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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

New technology advances genome engineering

In his mind, Basil Hubbard can already picture a new world of therapeutic treatments for millions of patients just over the horizon. It's a future in which diseases like muscular dystrophy, cystic fibrosis and many others are treated permanently through the science of genome engineering.

11 Aug 2015

Study finds that gene therapy can clip out genetic material associated with heart failure

Gene therapy can clip out genetic material linked to heart failure and replace it with the normal gene in human cardiac cells, according to a study led by researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.

30 Apr 2015

Researchers develop quality assurance tool for genome editing

A research team at Boston Children's Hospital's Program in Cellular and Molecular Medicine has developed a quality assurance tool for scientists using genome-editing technologies like the increasingly popular CRISPR. The assay, called high throughput genome translocation sequencing (HTGTS), rapidly gauges—for any given gene—these technologies' accuracy and their risk for causing potentially dangerous genomic collateral damage.

9 Feb 2015

Johns Hopkins scientists use genome editing technology to alter human stem cells

A powerful “genome editing” technology known as CRISPR has been used by researchers since 2012 to trim, disrupt, replace or add to sequences of an organism’s DNA.

6 Jan 2015

Scientists demonstrate technique for editing genome in sperm-producing adult stem cells

Scientists at Indiana University and colleagues at Stanford and the University of Texas have demonstrated a technique for "editing" the genome in sperm-producing adult stem cells, a result with powerful potential for basic research and for gene therapy.

16 Dec 2014

MGH investigators develop next-generation genome editing system

A next-generation genome editing system developed by Massachusetts General Hospital investigators substantially decreases the risk of producing unwanted, off-target gene mutations. In a paper receiving online publication in Nature Biotechnology, the researchers report a new CRISPR-based RNA-guided nuclease technology that uses two guide RNAs, significantly reducing the chance of cutting through DNA strands at mismatched sites.

26 Apr 2014

Researchers find new way to repair genetic defects in the skin cells of epidermolysis bullosa patients

A research team led by pediatric blood and marrow transplantation experts Mark Osborn, Ph.D. and Jakub Tolar, M.D., Ph.D. from the Masonic Cancer Center, University of Minnesota, have discovered a remarkable new way to repair genetic defects in the skin cells of patients with the skin disease epidermolysis bullosa.

10 Jun 2013

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

5 Jun 2013

Fralin Life Science Institute researchers use TALENS to alter mosquito genome

Virginia Tech researchers successfully used a gene disruption technique to change the eye color of a mosquito - a critical step toward new genetic strategies aimed at disrupting the transmission of diseases such as dengue fever.

22 Mar 2013

Simple and inexpensive method to cut DNA could transform genetic medicine

A simple, precise and inexpensive method for cutting DNA to insert genes into human cells could transform genetic medicine, making routine what now are expensive, complicated and rare procedures for replacing defective genes in order to fix genetic disease or even cure AIDS.

10 Jan 2013

New technique can precisely add or delete genes in living cells

Researchers at MIT, the Broad Institute and Rockefeller University have developed a new technique for precisely altering the genomes of living cells by adding or deleting genes.

4 Jan 2013

Scientists use two methods to manipulate disease-causing mutations in stem cells

Using two distinct methods, Whitehead Institute researchers have successfully and consistently manipulated targeted genes in both human embryonic stem (ES) cells and induced pluripotent stem (iPS) cells.

16 Jul 2011