Archive for the ‘Gene Therapy Research’ Category

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Newswise STRATFORD Paola Leone, PhD, the director of the Cell and Gene Therapy Center and a professor of Cell Biology at the Rowan University School of Osteopathic Medicine (RowanSOM), has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke (NINDS) to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a childs life by age 10.

Canavan disease is a fatal, inherited disease caused by a mutation in the aspartaocylase gene, Dr. Leone explained. The disease is characterized by progressive and severe brain atrophy that manifests in delayed development, developmental regression, microcephaly, spasticity, seizures, visual impairment and short life expectancy. There, currently, is no treatment or cure for Canavan disease.

Under Dr. Leones direction, a team of RowanSOM researchers and students will examine the potential of stem cells for the treatment of Canavan disease in an animal model. This new study will build on the research teams preliminary data that demonstrated the successful engraftment of stem cells in animal models.

Our project will generate pre-clinical data to support the development of a stem-cell based therapy for Canavan disease, Dr. Leone said. It will also provide an important opportunity for a new generation of clinical researchers. Both undergraduate and graduate students will participate in this project, providing them with valuable experience to work with an extremely promising therapeutic intervention.

The symptoms of Canavan disease usually appear within the first six months of a childs life. The disease is caused by a genetic mutation that stops cells, called oligodendrocytes, from developing myelin, the fatty substance that coats the nerves in the brain. Without the protective myelin covering, the nerves do not form properly, causing the brain to atrophy. The preliminary research that Dr. Leone conducted showed that the engraftment of stem cells promoted significant recovery of the myelin sheath surrounding the nerves.

Our research represents a significant departure from other studies that have focused solely on strategies to augment the loss of the aspartaocylase function that is highly reduced in the brains of these patients, Dr. Leone said. We believe that any strategy seeking to treat Canavan must include a way to restore the myelin development that is disrupted in children with this disease.

This research is supported by the NINDS of the National Institutes of Health, under grant number 1R15NS088763-01A1.

Journalists wishing to speak with Dr. Leone, should contact Jerry Carey, Rowan University Media and Public Relations at 856-566-6171 or at careyge@rowan.edu.

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Rowan Researcher Targets Stem Cell-Based Therapy for Rare Childhood Disease

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Newswise Working with cells taken from children with a very rare but ferocious form of brain cancer, Johns Hopkins Kimmel Cancer Center scientists have identified a genetic pathway that acts as a master regulator of thousands of other genes and may spur cancer cell growth and resistance to anticancer treatment.

Their experiments with cells from patients with atypical teratoid/rhabdoid tumor (AT/RT) also found that selumetinib, an experimental anticancer drug currently in clinical trials for other childhood brain cancers, can disrupt part of the molecular pathway regulated by one of these factors, according to a research team led by Eric Raabe, M.D., Ph.D., an assistant professor of oncology at the Johns Hopkins University School of Medicine.

AT/RT mostly strikes children 6 and younger, and the survival rate is less than 50 percent even with aggressive surgery, radiation and chemotherapy, treatments that can also disrupt thinking, learning and growth. AT/RT accounts for 1 percent of more than 4,500 reported pediatric brain tumors in the U.S., but it is more common in very young children, and it represents 10 percent of all brain tumors in infants.

Whats exciting about this study is that it identifies new ways we can treat AT/RT with experimental drugs already being tested in pediatric patients, Raabe says. Because few outright genetic mutations and potential drug targets have been linked to AT/RT, Raabe and his colleagues turned their attention to genes that could regulate thousands of other genes in AT/RT cancer cells. Experiments in fruit flies had already suggested a gene known as LIN28 could be important in regulating other genes involved in the development of brain tumors. Specifically, the LIN28 protein helps regulate thousands of RNA molecules in normal stem cells, giving them the ability to grow, proliferate and resist damage.

These factors provide stem cells with characteristics that cancer cells also have, such as resistance to environmental insults. These help tumor cells survive chemotherapy and radiation, says Raabe. These proteins also help stem cells move around the body, an advantage cancer cells need to metastasize.

In a report on one of their studies, published Dec. 26 in the journal Oncotarget, the researchers examined cell lines derived from pediatric AT/RT patients and the tumors themselves. They found that the two members of the LIN28 family of genes were highly expressed in 78 percent of the samples, and that blocking LIN28 expression with specially targeted gene silencers called short hairpin RNAs curbed the tumor cells growth and proliferation and triggered cell death. When Raabe and colleagues blocked LIN28A in AT/RT tumor cells transplanted into mice, they were able to more than double the mices life span, from 48 to 115 days.

Using selumetinib in cell line experiments, the scientists cut AT/RT tumor cell proliferation in half and quadrupled the rate of cell death in some cell lines. Raabe says the drug appeared to be disrupting a key molecular pathway controlled by LIN28.

In a second study, described in the Journal of Neuropathology and Experimental Neurology, Raabe and his colleagues examined another factor in the LIN28 pathway, called HMGA2, which is also highly expressed in AT/RT tumors. They again used short pieces of RNA to silence HMGA2, which led to lower levels of cell growth and proliferation and increased cell death. Blocking HMGA2 also doubled the survival rate of mice implanted with tumors derived from pediatric AT/RT cell lines from 58 to 153 days.

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Master Gene Regulatory Pathway Revealed as Key Target for Therapy of Aggressive Pediatric Brain Cancer

DUBLIN, Feb .10, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/7zf9mz/cell_therapy) has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.

Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.

The cell-based markets was analyzed for 2014, and projected to 2024.The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 294 of these are profiled in part II of the report along with tabulation of 285 alliances. Of these companies, 160 are involved in stem cells. Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 61 Tables and 16 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

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Cell Therapy Report 2014-2020 - Technologies, Markets and Companies

Cancer Center News

New insights into specific gene mutations that arise in glioma, an often deadly form of brain cancer, have pointed to the potential of gene therapy, but it's very difficult to effectively deliver toxic or missing genes to cancer cells in the brain. Now, researchers have used nanoparticles to deliver a new therapy to glioma cells in the brains of rats, prolonging their lives.

Click here to read the full press release.

