic disorder that affects millions worldwide. Allogeneic hematopoietic stem cell transplantation is the only available cure. Here, we demonstrate the use of CRISPR/Cas9 and a short single-stranded oligonucleotide...

Authors: So Hyun Park , Ciaran M Lee , Daniel P Dever , Timothy H Davis , Joab Camarena , Waracharee Srifa ,...

SST Peer Reviewed

Functional CRISPR dissection of gene networks controlling human regulatory T cell identity

Human regulatory T (Treg) cells are essential for immune homeostasis. The transcription factor FOXP3 maintains Treg cell identity, yet the complete set of key transcription factors that control Treg cell gene expression remains unknown....

Authors: Schumann K, Raju SS, Lauber M, Kolb S, Shifrut E, Cortez JT, Skartsis N, Nguyen VQ, Woo JM, Roth TL,...

CRISPR/Cas9 mediated deletion of the adenosine A2A receptor enhances CAR T cell efficacy

Adenosine is an immunosuppressive factor that limits anti-tumor immunity through the suppression of multiple immune subsets including T cells via activation of the adenosine A2A receptor (A2AR). Using both murine and human chimeric antigen...

Authors: Giuffrida L, Sek K, Henderson MA, Lai J, Chen AXY, Meyran D, Todd KL, Petley EV, Mardiana S, Mølck...

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,...

Phenotypic manifestation of a-synuclein strains derived from Parkinson's disease and multiple system atrophy in human dopaminergic neurons

a-Synuclein is critical in the pathogenesis of Parkinson’s disease and related disorders, yet it remains unclear how its aggregation causes degeneration of human dopaminergic neurons. In this study, we induced a-synuclein aggregation in human...

Authors: Tanudjojo B, Shaikh SS, Fenyi A, Bousset L, Agarwal D, Marsh J, Zois C, Heman-Ackah S, Fischer R,...

High-throughput T cell receptor engineering by functional screening identifies candidates with enhanced potency and specificity

A major challenge in adoptive T cell immunotherapy is the discovery of natural T cell receptors (TCRs) with high activity and specificity to tumor antigens. Engineering synthetic TCRs for increased tumor antigen recognition is complicated...

Authors: Rodrigo Vazquez-Lombardi, Johanna S. Jung, Fabrice S. Schlatter, Anna Mei, Natalia Rodrigues...

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

CRISPR-Cas9-Mediated Gene Editing in Mouse Spermatogonial Stem Cells

Precise genome editing is a powerful tool for analysis of gene function. However, in spermatogonial stem cells (SSCs), this still remains a big challenge mainly due to low efficiency and complexity of currently available gene editing techniques. The...

Authors: Wang Y, Ding Y, Li J

Timed inhibition of CDC7 increases CRISPR-Cas9 mediated templated repair

Repair of double strand DNA breaks (DSBs) can result in gene disruption or gene modification via homology directed repair (HDR) from donor DNA. Altering cellular responses to DSBs may rebalance editing outcomes towards HDR and away from other repair...

Authors: Wienert B, Nguyen DN, Guenther A, Feng SJ, Locke MN, Wyman SK, Shin J, Kazane KR, Gregory GL, Carter...

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Functional CRISPR dissection of gene networks controlling human regulatory T cell identity

Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

Functional CRISPR dissection of gene networks controlling human regulatory T cell identity

CRISPR/Cas9 mediated deletion of the adenosine A2A receptor enhances CAR T cell efficacy

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

Phenotypic manifestation of a-synuclein strains derived from Parkinson's disease and multiple system atrophy in human dopaminergic neurons

High-throughput T cell receptor engineering by functional screening identifies candidates with enhanced potency and specificity

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

CRISPR-Cas9-Mediated Gene Editing in Mouse Spermatogonial Stem Cells

Timed inhibition of CDC7 increases CRISPR-Cas9 mediated templated repair