Search Results

Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting

Mutations of the Janus family kinase JAK3 gene cause severe combined immunodeficiency (SCID). JAK3 deficiency in humans is characterized by the absence of circulating T cells and natural killer (NK) cells with normal numbers of poorly functioning B...

Authors: Chia-Wei Chang, Yi-Shin Lai, Erik Westin, Alireza Khodadadi-Jamayran, Kevin M. Pawlik, Lawrence S....

SST Peer Reviewed

Proteins and Autocrine Signaling Differentially Regulate Gonadotropin Subunit Expression in Pituitary Gonadotrope

Gonadotropin-releasing hormone (GnRH) acts at gonadotropes to direct the synthesis of the gonadotropins, follicle-stimulating hormone (FSH), and luteinizing hormone (LH). The frequency of GnRH pulses determines the pattern of gonadotropin synthesis....

Authors: Soon-Gang Choi, Jingjing Jia, Robert L. Pfeffer, and Stuart C. Sealfon

Differential activation of human constitutive androstane receptor and its SV23 and SV24 splice variants by rilpivirine and etravirine

BACKGROUND AND PURPOSE: Rilpivirine and etravirine are second-generation non-nucleoside reverse transcriptase inhibitors (NNRTIs) indicated for the treatment of HIV/AIDS. The constitutive androstane receptor (CAR) regulates the expression of...

Authors: Sharma D1, Lau AJ, Sherman MA, Chang TK.

Hormone and Drug-Mediated Modulation of Glucose Metabolism in a Microscale Model of the Human Liver.

Due to its central role in glucose homeostasis, the liver is an important target for drug development efforts for type 2 diabetes mellitus (T2DM). Significant differences across species in liver metabolism necessitate supplementation of animal data...

Authors: Davidson MD1, Lehrer M2, Khetani SR1,3

Reprogramming fibroblasts toward cardiomyocytes, neural stem cells and hepatocytes by cell activation and signaling-directed lineage conversion

Induction of tissue-specific cell types via a conventional transdifferentiation strategy typically uses overexpression of the corresponding lineage-specific transcription factors. Alternatively, somatic cells can be temporarily activated via a common...

Authors: Saiyong Zhu, Haixia Wang, Sheng Ding

SST Peer Reviewed

Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.

Mutations of the Janus family kinase JAK3 gene cause severe combined immunodeficiency (SCID). JAK3 deficiency in humans is characterized by the absence of circulating T cells and natural killer (NK) cells with normal numbers of poorly functioning B...

Authors: Chia-Wei Chang, Yi-Shin Lai, Erik Westin, Alireza Khodadadi-Jamayran, Kevin M. Pawlik, Lawrence S....

SST Peer Reviewed

Development of an in vitro human liver system for interrogating nonalcoholic steatohepatitis

A barrier to drug development for nonalcoholic steatohepatitis (NASH) is the absence of translational preclinical human-relevant systems. An in vitro liver model was engineered to incorporate hepatic sinusoidal flow, transport, and lipotoxic stress...

Authors: Feaver RE1, Cole BK1, Lawson MJ1, Hoang SA1, Marukian S1, Blackman BR2, Figler RA1, Sanyal AJ3,...

Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells

Spermatogonial stem cells (SSCs) can produce numerous male gametes after transplantation into recipient testes, presenting a valuable approach for gene therapy and continuous production of gene-modified animals. However, successful genetic...

Authors: Wu Y, Zhou H, Fan X, Zhang Y, Zhang M, Wang Y, Xie Z, Bai M, Yin Q, Liang D, Tang W, Liao J, Zhou C,...

SST Peer Reviewed

CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs

Developing technologies for efficient and scalable disruption of gene expression will provide powerful tools for studying gene function, developmental pathways, and disease mechanisms. Here, we develop clustered regularly interspaced short...

Authors: Mohammad A. Mandegar, Nathaniel Huebsch, Ekaterina B. Frolov, Edward Shin, Annie Truong, Michael P....

SST Peer Reviewed

Combining single-strand oligodeoxynucleotides and CRISPR/Cas9 to correct gene mutations in ß-thalassemia-induced Pluripotent Stem Cells

ß-Thalassemia (ß-Thal) is one of the most common genetic diseases in the world. The generation of patient-specific ß-Thal-induced pluripotent stem cells (iPSCs), correction of the disease-causing mutations in those cells, and then differentiation...

Authors: Xiaohua Niu, Wenyin He, Bing Song, Zhanhui Ou, Di Fan, Yuchang Chen, Yong Fan, Xiaofang Sun

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Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting

Proteins and Autocrine Signaling Differentially Regulate Gonadotropin Subunit Expression in Pituitary Gonadotrope

Differential activation of human constitutive androstane receptor and its SV23 and SV24 splice variants by rilpivirine and etravirine

Hormone and Drug-Mediated Modulation of Glucose Metabolism in a Microscale Model of the Human Liver.

Reprogramming fibroblasts toward cardiomyocytes, neural stem cells and hepatocytes by cell activation and signaling-directed lineage conversion

Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.

Development of an in vitro human liver system for interrogating nonalcoholic steatohepatitis

Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells

CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs

Combining single-strand oligodeoxynucleotides and CRISPR/Cas9 to correct gene mutations in ß-thalassemia-induced Pluripotent Stem Cells