Knockout News and Research

RSS

Researchers develop first non-human primate X-SCID models using genome editing techniques

Researchers affiliated with the Kawasaki INnovation Gateway at SKYFRONT, have successfully generated the first ever non-human primate X-SCID models by using two genome editing techniques. The findings were published in Cell Stem Cell, July 2016.

3 Jan 2017

Novel murine model helps shed light on mechanisms involved in reversible infantile liver failure

Reversible infantile liver failure (RILF) is a heritable mitochondrial condition that causes severe liver dysfunction in infancy, but those who survive the acute stage typically recover and have no further problems.

27 Dec 2016

New TSRI study shows how Piezo2 protein plays critical role in sensing lung expansion

The new study might help shed light on sudden infant death syndrome (SIDS) in human babies, which is thought to be associated with dysfunctional airway sensory neurons.

22 Dec 2016

New study explores circadian timekeeping to create genetic knock-out rescue mice

A new study from the laboratory of Hiroki Ueda at the RIKEN Quantitative Biology Center investigates circadian timekeeping with a novel approach to creating genetic knock-out rescue mice.

22 Dec 2016

Penn researchers use CRISPR/Cas9 gene targeting approach to treat hemophilia B in mice

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations.

1 Dec 2016

Research shows way to directly target genes within specific cell type

The cornerstone of genetics is the loss-of-function experiment. In short, this means that to figure out what exactly gene X is doing in a tissue of interest—be it developing brain cells or a pancreatic tumor—you somehow cut out, switch off or otherwise destroy gene X in that tissue and then watch what happens.

1 Nov 2016

Breakthrough research links sphingolipid-mediated cell junctions and tumorigenesis

Almost all cell types exhibit some sort of polarity, which enables them to carry out specialized functions. Adherens junctions, which consist of the transmembrane protein cadherin and the intracellular components beta-catenin, alpha-catenin, and actin filaments, initiate cell-cell contacts and maintenance of cell polarity.

18 Oct 2016

Discovery of neuropilin 2 deficiency may lead to new therapies for edema and lymphedema

Fluid accumulation and swelling (edema) may result from the malfunctioning of regulatory processes controlling vessel permeability in the body.

14 Oct 2016

New research reveals key protein involved in heart failure

A key protein that causes heart failure has been revealed through new research from a collaboration based in Kumamoto University, Japan.

13 Oct 2016

UTA research opens new doors for treatment of autoimmune diseases

Biologists at The University of Texas at Arlington have demonstrated that removing water molecules can deactivate caspase-3 enzymes, which opens new doors for treatment of autoimmune diseases like arthritis, which have been linked to overactive enzymes.

4 Oct 2016

Imran Noorani receives Brainlab Community Neurosurgery Abstract Award at 2016 CNS annual meeting

Imran Noorani MD, MRCS, of Cambridge, England, received the Brainlab Community Neurosurgery Abstract Award at the Congress of Neurological Surgeons Annual Meeting in San Diego.

28 Sep 2016

Researchers identify mutant traits in mouse for many human disease genes

About one-third of all genes in the mammalian genome are essential for life. An international, multi-institutional research collaboration identified, for the first time, mutant traits in the mouse for 52 human disease genes, which significantly contributes to the understanding of the genetic bases for some human diseases, including cardiovascular defects, spina bifida, and metabolic disorders, among many others. The study was published this week in Nature.

19 Sep 2016

UTHealth scientists discover powerful predictors of congestive heart failure

A team of scientists at The University of Texas Health Science Center at Houston and Baylor College of Medicine, led by Eric Boerwinkle, Ph.D., Richard Gibbs, Ph.D., and Bing Yu, Ph.D., have identified powerful predictors of congestive heart failure, a major cause of hospitalization and death in the United States.

31 Aug 2016

Scientists find way to boost efficiency with which CRISPR-Cas9 cuts and disables genes

CRISPR-Cas9 is the go-to technique for knocking out genes in human cell lines to discover what the genes do, but the efficiency with which it disables genes can vary immensely.

18 Aug 2016

Former SpaceX engineers launch Synthego, CRISPRevolution to accelerate genome engineering research

Synthego, a leading provider of genome engineering solutions, officially launches today. Synthego’s flagship product line, CRISPRevolution, features synthetic guide RNA products designed to accelerate CRISPR/Cas9 and Cpf1 genome engineering research. CRISPRevolution is the first product line to offer economical access to fully synthetic RNA for high fidelity editing and increased precision in genome editing.

12 Aug 2016

Penn study sheds light on role of essential compound in maintaining optimal muscle function

Maintaining proper levels of an essential helper molecule is crucial for optimal muscle function, according to a study led by Joseph Baur, PhD, an assistant professor of Physiology in the Perelman School of Medicine at the University of Pennsylvania.

10 Aug 2016

Scientists link malfunctioning molecular pathways to specific heart abnormalities in SCA

Patients with sickle cell anemia (SCA) develop heart complications and nearly a quarter die a sudden death. Now, researchers have linked malfunctioning molecular pathways to specific heart anomalies in SCA that result from progressive fibrosis and result in sudden death.

9 Aug 2016

Researchers find conceivable alternative way to treat Pompe disease

Researchers at Duke Health have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal muscle, the liver and other tissues, due to deficiency of a particular enzyme.

25 Jul 2016

New TSRI study may bring doctors closer to using gene therapies to grow, repair tendons

With the Rio Olympics just weeks away, many are wondering how Jamaican sprinter Usain Bolt will perform.

19 Jul 2016

Bone marrow transplantation may one day be beneficial to subset of ALS, say researchers

Harvard Stem Cell Institute researchers at Harvard University and the Broad Institute of Harvard and MIT have found evidence that bone marrow transplantation may one day be beneficial to a subset of patients suffering from amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disorder more commonly known as Lou Gehrig's disease.

18 Jul 2016