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Researchers successfully use CRISPR to treat adult mouse model of Duchenne muscular dystrophy

Researchers have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. This marks the first time that CRISPR has successfully treated a genetic disease inside a fully developed living mammal with a strategy that has the potential to be translated to human therapy.

4 Jan 2016

Computers could discover future drugs

The drugs of tomorrow may be discovered by computers. A proof-of-concept study published December 23 in Cell Systems demonstrates that with the right input of data about infectious yeast, a machine algorithm can learn to identify combinations of existing and previously unknown compounds that can work together as antifungal agents.

24 Dec 2015

CRISPR/Cas9 turns hPSCs into cell-based lab model system for polycystic kidney disease

CRISPR/Cas9 is hot. News of the revolutionary gene editing technique that is already shaking up bioscience has finally reached the news media and the public. Now comes a first rate example of how CRISPR is changing the pace of biomedical research by linking up with another cutting edge technology -- human pluripotent stem cells (hPSCs).

12 Dec 2015

CRISPR-Cas9 can help knock out genes in exotic animals

The simplicity of CRISPR-Cas9 gene editing will soon make studying the genes of any organism, from the simplest slime mold to the octopus, as easy as it now is to study the genes controlling development in standard lab animals such as nematodes, fruit flies, frogs and mice.

12 Dec 2015

Johns Hopkins researchers develop method to turn stem cells into retinal ganglion cells

Johns Hopkins researchers have developed a method to efficiently turn human stem cells into retinal ganglion cells, the type of nerve cells located within the retina that transmit visual signals from the eye to the brain. Death and dysfunction of these cells cause vision loss in conditions like glaucoma and multiple sclerosis.

1 Dec 2015

New technique improves accuracy of CRISPR/Cas9 gene editing system

A new CRISPR/Cas9 technology developed by scientists at the University of Massachusetts Medical School is precise enough to surgically edit DNA at nearly any genomic location, while avoiding potentially harmful off-target changes typically seen in standard CRISPR gene editing techniques.

19 Nov 2015

RNA-based drugs provide many advantages over CRISPR/Cas9 gene editing system

In just the past few years, researchers have found a way to use a naturally occurring bacterial system known as CRISPR/Cas9 to inactivate or correct specific genes in any organism. CRISPR/Cas9 gene editing activity runs continuously, though, leading to risk of additional editing at unwanted sites. Now, researchers at University of California, San Diego School of Medicine, Ludwig Cancer Research and Isis Pharmaceuticals demonstrate a commercially feasible way to use RNA to turn the CRISPR-Cas9 system on and off as desired -- permanently editing a gene, but only temporarily activating CRISPR-Cas9.

17 Nov 2015

IDT’s innovative Alt-R CRISPR-Cas9 System improves genome editing potency, reduces cell toxicity

Integrated DNA Technologies, introduces a new approach to CRISPR—its innovative Alt-R CRISPR-Cas9 System, based on the naturally occurring S. pyogenes CRISPR RNA system.

From Integrated DNA Technologies 29 Oct 2015

Researchers develop multiplexed screening approach to study cancer development in mice

In a novel use of the CRISPR/Cas9 system, which can be deployed to switch genes off, researchers from Germany, the UK and Spain have developed a multiplexed screening approach to study and model cancer development in mice. The scientists mutated genes in the adult mouse liver uncovering their cancer-causing roles and determining which combinations of genes cooperate to cause liver cancer.

27 Oct 2015

DNA study reveals basic principle of gene regulation

A study of where and how an enzyme cuts DNA may have inadvertently revealed a basic principle of gene regulation, say researchers in Boston Children's Hospital's Program in Cellular and Molecular Medicine (PCMM).

23 Oct 2015

New RNA tools for CRISPR/Cas9

Integrated DNA Technologies (IDT) will host an informative webinar, entitled ‘New RNA Tools for Optimized CRISPR/Cas9 Genome Editing’, on Wednesday, October 7, 2015. Ashley Jacobi, a scientist in the Molecular Genetics Applied Research Group at IDT, and an expert in the application of synthetic nucleic acids, will present the webinar at 9 am and 1 pm Central Standard Time.

From Integrated DNA Technologies 1 Oct 2015

Scientists identify different CRISPR system with potential to advance genetic engineering

A team including the scientist who first harnessed the revolutionary CRISPR-Cas9 system for mammalian genome editing has now identified a different CRISPR system with the potential for even simpler and more precise genome engineering.

26 Sep 2015

Study uses genome- and epigenome-editing tools to determine disease-causing variants

A paper receiving advance online release in Nature Medicine describes a strategy for meeting one of today's most significant challenges in genomic medicine - determining whether a specific DNA variant in the non-protein-coding genome is the actual disease-causing variant of an associated disease risk.

24 Sep 2015

CRISPR tool streamlines gene editing

Within the past few years, a new technology has made altering genes in plants and animals much easier than before. The tool, called CRISPR/Cas9 or just CRISPR, has spurred a flurry of research that could one day lead to hardier crops and livestock, as well as innovative biomedicines. But along with potential benefits, it raises red flags, according to an article in Chemical & Engineering News (C&EN), the weekly newsmagazine of the American Chemical Society.

10 Sep 2015

UCSF study reveals molecular timing mechanism of stem cells

UC San Francisco researchers have for the first time developed a method to precisely control embryonic stem cell differentiation with beams of light, enabling them to be transformed into neurons in response to a precise external cue.

27 Aug 2015

Researchers uncover genetic circuit involved in obesity

Like many other conditions, obesity is caused by an interplay between genetic and environmental factors. While efforts to combat the obesity epidemic will need to include changes in diet and exercise, insights into the genes involved may also help with prevention and treatment.

20 Aug 2015

Deerfield provides $20 million financing to Editas Medicine

Deerfield Management Company, L.P.'s venture capital fund, Deerfield Healthcare Innovations Fund, L.P., today announced its first therapeutics-based investment by participating, along with Deerfield Private Design Fund III, in providing financing to Editas Medicine. In total, Deerfield invested $20 million in this early-stage institutional financing.

10 Aug 2015

Researchers find way to reverse clotting factor deficiency that triggers hemophilia A

Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end. Their bodies lack the ability to make the clotting factor responsible for the coagulation of blood so any cut or bruise can turn into an emergency without immediate treatment.

24 Jul 2015

Georgia researchers develop new tools to genetically manipulate parasite that causes cryptosporidiosis

Researchers at the University of Georgia have developed new tools to study and genetically manipulate cryptosporidium, a microscopic parasite that causes the diarrheal disease cryptosporidiosis.

16 Jul 2015

Umeå University to present special Jubilee Award to Emmanuelle Charpentier at Annual Celebration Ceremony

Umeå University honours Professor Emmanuelle Charpentier for her pioneering research with the powerful discovery of CRISPR-Cas9, which has already been of great importance by providing new opportunities for Life Science research and its applications in Biology and Medicine. Umeå University will present a special Jubilee Award to Emmanuelle Charpentier

25 Jun 2015