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Among the research institutions NCI funds across the United States, it currently designates 68 as Cancer Centers. Largely based in research universities, these facilities are home to many of the NCI-supported scientists who conduct a wide range of intense, laboratory research into cancers origins and development. The Cancer Centers Program also focuses on trans-disciplinary research, including population science and clinical research. The centers research results are often at the forefront of studies in the cancer field.

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Hopkins scientists find that in rats new nanoparticle gene therapy strategy effectively treats deadly brain cancer

London, UK (PRWEB) February 02, 2015

Market Publishers Ltd announces that in-demand pipelines assessments worked out by DelveInsight have been recently added to its catalogue.

Gene Therapy Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape. The novel report comprises an all-round analysis of the world gene therapy market by main regions, includes an insightful review of the prevalent market trends and also examines the key market drivers and resistors. It canvasses the competitive pattern and includes gene therapies profiles with detailed product descriptions. Additionally, the research report overviews the latest partnerships and unveils information on the dormant and discontinued pipeline projects. The latest technologies and innovations within the marketplace are overviewed.

Gene Therapy Oncology Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape. The new study offers unbiased insights into the development of the world oncology gene therapy market, identifies the current research therapeutic areas and overviews the key marketed products for gene therapy. Additionally, the new report evaluates the latest developments, outlines the licensing opportunities for gene therapy and unveils data on the pre-clinical and clinical outcomes of the gene therapies. Detailed discussion of the competitive environment, a review of the top market players and their product pipelines are included in the study.

Gene Therapy Central Nervous System Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape. The topical report includes reliable data on the central nervous system gene therapy market. It highlights the competitive environment and contains profiles of the leading firms in the market. The new report proceeds with a profound analysis of the research therapeutic areas within the market. It investigates the marketed and pipeline products for gene therapy and overviews the early market winners for both clinical and preclinical gene therapies. Besides, a deep investigation of the key pipeline projects is presented in the report as well.

Gene Therapy Ophthalmology Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape. The new research publication gives a detailed discussion of the world ophthalmology gene therapy market development along with an outline of the main market drivers and latest trends. It assesses the licensing opportunities for gene therapies, offers unbiased information on the clinical and pre-clinical outcomes of the gene therapies and reviews the pipeline projects. Moreover, the report analyses the leading companies targeting key therapeutic areas, investigates the early market winners for gene therapy and scrutinizes the pipeline for gene therapy.

More in-demand pipelines assessments and market research studies by the publisher can be found at DelveInsight page.

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In-demand Pipelines Assessments by DelveInsight Now Available at MarketPublishers.com

DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/2jkkx2/nucleic_acid) has announced the addition of the "Nucleic Acid Therapies in Oncology Drug Pipeline Update 2015" report to their offering.

Nucleic acid therapies in oncology includes: Gene Therapy, DNA vaccine, Other DNA technologies, RNA Interference (RNAi), Small interfering RNA (siRNA), Antisense RNA and Ribozymes.

There are today 238 companies plus partners developing 274 nucleic acid therapy drugs in 648 developmental projects in cancer. In addition, there are 9 suspended drugs and the accumulated number of ceased drugs over the last years amount to another 201 drugs.

Nucleic Acid Therapies In Oncology Drug Pipeline Update lists all drugs and gives you a progress analysis on each one of them. Identified drugs are linked to 210 different targets. All included targets have been cross-referenced for the presence of mutations associated with human cancer. To date 179 out of the 179 studied drug targets so far have been recorded with somatic mutations.

The software application lets you narrow in on these mutations and links out to the mutational analysis for each of the drug targets for detailed information. All drug targets are further categorized in the software application by 60 classifications of molecular function and with pathway referrals to BioCarta, KEGG, NCI-Nature and NetPath.

Pipeline Breakdown According to Number of Drugs

Delivery Format: Desktop App plus Online Access to Updates (One Year)

For more information visit http://www.researchandmarkets.com/research/2jkkx2/nucleic_acid

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Research and Markets: Nucleic Acid Therapies in Oncology Drug Pipeline Report 2015

Volume 9, Issue 2 Summary

In patients with type 1 diabetes, pancreatic beta cells self-destruct, leaving the body bereft of insulin. Yasuhiro Ikeda, D.V.M., Ph.D., is working to create a customizable gene and stem cell therapy system that will arrest the loss of these beta cells possibly permanently eliminating the need for insulin injections.

Yasuhiro Ikeda, D.V.M., Ph.D., is spearheading stem cell research in the Mayo Clinic Center for Regenerative Medicine.

Nearly everyone knows someone with diabetes it's hard not to. In the United States, 1 in 3 adults and 1 in 6 children have high blood sugar, according to the National Institutes of Health.

After you eat, glucose is absorbed into your bloodstream and carried throughout your body. Insulin a hormone made by beta cells in your pancreas then signals your cells to take up glucose, helping your body turn the food into energy.

With diabetes, this process can go wrong in two basic ways:Type 1 diabetes results from the body's failure to produce insulin;type 2 diabetes occurs when there's plenty of insulin but the cells lose their ability to perceive its signal. In both cases, cells starve.

Living well with diabetes requires a lifelong commitment to monitoring blood sugar, eating properly, exercising regularly and maintaining a healthy weight. People with type 1 diabetes must also rely on insulin replacement therapy, usually through insulin injections. People with type 2 diabetes might need oral medication.

Still, every year, diabetes kills about 70,000 people in the United States and is a contributing cause in another 160,000 deaths each year, according to the Centers for Disease Control and Prevention.

Yasuhiro Ikeda, D.V.M., Ph.D., a molecular biologist at Mayo Clinic in Rochester, Minn., wants to change that.

After beginning his career as a veterinary feline specialist, Dr. Ikeda had to change course when he developed an allergy to his four-legged patients that made it impossible to be in a room with them. He turned his attention toward research and discovered that his interest in molecular virology had human as well as feline applications.

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Gene therapy and regenerative medicine lend hope to ...

THE WOODLANDS VGXI Inc., a Woodlands-based manufacturer of plasmid DNA used in vaccine and gene therapy clinical trials announced an expansion that will double its capacity and likely create jobs, according to VGXI Inc. Business Development Manager Christy Franco.

