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Recognition determinants of broadly neutralizing human antibodies against dengue viruses.

Dengue disease is caused by four different flavivirus serotypes, which infect 390 million people yearly with 25% symptomatic cases and for which no licensed vaccine is available. Recent phase III vaccine trials showed partial protection, and in...

Authors: Rouvinski A, Guardado-Calvo P, Barba-Spaeth G, Duquerroy S, Vaney MC1, Kikuti CM, Navarro Sanchez...

SST Peer Reviewed

Production of Mutated Porcine Embryos Using Zinc Finger Nucleases and a Reporter-based Cell Enrichment System.

To facilitate the construction of genetically-modified pigs, we produced cloned embryos derived from porcine fibroblasts transfected with a pair of engineered zinc finger nuclease (ZFN) plasmids to create targeted mutations and enriched using a...

Authors: Koo OJ, Park SJ, Lee C, Kang JT, Kim S, Moon JH, Choi JY, Kim H, Jang G, Kim JS, Kim S, Lee BC.

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

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA.

Targeted genomic manipulation by Cas9 can efficiently generate knockout cells and organisms via error-prone nonhomologous end joining (NHEJ), but the efficiency of precise sequence replacement by homology-directed repair (HDR) is substantially lower....

Authors: Richardson CD, Ray GJ, DeWitt MA, Curie GL, Corn JE.

SST Peer Reviewed

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

Leveraging cross-species transcription factor binding site patterns: from diabetes risk loci to disease mechanisms.

Genome-wide association studies have revealed numerous risk loci associated with diverse diseases. However, identification of disease-causing variants within association loci remains a major challenge. Divergence in gene expression due to...

Authors: Claussnitzer M, Dankel SN, Klocke B, Grallert H, Glunk V, Berulava T, Lee H, Oskolkov N, Fadista J,...

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

Allosteric and hyperekplexic mutant phenotypes investigated on an a1 glycine receptor transmembrane structure.

The glycine receptor (GlyR) is a pentameric ligand-gated ion channel (pLGIC) mediating inhibitory transmission in the nervous system. Its transmembrane domain (TMD) is the target of allosteric modulators such as general anesthetics and ethanol and is...

Authors: Moraga-Cid G, Sauguet L, Huon C, Malherbe L, Girard-Blanc C, Petres S, Murail S, Taly A, Baaden M,...

SST Peer Reviewed

Unraveling CRISPR-Cas9 genome engineering parameters via a library-on-library approach

We developed an in vivo library-on-library methodology to simultaneously assess single guide RNA (sgRNA) activity across ~1,400 genomic loci. Assaying across multiple human cell types and end-processing enzymes as well as two Cas9 orthologs, we...

Authors: Raj Chari, Prashant Mali, Mark Moosburner, George M Church

The functional consequences of variation in transcription factor binding.

One goal of human genetics is to understand how the information for precise and dynamic gene expression programs is encoded in the genome. The interactions of transcription factors (TFs) with DNA regulatory elements clearly play an important role...

Authors: Cusanovich DA, Pavlovic B, Pritchard JK, Gilad Y.

SST Peer Reviewed

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Recognition determinants of broadly neutralizing human antibodies against dengue viruses.

Production of Mutated Porcine Embryos Using Zinc Finger Nucleases and a Reporter-based Cell Enrichment System.

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

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA.

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

Leveraging cross-species transcription factor binding site patterns: from diabetes risk loci to disease mechanisms.

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

Allosteric and hyperekplexic mutant phenotypes investigated on an a1 glycine receptor transmembrane structure.

Unraveling CRISPR-Cas9 genome engineering parameters via a library-on-library approach

The functional consequences of variation in transcription factor binding.