Search Results

Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells

CRISPR/Cas9 has demonstrated a high-efficiency in site-specific gene targeting. However, potential off-target effects of the Cas9 nuclease represent a major safety concern for any therapeutic application. Here, we knock out the Tafazzin gene by...

Authors: Luhan Yang, Dennis Grishin, Gang Wang, John Aach, Cheng-Zhong Zhang, Raj Chari, Jason Homsy, Xuyu...

SST Peer Reviewed

Human bronchial epithelial cells differentiate to 3D glandular acini on basement membrane matrix.

To create a model system that investigates mechanisms resulting in hyperplasia and hypertrophy of respiratory tract submucosal glands, we developed an in vitro three-dimensional (3D) system wherein normal human bronchial epithelial (HBE) cells...

Authors: Wu X, Peters-Hall JR, Bose S, Peña MT, Rose MC

SST Peer Reviewed

Multi-kilobase homozygous targeted gene replacement in human induced pluripotent stem cells

Sequence-specific nucleases such as TALEN and the CRISPR/Cas9 system have so far been used to disrupt, correct or insert transgenes at precise locations in mammalian genomes. We demonstrate efficient 'knock-in' targeted replacement of multi-kilobase...

Authors: Susan M. Byrne, Luis Ortiz, Prashant Mali, John Aach, George M. Church

SST Peer Reviewed

IL-27 inhibits HIV-1 infection in human macrophages by down-regulating host factor SPTBN1 during monocyte to macrophage differentiation

The susceptibility of macrophages to HIV-1 infection is modulated during monocyte differentiation. IL-27 is an anti-HIV cytokine that also modulates monocyte activation. In this study, we present new evidence that IL-27 promotes monocyte...

Authors: Dai L, Lidie KB, Chen Q, Adelsberger JW, Zheng X, Huang D, Yang J, Lempicki RA, Rehman T, Dewar RL,...

SST Peer Reviewed

A Cas9 Ribonucleoprotein Platform for Functional Genetic Studies of HIV-Host Interactions in Primary Human T Cells.

New genetic tools are needed to understand the functional interactions between HIV and human host factors in primary cells. We recently developed a method to edit the genome of primary CD4+ T cells by electroporation of CRISPR/Cas9 ribonucleoproteins...

Authors: Hultquist JF, Schumann K, Woo JM, Manganaro L, McGregor MJ, Doudna J, Simon V, Krogan NJ, Marson A.

SST Peer Reviewed

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects

CRISPR-Cas9 nucleases are widely used for genome editing but can induce unwanted off-target mutations. Existing strategies for reducing genome-wide off-target effects of the widely used Streptococcus pyogenes Cas9 (SpCas9) are imperfect, possessing...

Authors: Kleinstiver BP, Pattanayak V, Prew MS, Tsai SQ, Nguyen NT, Zheng Z, Joung JK

SST Peer Reviewed

Human corneal stromal stem cells support limbal epithelial cells cultured on RAFT tissue equivalents.

Human limbal epithelial cells (HLE) and corneal stromal stem cells (CSSC) reside in close proximity in vivo in the corneal limbal stem cell niche. However, HLE are typically cultured in vitro without supporting niche cells. Here, we re-create the...

Authors: Kureshi AK, Dziasko M, Funderburgh JL, Daniels JT.

SST Peer Reviewed

The Src and c-Kit kinase inhibitor dasatinib enhances p53-mediated targeting of human acute myeloid leukemia stem cells by chemotherapeutic agents

The SRC family kinases (SFKs) and the receptor tyrosine kinase c-Kit are activated in human acute myeloid leukemia (AML) cells. We show here that the SFKs LYN, HCK, or FGR are overexpressed and activated in AML progenitor cells. Treatment with the...

Authors: Dos Santos C, McDonald T, Ho YW, Liu H, Lin A, Forman SJ, Kuo YH, Bhatia R.

Hsp70 Isoforms Are Essential for the Formation of Kaposi\'s Sarcoma-Associated Herpesvirus Replication and Transcription Compartments.

Kaposi's sarcoma-associated herpesvirus (KSHV) is an oncogenic herpesvirus associated with various AIDS-related malignancies. Like other herpesviruses, multiple processes required for KSHV lytic replication, including viral transcription, viral DNA...

Authors: Baquero-Pérez B, Whitehouse A.

Cold-aggravated pain in humans caused by a hyperactive NaV1.9 channel mutant.

Gain-of-function mutations in the human SCN11A-encoded voltage-gated Na(+) channel NaV1.9 cause severe pain disorders ranging from neuropathic pain to congenital pain insensitivity. However, the entire spectrum of the NaV1.9 diseases has yet to be...

Authors: Leipold E, Hanson-Kahn A, Frick M, Gong P, Bernstein JA, Voigt M, Katona I, Oliver Goral R,...

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Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells

Human bronchial epithelial cells differentiate to 3D glandular acini on basement membrane matrix.

Multi-kilobase homozygous targeted gene replacement in human induced pluripotent stem cells

IL-27 inhibits HIV-1 infection in human macrophages by down-regulating host factor SPTBN1 during monocyte to macrophage differentiation

A Cas9 Ribonucleoprotein Platform for Functional Genetic Studies of HIV-Host Interactions in Primary Human T Cells.

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects

Human corneal stromal stem cells support limbal epithelial cells cultured on RAFT tissue equivalents.

The Src and c-Kit kinase inhibitor dasatinib enhances p53-mediated targeting of human acute myeloid leukemia stem cells by chemotherapeutic agents

Hsp70 Isoforms Are Essential for the Formation of Kaposi\'s Sarcoma-Associated Herpesvirus Replication and Transcription Compartments.

Cold-aggravated pain in humans caused by a hyperactive NaV1.9 channel mutant.