Category Biology/Biotechnology

In CRISPR/Cas9 advance, T cells successfully edited

CXCR4 is a receptor broadly expressed in cells of immune and the central nervous systems and can mediate migration of resting leukocytes and haematopoietic progenitors. It is also used by strains of HIV1 to gain entry into Helper T cells, and are typically detected at the later stages, associated with a rapid decline in CD4+ T cells and progression to AIDS.

CXCR4 is a receptor broadly expressed in cells of immune and the central nervous systems and can mediate migration of resting leukocytes and haematopoietic progenitors. It is also used by strains of HIV1 to gain entry into Helper T cells, and are typically detected at the later stages, associated with a rapid decline in CD4+ T cells and progression to AIDS.

They disabled a T-cell surface protein CXCR4, exploited by HIV when the virus infects T cells and causes AIDS. The group also successfully shut down PD-1, a protein that has attracted intense interest in the burgeoning field of cancer immunotherapy, as scientists have shown that using drugs to block PD-1 coaxes T cells to attack tumors.

T cells not only stand at the center of many disease processes, but could be easily gathered from pat...

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How the important TOPLESS Plant Protein Interacts with other molecules responsible for Turning Genes Off

The tetrameric TOPLESS complex with the EAR motif peptides bound at its repressor-peptide binding grooves. The repressor peptides are shown as a ball presentation. Credit: Karsten Melcher, Ph.D., Van Andel Research Institute

The tetrameric TOPLESS complex with the EAR motif peptides bound at its repressor-peptide binding grooves. The repressor peptides are shown as a ball presentation. Credit: Karsten Melcher, Ph.D., Van Andel Research Institute

Scientists at Van Andel Research Institute (VARI) have revealed an important molecular mechanism in plants that has significant similarities to certain signaling mechanisms in humans, which are closely linked to early embryonic development and to diseases such as cancer. In plants as in animals and humans, intricate molecular networks regulate key biological functions, such as development and stress responses. When the wrong switches are flipped, genes can be inappropriately turned on or off, leading to the onset of diseases.

“This is really a fundamental discovery –...

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For 1st time, Chromosomal Defects in Hemophilia have been Corrected in patient-specific iPSCs using CRISPR-Cas9 nucleases

CRISPR-Cas9 and targeted sgRNAs can revert large inversions in hemophilia A iPSCs •Endothelial cells derived from corrected iPSCs express correctly spliced Factor VIII •Transplantation of corrected iPSCs can rescue injury mortality in hemophiliac mice •Whole-genome and targeted deep sequencing did not detect off-target mutations Credit: Chul-Yong Park, Duk Hyoung Kim, Jeong Sang Son, Jin Jea Sung, Jaehun Lee, Sangsu Bae, Jong-Hoon Kim, Dong-Wook Kim, Jin-Soo Kim. Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9. Cell Stem Cell, 2015; DOI:

CRISPR-Cas9 and targeted sgRNAs can revert large inversions in hemophilia A iPSCs •Endothelial cells derived from corrected iPSCs express correctly spliced Factor VIII •Transplantation of corrected iPSCs can rescue injury mortality in hemophiliac mice •Whole-genome and targeted deep sequencing did not detect off-target mutations Credit: Chul-Yong Park, Duk Hyoung Kim, Jeong Sang Son, Jin Jea Sung, Jaehun Lee, Sangsu Bae, Jong-Hoon Kim, Dong-Wook Kim, Jin-Soo Kim. Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9. Cell Stem Cell, 2015; DOI:

Hemophilia A occurs in about 1 in 5,000 male births and almost half of severe cases are caused by identified “chromosomal inversions” involving introns 1 and 22 of the F...

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“DNA Ambulance” Researchers Discovered how Severely Damaged DNA is Transported in a Cell and Repaired

 Kinesin-14 promotes DSB mobility.

Kinesin-14 promotes DSB mobility.

It’s a discovery that could unlock secrets into how cancer operates. “Scientists knew that severely injured DNA was taken to specialized ‘hospitals’ in the cell to be repaired, but the big mystery was how it got there,” said Professor Karim Mekhail, He discovered this DNA ambulance, which is a kinesin-14 motor protein complex, by using yeast cells.

Mekhail’s team also found that the DNA hospital, ie nuclear pore complex, repairs damaged DNA inaccurately. This inaccurate fix is important because DNA contains the instructions for all our genetic information. While the repaired DNA can still replicate, it has irregular cell instructions – a scenario that could cause cancer.

Daniel Durocher, Senior Investigator at Mount Sinai’s Lunenfeld-Tanenbaum Research I...

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