Genome Editing News and Research

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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

New gene-editing system holds potential for treating many genetic disorders

Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation.

31 Mar 2014

Researchers reveal how genome-editing tool works as 'guided missile' to correct errors in genetic code

Researchers from the Broad Institute and MIT have teamed up with colleagues from the University of Tokyo to form the first high definition picture of the Cas9 complex - a key part of the CRISPR-Cas system used by scientists as a genome-editing tool to silence genes and probe the biology of cells. Their findings, which are reported this week in Cell, are expected to help researchers refine and further engineer the tool to accelerate genomic research and bring the technology closer to use in the treatment of human genetic disease.

13 Feb 2014

Biogen Idec, Sangamo BioSciences partner to develop therapeutics for hemoglobinopathies

Biogen Idec and Sangamo BioSciences, Inc. announced today an exclusive worldwide collaboration and license agreement focused on the development of therapeutics for hemoglobinopathies, inherited conditions that result from the abnormal structure or underproduction of hemoglobin.

9 Jan 2014

Horizon, AstraZeneca partner to explore oncology-relevant genotypes to identify novel drug targets

Horizon Discovery and AstraZeneca have entered into a research, collaboration and license agreement to explore a range of oncology-relevant genotypes with the aim of identifying and validating a number of novel drug targets. This deal is the second collaboration between AstraZeneca and Horizon, and follows the announcement in April 2013 of an oncology discovery program to explore Horizon's first-in-class kinase target program, HD-001.

7 Jan 2014

Researchers use new approach to parse roles of key mutated protein that causes familial AD

Researchers at the University of California, San Diego School of Medicine have used genetic engineering of human induced pluripotent stem cells to specifically and precisely parse the roles of a key mutated protein in causing familial Alzheimer's disease, discovering that simple loss-of-function does not contribute to the inherited form of the neurodegenerative disorder.

15 Nov 2013

Sigma Life Science releases genome editing tool for screening and exploratory studies

Sigma-Aldrich Corporation today announced that Sigma Life Science, its innovative biological products and services business, released Sigma CRISPRs, an inexpensive mammalian genome editing tool suitable for screening and exploratory studies.

27 Aug 2013

Study opens door to possible treatments for variety of mitochondrial diseases

By borrowing a tool from bacteria that infect plants, scientists have developed a new approach to eliminate mutated DNA inside mitochondria-the energy factories within cells. Doctors might someday use the approach to treat a variety of mitochondrial diseases, including the degenerative eye disease Leber hereditary optic neuropathy. The research, published online today in Nature Medicine, was funded by the National Eye Institute, a part of the National Institutes of Health.

5 Aug 2013

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

Horizon forms 3 new Centers of Excellence for gene editing in Asia

Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has established three new Centers of Excellence (CoE's) for gene editing in Asia.

4 Mar 2013

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences, Inc. today reported fourth quarter and full year 2012 financial results and accomplishments.

7 Feb 2013

Novel way to engineer specialized immune cells targeted by the AIDS virus

Researchers at the Stanford University School of Medicine have found a novel way to engineer key cells of the immune system so they remain resistant to infection with HIV, the virus that causes AIDS.

23 Jan 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

Safer alternative to experimental gene therapy against HIV infection

Scientists at The Scripps Research Institute have discovered a surprisingly simple and safe method to disrupt specific genes within cells. The scientists highlighted the medical potential of the new technique by demonstrating its use as a safer alternative to an experimental gene therapy against HIV infection.

2 Jul 2012

Nature publishes Sangamo's gene correction strategy for A1AT deficiency

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a preclinical study demonstrating highly specific, functional correction of the alpha 1-antitrypsin (A1AT) gene defect in patient-derived induced pluripotent stem cells (iPSCs) using zinc finger nucleases.

14 Oct 2011

Sangamo's SB-509 fails to meet key endpoints in Phase 2b study for diabetic neuropathy

Sangamo BioSciences, Inc. today announced that its Phase 2b study (SB-509-901) did not meet its primary or secondary clinical endpoints in subjects with moderate severity diabetic neuropathy (DN) as compared to placebo.

3 Oct 2011

Advancements in use of zinc finger nucleases to produce genetically modified pigs

Sangamo BioSciences, Inc. announced the publication of a study demonstrating the use of zinc finger nucleases (ZFNs) to produce genetically modified pigs. The study, which was published in the Proceedings of the National Academy of Sciences (PNAS), represents a significant advancement in the development of improved, less immunogenic animal tissue as a source for transplant into humans.

22 Jul 2011

STSI receives NIH grant to conduct heart-based research

Researchers looking to find a root cause for heart attacks and coronary artery disease will soon begin using a novel investigative approach that borders on science fiction as they work toward the holy grail of American medicine: preventing the nation's No. 1 killer.

2 Jul 2011

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice.

27 Jun 2011