Genome Editing News and Research

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MIT engineers find new strategy to combat superbugs

In recent years, new strains of bacteria have emerged that resist even the most powerful antibiotics. Each year, these superbugs, including drug-resistant forms of tuberculosis and staphylococcus, infect more than 2 million people nationwide, and kill at least 23,000. Despite the urgent need for new treatments, scientists have discovered very few new classes of antibiotics in the past decade.

22 Sep 2014

Researchers use new gene editing method to correct mutation that leads to DMD

UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.

17 Aug 2014

New approach to knocking out parasite's genes

Plasmodium falciparum, the parasite that causes malaria, has proven notoriously resistant to scientists' efforts to study its genetics.

12 Aug 2014

Pfizer and Cellectis enter into global strategic collaboration to develop CAR-T immunotherapies

Pfizer Inc. and Cellectis today announced that they have entered into a global strategic collaboration to develop Chimeric Antigen Receptor T-cell (CAR-T) immunotherapies in the field of oncology directed at select targets.

18 Jun 2014

CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted a $5.6 million Strategic Partnership Award to fund clinical studies at City of Hope to develop a potentially curative ZFP Therapeutic for HIV/AIDS based on the application of Sangamo's zinc finger nuclease (ZFN) genome-editing technology in hematopoietic stem/progenitor cells (HSPCs).

31 May 2014

BRCA gene loss may accelerate cancer-promoting chromosome rearrangements

Inherited mutations in the BRCA1 or BRCA2 tumor suppressor genes are by far the most frequent contributors to hereditary cancer risk in the human population, often causing breast or ovarian cancer in young women of child-bearing age.

29 Apr 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

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