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Archive - Dec 10, 2014

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New Technique Permits Rapid, Large-Scale Studies of Gene Function

Using a gene-editing system originally developed to delete specific genes, MIT researchers have now shown that they can reliably turn on any gene of their choosing in living cells. This new application for the CRISPR/Cas9 gene-editing system should allow scientists to more easily determine the function of individual genes, according to Dr. Feng Zhang, the W.M. Keck Career Development Professor in Biomedical Engineering in MIT’s Departments of Brain and Cognitive Sciences and Biological Engineering, and a member of the Broad Institute and MIT’s McGovern Institute for Brain Research. This approach also enables rapid functional screens of the entire genome, allowing scientists to identify genes involved in particular diseases. In a study published online on December 10, 2014 in Nature, Dr. Zhang and colleagues identified several genes that help melanoma cells become resistant to a cancer drug. Silvana Konermann, a graduate student in Dr. Zhang’s lab, and Dr. Mark Brigham, a McGovern Institute postdoc, are the paper’s lead authors. The CRISPR system relies on cellular machinery that bacteria use to defend themselves from viral infection. Researchers have previously harnessed this cellular system to create gene-editing complexes that include a DNA-cutting enzyme called Cas9 bound to a short RNA guide strand that is programmed to bind to a specific genome sequence, telling Cas9 where to make its cut. In the past two years, scientists have developed Cas9 as a tool for turning genes off or replacing them with a different version. In the new study, Dr. Zhang and colleagues engineered the Cas9 system to turn genes on, rather than knock them out.

TriLink BioTech Secures Contract for the Synthesis of Ebola Vaccine

In a December 10, 2014 press release, TriLink BioTechnologies (http://www.trilinkbiotech.com), a leader in manufacturing high-quality nucleic acids, today announced that it secured a contract with Battelle for the manufacture of mRNA to support vaccine development against filoviruses. The contract is in support of a project conducted by Medical Countermeasure Systems (MCS) – Joint Vaccine Acquisition Program (JVAP). MCS-JVAP works in conjunction with the Department of Defense to advance development, testing, FDA licensing, production, and storage of biological defense vaccines. The overall goal of the MCS-JVAP project is to create a trivalent vaccine effective against aerosolized exposure to Ebola Sudan, Ebola Zaire, and Marburg viruses. These viruses are highly lethal and may be used as biological warfare agents. Currently there are limited measures to fight against filovirus infections. TriLink’s role is to synthesize the mRNA needed for preclinical studies and to scale up the process in preparation for the synthesis of clinical grade material. "We are very excited to have been awarded this contract with Battelle for the MCS-JVAP project. TriLink is well known for our expertise in modified nucleic acid chemistry and mRNA manufacturing. This, coupled with our experience in the production of alphavirus replicons aligns well with the fundamental needs of this research, " said TriLink President and CEO, Dr. Richard Hogrefe. “Because we are a pharmaceutical-grade GMP manufacturer, we will be able to see this project into the clinic.” The project is sponsored by the Defense Technical Information Center. ATTN: DTIC-AI, B723 John J. Kingman Rd., Ste 0944, Fort Belvoir, VA 22060-6218.

New Drug Combination for Advanced Breast Cancer Delays Disease Progression

A new combination of cancer drugs delayed disease progression for patients with hormone-receptor-positive metastatic breast cancer, according to a multi-center phase II trial. The findings of the randomized study (S6-03) were to be presented on December 12. 2014 at the 2014 San Antonio Breast Cancer Symposium, held December 9-13, 2014, by Dr. Kerin Adelson, assistant professor of medical oncology at Yale Cancer Center and chief quality officer at Smilow Cancer Hospital at Yale-New Haven. The trial enrolled 118 post-menopausal women with metastatic hormone-receptor-positive breast cancer whose cancer continued to progress after being treated with an aromatase inhibitor. The study, based on work done by Dr. Doris Germain of Mt. Sinai Hospital in New York, found that the combination of the drugs bortezomib and fulvestrant — versus fulvestrant alone — doubled the rate of survival at 12 months and reduced the chance of cancer progression overall. Bortezomib, used most commonly in treating multiple myeloma, is a proteasome inhibitor that prevents cancer cells from clearing toxic material. Fulvestrant causes clumping of the estrogen-receptor protein. When bortezomib blocks the ability of the cell to clear these protein clumps, they grow larger and become toxic to the cancer cells. This, in turn, amplifies the effectiveness of fulvestrant, a drug commonly used in this subset of patients. The drug combination doubled the number of patients whose cancer had not progressed after one year from 14% to 28%, according to Dr. Adelson.