Gene Editing News and Research

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New way to more efficiently deliver CRISPR/Cas9 therapeutic to mice with Tyrosinemia type I

University of Massachusetts Medical School researchers have found a way to more efficiently delivery a CRISPR/Cas9 therapeutic to adult mice with the metabolic disease Tyrosinemia type I that may also prove to be safer for use in humans.

2 Feb 2016

CUMC, Iowa scientists use CRISPR to repair genetic mutation responsible for retinitis pigmentosa

Columbia University Medical Center and University of Iowa scientists have used a new gene-editing technology called CRISPR, to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide.

28 Jan 2016

PGD model could provide policy guideposts for human genome editing

Human genome editing for both research and therapy is progressing, raising ethical questions among scientists around the world.

22 Jan 2016

UC Berkeley researchers make major improvement in CRISPR-Cas9 gene editing technology

University of California, Berkeley, researchers have made a major improvement in CRISPR-Cas9 technology that achieves an unprecedented success rate of 60 percent when replacing a short stretch of DNA with another.

21 Jan 2016

CRISPR/Cas9 gene editing technique could be controlled using RNA-based drugs

19 Jan 2016

Novel drug shows promise against metastatic breast cancer in mouse models

12 Jan 2016

New gene editing technique could hinder retinal degeneration in rats with inherited blindness

A new technique that has the potential to treat inherited diseases by removing genetic defects has been shown for the first time to hinder retinal degeneration in rats with a type of inherited blindness, according to a Cedars-Sinai study.

11 Jan 2016

UT Southwestern scientists use new gene-editing technique to prevent progression of DMD in young mice

Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice.

4 Jan 2016

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

Novel drug shows promise in mouse models of human metastatic breast cancer

A doctor treating a patient with a potentially fatal metastatic breast tumor would be very pleased to find, after administering a round of treatment, that the primary tumor had undergone a change in character - from aggressive to static, and no longer shedding cells that can colonize distant organs of the body. Indeed, most patients with breast and other forms of cancer who succumb to the illness do so because of the cancer's unstoppable spread.

23 Dec 2015

AES Annual Meeting takes our understanding of epilepsy to next level

Epilepsy remains one of the most common neurological conditions, affecting one in 26 Americans in their lifetime, with one-third having a form of the condition that resists treatment or effective management. With those statistics in mind, more than 5,200 neurologists, scientists, nurses and health professionals came to Philadelphia December 4-8 for the 2015 American Epilepsy Society Annual Meeting to discuss new discoveries and emerging technologies that can lead to more effective treatment.

18 Dec 2015

Four journalists from China and India win 2016 EurekAlert! Fellowships for International Science Reporters

Four early-career journalists from China and India have emerged from the fiercest competition to date to win the 2016 EurekAlert! Fellowships for International Science Reporters.

13 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

UB researchers develop a way to ramp up conversion of skin cells into dopamine neurons

For decades, the elusive holy grail in Parkinson's disease research has been finding a way to repair faulty dopamine neurons and put them back into patients, where they will start producing dopamine again. Researchers have used fetal material, which is difficult to obtain and of variable quality. Embryonic stem cells represented a tremendous innovation, but making dopamine neurons from stem cells is a long process with a low yield.

8 Dec 2015

Scientists map out genes that keep cancer cells alive

Scientists have mapped out the genes that keep our cells alive, creating a long-awaited foothold for understanding how our genome works and which genes are crucial in disease like cancer.

26 Nov 2015

CRISPR tool unlocks potential for novel treatments across different therapeutic areas

CRISPR is one of the most exciting advances in gene-based medicine in decades. A uniquely powerful tool for editing the genome, it allows scientists to manipulate DNA with unprecedented precision by deleting unwanted or faulty genes and replacing them with different ones. Its promise in this field of medicine is immense, unlocking the potential for novel treatments across a wide range of therapeutic areas.

22 Nov 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