The expansion will add 3,000 square feet to the building, located at 2700 Research Forest Drive, and will include a small-scale cGMP (compliant with the U.S. Food and Drug Administrations current Good Manufacturing Practice regulations) production plant.

The expansion is in response to recent growth in the market for DNA-based pharmaceuticals.

Weve been seeing it coming, Franco said of the need to expand facilities. Clients need quality DNA for clinical trials.

This is an exciting time for the field of gene therapy and DNA vaccines, Franco state d in a press release. As these technologies mature, more and more companies are moving their products into clinical trials and closer to regulatory approval.

Franco said VGXI is contracted at capacity and the expansion will allow the company to perform multiple client campaigns simultaneously.

VGXI Inc., which employees 40-50 people in The Woodlands, has manufactured plasmid products for clinical trials in the U.S., EU, Asia and Australia over the past 15 years using a patented manufacturing process.

VGXI is a wholly owned subsidiary contract manufacturing organization of GeneOne Life Science Inc. and received the Vaccine Industry Excellence Award for best CMO in 2013 and 2014.

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Woodlands-based DNA manufacturer expands facilities

Pfizer Inc. (NYSE:PFE) announced today two strategic decisions to expand the companys rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. First is an agreement with Spark Therapeutics to develop SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential treatment of Hemophilia B expected to enter Phase 1/2 clinical trials in the first half of 2015. Additionally, Pfizer has appointed Michael Linden, Ph.D., Professor at Kings College London and Director of the University College London Gene Therapy Consortium, who will be with the company for a two-year secondment to lead gene therapy research in the rare disease area.

The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients, said Mikael Dolsten, M.D., Ph.D., president of Worldwide Research and Development at Pfizer. By establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for those suffering from devastating hematologic and neuromuscular diseases.

Agreement with Spark Therapeutics for Hemophilia Research

Philadelphia-based Spark Therapeutics and Pfizer will collaborate to progress the clinical program for SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential treatment of hemophilia B. Pfizer has a long-standing commitment to the hemophilia community and has been providing hemophilia products to patients for more than 17 years.

Pfizer strives to provide meaningful enhancements to the lives of patients with hemophilia, and the agreement with Spark Therapeutics offers an important expansion of Pfizers commitment to the bleeding disorder community and builds on our leading hemophilia portfolio, said Geno Germano, group president, Global Innovative Pharma Business at Pfizer. We believe the SPK-FIX program could add to our existing portfolio of hemophilia products and could pioneer a potential new treatment technology for patients with bleeding disorders.

Under the terms of the agreement, Spark will maintain responsibility for clinical development through Phase 1/2 studies. Pfizer will assume responsibility for pivotal studies, any regulatory approvals and potential global commercialization of the product.

Establishment of Gene Therapy Research in Pfizer Rare Disease

Effective December 1, 2014, Professor Michael Linden has joined Pfizer from his current position at Kings College London, for a two-year secondment to lead gene therapy research within the companys rare disease research area.

The establishment of a gene therapy group under the leadership of Professor Linden will help Pfizer explore the potential of this important technology that could possibly benefit patients living with serious diseases, said Kevin Lee, Ph.D., senior vice president and chief scientific officer of Pfizers Rare Disease Research Unit. Professor Linden brings to Pfizer his extensive expertise in AAV technology obtained from over 20 years working in the field.

Pfizer and Rare Diseases

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Pfizer Expands Rare Disease Research with Establishment of ...

summary

We are investigating possible ways of treating genetic disorders. One method involves gene therapy - introducing 'healthy' copies of genes into a patient's cells. This concept has proved harder to implement than previously thought. For example, the large size of most human genes has necessitated the use of 'stripped-down' versions of these genes. However, minimising the amount of genetic material used can exclude stretches of DNA that would normally control the gene's function.

Safer and more efficient ways of delivering these 'replacement' genes directly to their target cells need to be devised. Research is also required on how to keep the inserted DNA intact and retain its normal functions in the cell. We are very aware of the serious concerns about the safety and effectiveness of gene therapy, and are committed to addressing these issues.

We are also investigating treatments based on cell therapy, and the use of drugs to modify gene expression. In many genetic illnesses, it may even be possible to alter other genes pharmacologically so as to overcome the disease.

Dr Jim Vadolas Cell & Gene Therapy Murdoch Childrens Research Institute Royal Children's Hospital Flemington Road Parkville Victoria 3052 Australia

T +61 3 8341 6232 (Office) T +61 3 8341 6236 (Lab) F +61 3 9348 1391 E jim.vadolas@mcri.edu.au

Group Leader Biography

Dr Jim Vadolas completed his PhD at the Departments of Microbiology and Immunology, University of Melbourne, and postdoctoral training at the Murdoch Childrens Research Institutewith Panos Ioannou and Bob Williamson. In 2005, Jim became group leader of the Cell and Gene Therapy group at the MCRI.He is primarily interested in the development ofnew therapeutic strategies for thalassaemia and related haemoglobinopathies. His work has led the establishment of several new model systems that can be used to identify and evaluate potential therapies. He is currently an Executive Committee member of the Australasian Gene Therapy Society. Jim is also an Executive Committee Member of Thalassaemia Australia.

Project 1: Development of RNAi therapy for thalassaemia

-Thalassaemia is an inherited disease caused by defective synthesis of the -globin chain of haemoglobin, leading to imbalanced globin chains. Excess -chains precipitate in erythroid progenitor cells resulting in cell death, ineffective erythropoiesis and severe anaemia. Decreased -globin chain synthesis leads to milder symptoms, exemplified by individuals who co-inherit -thalassaemia and -thalassaemia. Therefore, a possible therapeutic strategy in the treatment of -thalassaemia could include targeted reduction of -globin chains to mimic co-inheritance of /-thalassaemia. One way of reducing -chain synthesis is by using RNA interfering (RNAi). Numerous studies have shown promising results utilising RNAi in vitro and in vivo. This study will investigate the use of RNAi-mediated reduction of -globin chains for the therapy for -thalassaemia.

