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CRISPR/Cas9-mediated heterozygous knockout of the autism gene CHD8 and characterization of its transcriptional networks in neurodevelopment

Abstract BACKGROUND: Disruptive mutation in the CHD8 gene is one of the top genetic risk factors in autism spectrum disorders (ASDs). Previous analyses of genome-wide CHD8 occupancy and reduced expression of CHD8 by shRNA knockdown in committed...

Authors: Ping Wang, Mingyan Lin, Erika Pedrosa, Anastasia Hrabovsky, Zheng Zhang, Wenjun Guo, Herbert M....

SST Peer Reviewed

Prediction of drug-induced nephrotoxicity and injury mechanisms with human induced pluripotent stem cell-derived cells and machine learning methods.

The renal proximal tubule is a main target for drug-induced toxicity. The prediction of proximal tubular toxicity during drug development remains difficult. Any in vitro methods based on induced pluripotent stem cell-derived renal cells had not been...

Authors: Kandasamy K1, Chuah JK1, Su R2, Huang P1, Eng KG1, Xiong S1, Li Y1, Chia CS1, Loo LH3, Zink D1

Enhanced NK cell adoptive antitumor effects against breast cancer in vitro via blockade of the transforming growth factor-ß signaling pathway.

Natural killer (NK) cells have great potential for improving cancer immunotherapy. Adoptive NK cell transfer, an adoptive immunotherapy, represents a promising nontoxic anticancer therapy. However, existing data indicate that tumor cells can...

Authors: Zhao Y1, Hu J2, Li R1, Song J1, Kang Y1, Liu S1, Zhang D1.

SST Peer Reviewed

Uptake and Presentation of Myelin Basic Protein by Normal Human B Cells

B cells may play both pathogenic and protective roles in T-cell mediated autoimmune diseases such as multiple sclerosis (MS). These functions relate to the ability of B cells to bind and present antigens. Under serum-free conditions we observed that...

Authors: Marie Klinge Brimnes, Bjarke Endel Hansen, Leif Kofoed Nielsen, Morten Hanefeld Dziegiel, Claus...

Genome Editing of the CYP1A1 Locus in iPSCs as a Platform to Map AHR Expression throughout Human Development

Thearyl hydrocarbon receptor (AHR) is a ligand activated transcription factor that increases the expression of detoxifying enzymes upon ligand stimulation. Recent studies now suggest that novel endogenous roles of the AHR exist throughout...

Authors: BrendenW. Smith, Elizabeth A. Stanford, David H. Sherr, and George J.Murphy

SST Peer Reviewed

Mesenchymal progenitor cells derived from traumatized human muscle

Mesenchymal stem cells (MSCs) derived from adult tissues are an important candidate cell type for cell-based tissue engineering and regenerative medicine. Currently, clinical applications for MSCs require additional surgical procedures to harvest...

Authors: WM Jackson1, AB Aragon1,2, F Djouad1, Y Song1, SM Koehler1, LJ Nesti1,2, and RS Tuan1

Anti-proliferative effects of T cells expressing a ligand-based chimeric antigen receptor against CD116 on CD34(+) cells of juvenile myelomonocytic leukemia.

BACKGROUND: Juvenile myelomonocytic leukemia (JMML) is a fatal, myelodysplastic/myeloproliferative neoplasm of early childhood. Patients with JMML have mutually exclusive genetic abnormalities in granulocyte-macrophage colony-stimulating factor...

Authors: Nakazawa Y, Matsuda K, Kurata T, Sueki A, Tanaka M, Sakashita K, Imai C, Wilson MH, Koike K

SST Peer Reviewed

A platform for rapid prototyping of synthetic gene networks in mammalian cells

Mammalian synthetic biology may provide novel therapeutic strategies, help decipher new paths for drug discovery and facilitate synthesis of valuable molecules. Yet, our capacity to genetically program cells is currently hampered by the lack of...

Authors: Duportet X, Wroblewska L, Guye P, Li Y, Eyquem J, Rieders J, Rimchala T, Batt G, Weiss R.

SST Peer Reviewed

Plasmodium evasion of mosquito immunity and global malaria transmission: The lock-and-key theory.

Plasmodium falciparum malaria originated in Africa and became global as humans migrated to other continents. During this journey, parasites encountered new mosquito species, some of them evolutionarily distant from African vectors. We have previously...

Authors: Molina-Cruz A1, Canepa GE2, Kamath N2, Pavlovic NV2, Mu J2, Ramphul UN2, Ramirez JL2, Barillas-Mury...

SST Peer Reviewed

Pathogenesis of ELANE-mutant severe neutropenia revealed by induced pluripotent stem cells

Severe congenital neutropenia (SCN) is often associated with inherited heterozygous point mutations in ELANE, which encodes neutrophil elastase (NE). However, a lack of appropriate models to recapitulate SCN has substantially hampered the...

Authors: Nayak RC, Trump LR, Aronow BJ, Myers K, Mehta P, Kalfa T, Wellendorf AM, Valencia CA, Paddison PJ,...

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CRISPR/Cas9-mediated heterozygous knockout of the autism gene CHD8 and characterization of its transcriptional networks in neurodevelopment

Prediction of drug-induced nephrotoxicity and injury mechanisms with human induced pluripotent stem cell-derived cells and machine learning methods.

Enhanced NK cell adoptive antitumor effects against breast cancer in vitro via blockade of the transforming growth factor-ß signaling pathway.

Uptake and Presentation of Myelin Basic Protein by Normal Human B Cells

Genome Editing of the CYP1A1 Locus in iPSCs as a Platform to Map AHR Expression throughout Human Development

Mesenchymal progenitor cells derived from traumatized human muscle

Anti-proliferative effects of T cells expressing a ligand-based chimeric antigen receptor against CD116 on CD34(+) cells of juvenile myelomonocytic leukemia.

A platform for rapid prototyping of synthetic gene networks in mammalian cells

Plasmodium evasion of mosquito immunity and global malaria transmission: The lock-and-key theory.

Pathogenesis of ELANE-mutant severe neutropenia revealed by induced pluripotent stem cells