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Archive - Aug 13, 2015

Toward Permanent Engineered Solutions to Genetic Diseases; New Advance Enables Evolution of More Targeted Gene Editing Tools; Continuous Directed Evolution of DNA-Binding Proteins Improves TALEN Specificity 100-Fold

In his mind, Dr. Basil Hubbard can already picture a new world of therapeutic treatments for millions of patients just over the horizon. It's a future in which diseases like muscular dystrophy, cystic fibrosis, polycystic kidney disease (PKD), and many others may be treated permanently through the science of genome engineering. Thanks to his latest work, Dr. Hubbard is bringing that future closer to reality. His latest research, published online on August 10, 2015 in Nature Methods, demonstrates a new technology advancing the field of genome engineering. The method significantly improves the ability of scientists to target specific faulty genes, and then "edit" them, replacing the damaged genetic code with healthy DNA. "There is a trend in the scientific community to develop therapeutics in a more rational fashion, rather than just relying on traditional chemical screens," says Dr. Hubbard, now an Assistant Professor of Pharmacology in the University of Alberta's Faculty of Medicine & Dentistry. "We're moving towards a very logical type of treatment for genetic diseases, where we can actually say, 'Your disease is caused by a mutation in gene X, and we're going to correct this mutation to treat it'. In theory, genome engineering will eventually allow us to permanently cure genetic diseases by editing the specific faulty gene(s)." The Nature Methods article is titled “The article is titled “Continuous Directed Evolution of DNA-binding Proteins to Improve TALEN Specificity.” Genome engineering involves the targeted, specific modification of an organism's genetic information. Much as a computer programmer edits computer code, scientists could one day replace a person's broken or unhealthy genes with healthy ones through the use of sequence-specific DNA-binding proteins attached to DNA-editing tools.

Exosome Diagnostics Launches CLIA-Certified Laboratory in Cambridge, MA; Company Uses Exosome-Based Platform for Detection of Exosomal RNA and Cell-Free DNA; Cancer Liquid Biopsy Test to Launch Later in 2015

Exosome Diagnostics, Inc., a developer of revolutionary, biofluid-based molecular diagnostics, today announced, August 12, 2015, that it has opened a Clinical Laboratory Improvement Amendments (CLIA)-Certified laboratory in Cambridge, Massacusetts (near Boston). The state-of-the-art clinical services laboratory will manage analysis and reporting for several liquid biopsy tests that the company plans to launch this year for prostate, lung, and other solid tumor cancers. These novel diagnostics will help clinicians detect disease sooner and obtain access to real-time molecular insights that can help better inform treatment decisions for patients with cancer and other serious diseases. “Exosome Diagnostics is dedicated to effectively solving challenges in health care and overcoming barriers to disease detection, monitoring, and treatment,” said Thomas McLain, CEO of Exosome Diagnostics. “We are taking this important step to centralize our operations at our U.S. corporate headquarters in Cambridge, as we work to seamlessly launch new molecular diagnostics, while also accelerating the application of our technology platform to other important disease areas.” Exosome Diagnostics is currently developing a suite of innovative plasma- and urine-based liquid biopsies that analyze exosomal RNA (exoRNA) for biomarkers. The company’s technology platform is uniquely versatile, offering the additional capability to simultaneously isolate and analyze exoRNA and cell-free DNA (cfDNA) to enhance detection of rare mutations. Exosomes are messenger-containing or bearing vesicles released by all living cells into biofluids, such as plasma/serum, urine, cerebrospinal fluid, and saliva. Exosomes can, and often do, contain RNA, DNA and proteins from their cell of origin.

Clinical Next-Gen Sequencing Reveals Ancient Origins and Evolution of Deadly Lassa Virus

Working as part of an international team in the United States and West Africa, a researcher at The Scripps Research Institute (TSRI) has published new findings showing the ancient roots of the deadly Lassa virus, a relative of Ebola virus, and how Lassa virus has changed over time. “This gives us a clear view of how the virus is evolving, which is important to know as we develop vaccines and therapies,” said TSRI biologist Dr. Kristian G. Andersen, a lead author of the new study. At least 5,000 people die each year from Lassa fever. The virus is spread through contact with urine and droppings from infected Mastomys natalensis rodents (sometimes called “multimammate rats” or “multimammate mice” because of the female’s multiple and prominent mammary glands), which are a natural “reservoir” of the virus, and the disease can spread from human to human. In the new study, published as the cover story of the August 13, 2013 issue of the prestigious journal Cell, the international research team used a technique called next-generation sequencing to analyze genomes of Lassa virus samples taken from wild Mastomys natalensis and human patients in Nigeria and Sierra Leone. —whose senior members included Dr. Pardis Sabeti and Dr. Joshua Levin of Harvard University and the Broad Institute, Dr. Robert F. Garry of Tulane University and Dr. Christian Happi of Nigeria’s Irrua Specialist Teaching Hospital and Sierra Leone’s Kenema Government Hospita. The article is titled “Clinical Sequencing Uncovers Origins and Evolution of Lassa Virus.” The genomic data showed that far-flung strains of Lassa virus share a common ancestor that can be traced back more than 1,000 years to an area today known as Nigeria. This surprised the researchers, as Lassa fever was first described in Nigeria only in 1969. “The virus has very ancient roots,” said Dr. Andersen.