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Murdoch Childrens Research Institute : cell & gene therapy

December 17, 2014

Credit: Thinkstock

Chuck Bednar for redOrbit.com Your Universe Online

In a new proof of concept experiment, scientists have managed to edit the genome of sperm-producing adult stem cells, creating a break in the DNA strands of a mutant gene in mouse cells then repairing it by replacing flawed segments with corrected ones.

The process utilized in the study is known as homologous recombination, and researchers from Indiana University, Stanford University and the University of Texas used spermatogonial stem cells (the building blocks for the production of sperm and the only adult stem cells that contribute genetic information to the next generation) to demonstrate their technique.

By repairing flaws in these cells, the study authors said that experts could prevent mutations from being passed onto to future generations. The technique, which is detailed in a recent edition of the journal PLOS One, has tremendous potential for gene therapy as well as basic research.

We showed a way to introduce genetic material into spermatogonial stem cells that was greatly improved from what had been previously demonstrated, co-author Christina Dann, an associate scientist in the Indiana University (IU) Department of Chemistry, said in a statement Monday. This technique corrects the mutation, theoretically leaving no other mark on the genome.

Dann, lead author and former IU research associate Danielle Fanslow, and their colleagues had to overcome a number of difficulties in their research including the fact that spermatogonial stem cells are difficult to isolate, culture and work with. They were only able to create the correct conditions in which to maintain and propagate the cells following years worth of work by scientists at multiple laboratories.

A primary hurdle was to find a way to make specific, targeted modifications to the mutant mouse gene without the risk of disease caused by random introduction of genetic material, the university explained. The researchers used specially designed enzymes, called zinc finger nucleases and transcription activator-like effector nucleases, to create a double strand break in the DNA and bring about the repair of the gene.

Stem cells that were modified in the laboratory were then transplanted into the testes of sterile mice where they grew or colonized, indicating that the stem cells were viable. However, the researchers were unable to breed the mice, though they are do not know if it was abnormalities in the transplanted cells or the recipient testes led to the rodents failure to produce sperm.

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Scientists take step forward in "editing" human genetic mutations

Myriad Genetics Inc. (MYGN) cant block competitors DNA tests to determine risk for breast and ovarian cancer after a U.S. appeals court said three patents on the tests never should have been issued.

The patents cover products of nature and ideas that arent eligible for legal protection, the U.S. Court of Appeals for the Federal Circuit said in an opinion posted today on the courts docket. The court upheld a trial judges decision to allow the competing tests, including those made by Ambry Genetics Corp., to remain on the market.

The tests check genes known as BRCA to determine if there is a hereditary risk of developing the diseases. Myriad had been the only company offering the tests until the U.S. Supreme Court last year limited the ability to obtain patents on human genetic sequences. Some patent claims in this case were similar to those invalidated by the high court, a three-judge panel ruled.

They are structurally identical to the ends of DNA strands found in nature, Circuit Judge Timothy Dyk wrote for the panel. A DNA structure with a function similar to that found in nature can only be patent eligible as a composition of matter if it has a unique structure, different from anything found in nature.

Other claims, involving diagnostic methods, do nothing more than spell out what practitioners already knew -- how to compare gene sequences using routine, ordinary techniques, Dyk said in the opinion.

Myriad said it was disappointed in the decision.

We are currently reviewing the decision and will consider all of our options, Ron Rogers, a Myriad spokesman, said.

Public awareness of the tests has grown since Academy Award-winning actress Angelina Jolie said she had a double mastectomy after Myriads product showed she had a mutation linked to the cancer that killed her mother at 56. Myriad gets the majority of its revenue from the tests, though it has been expanding into other types of tests, such as one for prostate cancer.

In saying the patents are invalid, the Federal Circuit panel went beyond what it was asked -- whether the trial judge was correct to deny Myriads request to block competing sales.

It also illustrates how once cutting-edge steps in diagnosing diseases have become routine, said Matthew Dowd, a patent lawyer with Wiley Rein in Washington who has represented James Watson, the co-discoverer of DNAs double helix.

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Myriad Loses Appeals Court Bid to Block Breast Cancer Tests

Q2115 Yun,J., Sonabend,A.M., Ulasov,I.V., Kim,D.H., Rozhkova,E.A., Novosad,V., Dashnaw,S., Brown,T., Canoll,P., Bruce,J.N., Lesniak,M.S. A novel adenoviral vector labeled with superparamagnetic iron oxide nanoparticles for real-time tracking of viral delivery.JOURNAL OF CLINICAL NEUROSCIENCE 2012; 19(-6-):875-880. >>> Rhodamine-dextran; protein, Ad5-green flourescent; Rat; 2ML1; 96 hours; Animal info (male, Harlan Sprague Dawley, adult); MRI; gene therapy.

Q1884 Jang,E., Albadawi,H., Watkins,M.T., Edelman,E.R., Baker,A.B. Syndecan-4 proteoliposomes enhance fibroblast growth factor-2 (FGF-2)-induced proliferation, migration, and neovascularization of ischemic muscle.PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA 2012; 109(-5-):1679-1684. >>> Fibroblast growth factor-2; syndecan-4, proteoliposome; SC; Rat; 1004; 7-16 days; Controls received mp w/ PBS; animal info (Sprague Dawley); wound clips used; ischemia.

Q1062 Fang,M.R., Wang,J., Huang,J.Y., Ling,S.C., Rudd,J.A., Hu,Z.Y., Yew,D.T., Han,S. The Neuroprotective Effects of Reg-2 Following Spinal Cord Transection Injury.Anatomical Record-Advances in Integrative Anatomy and Evolutionary Biology 2011; 294(-1-):24-45. >>> Regeneration gene protein 2; ciliary neurotrophic factor; Saline; CSF/CNS (intrathecal, subarachnoid space); Rat; 2001D; 2001; 7 days; 24 hours; Controls received mp w/ vehicle; animal info (adult, female, 200-250 g, Sprague Dawley); functionality of mp verified by residual volume; spinal cord injury.

Q0609 Chen,Q.M., Butler,D., Querbes,W., Pandey,R.K., Ge,P., Maier,M.A., Zhang,L.G., Rajeev,K.G., Nechev,L., Kotelianski,V., Manoharan,M., Sah,D.W.Y. Lipophilic siRNAs mediate efficient gene silencing in oligodendrocytes with direct CNS delivery.JOURNAL OF CONTROLLED RELEASE 2010; 144(-2-):227-232. >>> RNA, small interfering; cholesterol-conjugate siRNA; PBS; CSF/CNS (corpus callosum); Rat; 2ML1; 7 days; Controls received mp w/ vehicle; gene therapy; animal info (Male Sprague-Dawley); tissue perfusion (parenchyma); Agents are CNPase siRNA, Cholesterol-CNPas siRNA, Cholesterol-Luciferase siRNA.

P9637 Mookerjee,I., Hewitson,T.D., Halls,M.L., Summers,R.J., Mathai,M.L., Bathgate,R.A.D., Tregear,G.W., Samuel,C.S. Relaxin inhibits renal myofibroblast differentiation via RXFP1, the nitric oxide pathway, and Smad2.FASEB Journal 2009; 23(-4-):1219-1229. >>> Relaxin, recomb. human gene-2; Mice; 1007D; 7 days; Animal info (male, Rlx wt, KO).

P9511 Uchibori,R., Okada,T., Ito,T., Urabe,M., Mizukami,H., Kume,A., Ozawa,K. Retroviral vector-producing mesenchymal stem cells for targeted suicide cancer gene therapy.JOURNAL OF GENE MEDICINE 2009; 11(-5-):373-381. >>> Ganciclovir; IP; Mice (nude); 28 days; Controls received mp w/ PBS; animal info (6 wks old, male, Balb/c, nu/nu); gene therapy.

P9121 Qiang,Y.W., Shaughnessy,JD Jr, Yaccoby,S. Wnt3a signaling within bone inhibits multiple myeloma bone disease and tumor growth.Blood 2008; 112(-2-):374-382. >>> Gene, Wnt3a, recomb.; Bone; Mice (SCID); 1004; 4 weeks; Controls received mp w/ PBS; animal info (Myelomatous SCID-hu); tissue perfusion (myelomatous bone); Wnt3a is a human gene; ALZET pump was "directly connected to the open side of the implanted bone, allowing continual exposure of the myelomatous bone to rWnt3a".

P8865 Kobayashi,M., Okada,T., Murakami,T., Ozawa,K., Kobayashi,E., Morita,T. Tissue-targeted in vivo gene transfer coupled with histone deacetylase inhibitor depsipeptide (FK228) enhances adenoviral infection in rat renal cancer allograft model systems.UROLOGY 2007; 70(-6-):1230-1236. >>> Ganciclovir; IP; Rat; 7 days; Gene therapy; animal info (male, ACI, 6-8 wks old).

P8852 Wang,H.Y., Ghosh,A., Baigude,H., Yang,C.S., Qiu,L.H., Xia,X.G., Zhou,H.X., Rana,T.M., Xu,Z.S. Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression.Journal of Biological Chemistry 2008; 283(-23-):15845-15852. >>> RNA, small interfering, modified; RNA, small interfering; PBS; CSF/CNS (intrathecal, subarachnoid space); Mice (transgenic); 1007D; 2004; 7, 28 days; 72 hours; Controls received mp w/ vehicle; functionality of mp verified by residual volume; dose-response (Fig. 3); no stress (see pg. 15846, 15849); stability verified by 28 days in vivo (see Fig. 2); half-life (p. 15846) "short"; gene therapy; brain tissue distribution; animal info (SOD1G93A Tg); neurodegenerative (ALS); mp + catheter positioning confirmed; Target (SOD1); "when infused at disease onset at the therapeutic dose for 4 weeks, this siRNA slows disease progression without detectable adverse effects." The catheter was implanted between the L5 and L6 vertebra and connected to a primed Alzet osmotic pump with the PE50 tube. The catheter was stitched to the surface muscle, and the Alzet osmotic pumps were placed under the skin on the back of the mouse..

P8528 Neal,Z.C., Sondel,P.M., Bates,M.K., Gillies,S.D., Herweijer,H. Flt3-L gene therapy enhances immunocytokine-mediated antitumor effects and induces long-term memory.CANCER IMMUNOLOGY IMMUNOTHERAPY 2007; 56(-11-):1765-1774. >>> Interleukin-2, recomb. human; SC; Mice; 2001; 4 days; Controls received mp w/ no treatment; cancer (neuroblastoma); peptides; animal info (female, A/J, ICR, 6-8 weeks old); gene therapy.

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References on the Use of ALZET Osmotic Pumps in Gene Therapy

Optogenetics is one of the hottest tools in biomedical research today, a method that uses gene therapy to deliver light-sensitive proteins into specific cells. This new tool allows researchers to interact with a single cell or a network of cells with exquisite precision. Whereas imaging and other technologies allow researchers to watch the brain in action, optogenetics enables them to influence those actions.

With this innovative technique, it is now possible to record neuronal activity during and between seizures, and to test causality and identify potential new therapeutic approaches. Further research could lead to the development of new therapies that could aid more than 300,000 Americans who live with uncontrolled seizures.

A new study that will be featured at the American Epilepsy Society's (AES) 68th Annual Meeting examines the reliability of optogenetics as a method of intervention of temporal lobe seizures, and the role the cerebellum may play in hippocampal function and seizure reduction. Researchers at the University of California Irvine utilized custom-designed software to detect and record chronic, spontaneous seizures in the hippocampus of a mouse model of temporal lobe epilepsy.

Their findings show that when brain cells in the cerebellum were activated by optogenetic lasers in the lateral cerebellar cortex, the duration of temporal lobe seizures decreased significantly. The time between seizures did not dramatically change, indicating the absence of a rebound effect. When light was instead delivered to the midline cerebellum, targeting the vermis, not only were seizures shorter, but there was a significant prolongation of the time between seizures. This reaction far outlasted the duration of the applied light, and was unique to application in the vermis. Optogenetic inhibition, rather than excitation, of brain cells in the cerebellum produced no significant effect on time to next seizure, indicating that activation of these cells is required to see this unique effect on seizure frequency.

"These findings are really exciting," said Dr. Esther Krook-Magnuson, Postdoctoral Scholar at the University of California Irvine. "They demonstrate that two brain structures not typically thought to interact can powerfully influence each other, and that the cerebellum could be a good target for intervention in epilepsy."

The data collected from this study shows the incredible potential for developing novel optogenetics treatments for epilepsy and provides strong support for further research.

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The above story is based on materials provided by American Epilepsy Society (AES). Note: Materials may be edited for content and length.

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Optogenetics: Identifying new targets for intervention

Nov 12, 2014 The HIV protein integrase (blue) can insert viral DNA (red) at different locations in the DNA of its human host (orange). But how the virus selects its insertion points has puzzled virologists for over 20 years. Now a team of KU Leuven researchers have discovered that the answer lies in two -- of the more than 200 -- amino acids that make up integrase's structure. Credit: Jonas Demeulemeester

The human immunodeficiency virus (HIV) can insert itself at different locations in the DNA of its human host - and this specific integration site determines how quickly the disease progresses, report researchers at KU Leuven's Laboratory for Molecular Virology and Gene Therapy. The study was published online today in the journal Cell Host & Microbe.

When HIV enters the bloodstream, virus particles bind to and invade human immune cells. HIV then reprogrammes the hijacked cell to make new HIV particles.

The HIV protein integrase plays a key role in this process: it recognises a short segment in the DNA of its host and catalyzes the process by which viral DNA is inserted in host DNA.

Integrase can insert viral DNA at various places in human DNA. But how the virus selects its insertion points has puzzled virologists for over 20 years.

Now a team of KU Leuven researchers has discovered that the answer lies in two amino acids. Doctoral researcher Jonas Demeulemeester, first author of the study, explains: "HIV integrase is made up of a chain of more than 200 amino acids folded into a structure. By modelling this structure, we found two positions in the protein that make direct contact with the DNA of the host. These two amino acids determine the integration site. This is not only the case for HIV but also for related animal-borne viruses."

In a second phase of the study, the researchers were able to manipulate the integration site choice of HIV, explains Professor Rik Gijsbers. "We changed the specific HIV integrase amino acids for those of animal-borne viruses and found that the viral DNA integrated in the host DNA at locations where the animal-borne virus normally would have done so."

"We also showed that HIV integrases can vary," says Professor Rik Gijsbers. "Sometimes different amino acids appeared in the two positions we identified. These variant viruses also integrate into the host DNA at a different site than the normal virus does."

Together with Dr. Thumbi Ndung'u (University of KwaZulu-Natal, Durban, South Africa), the team studied the impact of these viral variants on the progression towards AIDS in a cohort of African HIV patients, continues Professor Zeger Debyser: "To our surprise, we found that the disease progressed more quickly when the integration site was changed. In other words, the variant viruses broke down the immune system more rapidly. This insight both increases our knowledge of the disease and opens new perspectives. By retargeting the integration site to a 'safer' part of the host DNA, we hope to eventually develop new therapies."

Explore further: Redesigned protein opens door for safer gene therapy

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HIV virulence depends on where virus inserts itself in host DNA

Cancer Gene Therapy is the essential gene and cellular therapy resource for cancer researchers and clinicians, keeping readers up to date with the latest developments in gene and cellular therapies for cancer. The journal publishes original laboratory and clinical research papers, case reports and review articles. Publication topics include RNAi approaches, drug resistance, hematopoietic progenitor cell gene transfer, cancer stem cells, cellular therapies, homologous recombination, ribozyme technology, antisense technology, tumor immunotherapy and tumor suppressors, translational research, cancer therapy, gene delivery systems (viral and non-viral), anti-gene therapy (antisense, siRNA & ribozymes), apoptosis; mechanisms and therapies, vaccine development, immunology and immunotherapy, DNA synthesis and repair.

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Volume 21, No 10 October 2014 ISSN: 0929-1903 EISSN: 1476-5500

2013 impact factor 2.553* 52/122 Medicine, Research & Experimental 58/165 Biotechnology & Applied Micobiology 115/202 Oncology 85/164 Genetics & Heredity

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Following the success of this previous study on p53, Cancer Gene Therapy has published another groundbreaking review on this molecule. An overview of results supported by The International Agency for Research on Cancer (part of the World Health Organization), the review highlights the biological properties of mutant p53, including novel molecular targets for the development of future cancer therapies.

Announcing Cancer Gene Therapy Open Cancer Gene Therapy now offers authors the option to publish their articles with immediate open access upon publication. Open access articles will also be deposited on PubMed Central at the time of publication and will be freely available immediately. Find out more from the press release or our FAQs page.

Latest research highlights and reviews from the NPG family of journals

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Cancer Gene Therapy - Nature

One of the countrys premiere pediatric hospitals is challenging the notion that human genes can be patented by filing a lawsuit that, if successful, could lead to a rewriting of patent law and sharply advance the advent of personalized medicine.

The Childrens Hospital of Eastern Ontario argues in court filings that restricting access to genetic information by researchers and clinicians undermines patient care and is morally and legally untenable.

No one should be able to patent human DNA. Its like trying to patent water or air, said Alex Munter, chief executive officer of CHEO. He noted that Canada is one of the only jurisdictions in the Western world that still allows gene patenting.

This poses a significant obstacle to diagnosing and caring for children with a genetic condition and that cant be tolerated, he said. Conversely, striking down the law, will open the door to an era of personalized medicine, where treatments are tailored to specific genetic characteristics.

Mr. Munter said CHEO is taking on the case because it is a leader in genetic research, particularly in the field of rare diseases, but many institutions will benefit if it is successful.

Lawyers for the hospital who are working pro bono filed papers in the Federal Court of Canada on Monday challenging five patents related to genes associated with a heart condition called long QT syndrome.

It is a test case that the hospital hopes will result in parts of the Patent Act being struck down. There are about 7,000 disease genes that are amenable to patenting under existing legislation in Canada.

This is the first Canadian court case to ask the question: Are human genes patentable? said Nathaniel Lipkus of the law firm Gilberts LLP.

Last year, the U.S. Supreme Court ruled that genes can no longer be patented. Biotech companies want to patent genes so they can profit from testing of those genes. When patents are struck down as in the U.S. a company can still market tests but cannot do so exclusively, so the price drops significantly.

The patents being challenged by CHEO are held by the University of Utah but were filed in Canada.

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Bad patents on human genes hinder health care, hospital says

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Newswise The recipient of the 2014 Van Meter Award, established in 1930 by the American Thyroid Association for outstanding contributions by a young clinical scientist to research on the thyroid gland is Christine Spitzweg, M.D., Professor in Internal Medicine/Endocrinology, Chair of the Thyroid Center, Co-chair of the Center for Neuroendocrine Tumors, and Head of the research laboratory for Molecular Endocrinology at Ludwig-Maximilians-University Munich, Germany. Dr. Spitzweg delivered the Van Meter Award lecture, entitled The sodium iodide symporter its evolving role as theranostic gene in and outside of the thyroid gland at the 84th Annual Meeting of the American Thyroid Association on Friday, October 31st, in Coronado, California. The Van Meter Award receives support from Mary Ann Liebert, Inc., publishers of the journal Thyroid, and a generous endowment gift in memory of Jack Robbins.

Dr. Spitzweg has had a pioneering role in pursuing the medical applications of the sodium-iodide symporter (NIS) in diagnostics and in therapeutic gene therapy. Her research helped lay the foundation for the field of therapeutic applications of NIS. She is a Principal Investigator on a Nationwide Priority Program in German focusing on "Translation of Thyroid Hormone Actions beyond Classical Concepts," which explores the use of NIS as a reporter gene to investigate non-genomic effects of the thyroid hormones T4 and T3 and the thyroid hormone analogue tetraiodothyroacetic acid (tetrac) on the tumor microenvironment.

Dr. Spitzweg is also the Principal Investigator of a large collaborative research grant to study image-guided, tumor-targeted radionuclide therapy in disseminated tumors using NIS as theranostic gene. She has conducted numerous studies designed to determine the factors that regulate endogenous NIS gene expression in various tissues and has applied NIS gene therapy to models of several cancers, including prostate cancer, hepatocellular and colon cancer and medullary thyroid cancer.

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The American Thyroid Association (ATA) is the leading worldwide organization dedicated to the advancement, understanding, prevention, diagnosis, and treatment of thyroid disorders and thyroid cancer. ATA is an international membership medical society with over 1,700 members from 43 countries around the world. Celebrating its 91st anniversary, the ATA delivers its mission of being devoted to thyroid biology and to the prevention and treatment of thyroid disease through excellence in research, clinical care, education, and public health through several key endeavors: the publication of highly regarded professional journals, Thyroid, Clinical Thyroidology, and VideoEndocrinology; annual scientific meetings; biennial clinical and research symposia; research grant programs for young investigators, support of online professional, public and patient educational programs; and the development of guidelines for clinical management of thyroid disease and thyroid cancer. The ATA promotes thyroid awareness and information through its online Clinical Thyroidology for the Public (distributed free of charge to over 11,000 patients and public subscribers) and extensive, authoritative explanations of thyroid disease and thyroid cancer in both English and Spanish. The ATA website serves as the clinical resource for patients and the public who look for reliable information on the Internet.

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2014 Van Meter Award Winner Announced

The latest research into HIV/AIDS is coming up with some exciting new treatments which can dramatically improve and prolong the life of someone infected with the virus.

Unfortunately the elusive cure is still a long way off though its not for want of trying. Numerous studies are underway looking at gene therapy and an HIV vaccine, all of which offers hope for the future.

One of the most interesting developments comes from the National Institute of Medical Research where scientists have discovered that a gene found in rhesus monkeys can prevent HIV. The same gene in humans cant block the virus but it appears that only one change is needed to enable it to do so. If this proves to be the case it would be a remarkable breakthrough in the search for a cure.

Its a brave new world but its complicated stuff and a cure certainly isnt just around the corner. In a nutshell, this pioneering gene therapy would involve removing all the white blood cells from a patient, cloning them and altering the genetics before introducing them back into the body. Existing technology cant actually do this so its something for the longer term. But its still a viable possibility and thanks to antiretroviral drugs keeping people alive longer, this treatment may be available in future to people currently living with the virus.

Other gene therapy studies involve inserting modified genes directly into cells to prevent the virus from reproducing itself. These cells produce the CD4 cells which can resist the HIV infection.

The hunt for an HIV vaccine has come a long way. Dozens of experimental vaccines have been tested so far. They are either preventative (designed to stop someone from getting the virus in the first place) or therapeutic with the aim of aiding an infected person to recover from the virus.

Once again these possible solutions to the HIV crisis are a very long way off despite nearly 15 years of research. People respond in different ways to the infection, HIV is still not fully understood and its a very changeable virus so different preventative vaccines may be needed.

Studies are underway into microbibe which is a form of lubricant capable of reducing the transmission of HIV when applied in the vagina or anus. Around 60 different products are being tested and 12 of these have been found to be safe and effective in animals. Microbibe is now being tested on humans and if successful could be available as early as 2007.

But the majority of current research is focusing on the development of antiretrovirals and improving their effectiveness.

These new drugs include:

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Latest AIDS Research, Vaccine, Gene Therapy, New Drugs

There are numerous uses for biotechnology, and the implementation of biotech solutions is becoming increasingly popular as more advances occur within the field. No-one quite knows where the technology may lead, and could possibly one day be found to be implemented in the production of products as diverse as puppy food to the creation of more sustainable buildings.

As mentioned elsewhere on this site, one of the most exciting developments for biotech has been in the domain of medical science: our increasing knowledge of the molecular processes in cells has multiple implications for the treatments of disease and illness.

A relatively new method of treatment made possible through the use of natural processes as a technology/tool is gene therapy. Although still in its infancy (and in many ways its experimental stage) gene therapy might well prove a miracle cure for a vast range of diseases in the future.

Gene therapy is earmarked as a highly plausible future means of treatment, or even cure, of a variety of genetic and acquired diseases. Notably, this list includes cancer and AIDS. In gene therapy, normal genes are inserted into pathological cells to either replace or bolster the cells normal functioning. The cells that gene therapy methods can target are somatic cells and gamete cells. The difference in effect between the two cell types is basically that the modification made to the genetic code of the somatic cell is not passed on to its daughter cell when it divides, but in the gamete cell the modification is passed on in cell division. The intervention in sperm and egg cells (gametes) is therefore for the purposes of generational change. Theoretically, one well designed intervention could remove a genetic defect from a family tree for all future generations: the gene therapy would therefore only have to be applied to one generation of gamete cells.

Gene therapy can be enacted either while cells are in the patients body or the cells can be removed and the process implemented within a laboratory: the former is known as In Vivo treatment and the latter as Ex Vivo treatment.

Unfortunately, gene therapy has many challenges to overcome before it can be used on a widespread and large scale. Four central issues have been identified as follows:

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Gene Therapy Biotechnology - Life Lab

PBR Staff Writer Published 27 October 2014

Bayer HealthCare has signed a two-year collaboration agreement with Kyoto Universitys Office of Society-Academia Collaboration for Innovation (KU-SACI) in Japan to jointly discover candidates for possible collaborative research projects.

Under the deal, the two parties will focus on key areas of unmet medical need such as cardiology, oncology, hematology, gynecology and ophthalmology, by combining Kyoto University's expertise and innovative approaches in diverse research areas with Bayer's expertise in drug discovery and development.

The deal is part of Bayer's aim to collaborate with external partners from academia and industry to develop new treatments for patients across the world.

The company has recently entered into strategic research alliance in the area of gynecological therapies with the University of Oxford as well as with Dimension Therapeutics to develop and commercialize new gene therapy to treat hemophilia A.

In addition, the company has established two research incubators for young life sciences companies in the context of its open innovation approach.

The joint effort will be supported by Bayer's newly established Open Innovation Center Japan (ICJ) in Osaka, Japan.

The threefold mission of the KU-SACI is to promote collaborative research among academia, industries and the government; manage and use the university's intellectual properties through licensing & research collaboration with industries; as well as to support business start-ups by university researchers or students.

Image: Bayer and KU-SACI members during signing of the agreement. Photo: courtesy of Bayer HealthCare AG.

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Bayer, Kyoto University partner on collaborative research projects

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20-Oct-2014

Contact: Todd Murphy murphyt@ohsu.edu 503-494-8231 Oregon Health & Science University @ohsunews

Oregon Health & Science University's Vaccine & Gene Therapy Institute (OHSU), has been awarded a $10 million contract from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). Kineta, Inc., the University of Washington Center for Innate Immunity and Immune Disease (CIIID), and the Vaccine & Gene Therapy Institute of Florida (VGTI Florida) will collaborate with OHSU as major sub-contractors to develop new vaccine adjuvants that could boost the effectiveness of a wide range of human vaccines for infectious diseases including West Nile Virus, Dengue and Japanese Encephalitis. The work will also provide novel applications for enhancing the immune response against Ebola virus, HIV, and other virus infections. Infectious diseases affect millions of people in developed and developing nations, many with no effective protective vaccines.

Vaccines are the first line of defense against infectious disease and have saved millions of lives over the years. However, some people with weakened immune systems and the elderly lose the ability to respond to vaccines. The effectiveness of vaccines can be improved by the addition of substances called adjuvants that not only enhance the body's immune response to the vaccine but also decrease the dose of the vaccine, allowing the vaccine supply to be extended.

Currently, the Food and Drug Administration has approved only three vaccine adjuvants. This award is part of an NIH push to develop more adjuvants.

"Although vaccines are extremely effective at preventing disease, the elderly and infants, who are the most vulnerable part of our population, are not efficiently protected," said Jay Nelson, Ph.D., professor and director of OHSU's VGTI, who will co-lead work on the NIH contract. "For example, while 80 percent of normal healthy adults are protected with the flu vaccine, more than 40 percent of people over 65 do not develop protective immunity. We have found that the addition of adjuvants to vaccines can better protect older animals from virus infection," Nelson added.

Nelson and other OHSU VGTI scientists will work with Shawn Iadonato, Ph.D., Chief Scientific Officer for Kineta, Inc., a biotechnology company in Seattle, on the development of new adjuvants using the company's innate immune drug development platform.

"Kineta has significant experience in high through-put methods to identify chemical compounds that stimulate the immune system and that are safe and effective. Developing these new adjuvants could change the paradigm for generating lasting immunity to pathogens," said Dr. Iadonato.

Michael Gale, Jr., Ph.D., Professor of Immunology and Director of the CIIID at the University of Washington, will co-lead this project with Nelson. "The identification of new adjuvants will also be important to make vaccines for other diseases such as Ebola virus, influenza A virus, HIV, bacterial infection, and cancer more effective," Gale said.

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OHSU, partners Kineta, UW, VGTI Florida awarded NIH contract to develop vaccine adjuvants

Researchers are looking at different ways of using gene therapy, including

Some types of gene therapy aim to boost the body's natural ability to attack cancer cells. Our immune system has cells that recognise and kill harmful things that can cause disease, such as cancer cells.

There are many different types of immune cell. Some of them produce proteins that encourage other immune cells to destroy cancer cells. Some types of therapy add genes to a patient's immune cells to make them better at finding or destroying particular types of cancer. There are a few trials using this type of gene therapy in the UK.

Some gene therapies put genes into cancer cells to make the cells more sensitive to particular treatments such as chemotherapy or radiotherapy. This type of gene therapy aims to make the other cancer treatments work better.

Some types of gene therapy deliver genes into the cancer cells that allow the cells to change drugs from an inactive form to an active form. The inactive form of the drug is called a pro drug.

After giving the carrier containing the gene, the doctor gives the patient the pro drug. The pro drug may be a tablet or capsule that you swallow, or you may have it into the bloodstream.

The pro drug circulates in the body and doesn't harm normal cells. But when it reaches the cancer cells, the gene activates it and the drug kills the cancer cells.

Some gene therapies block processes that cancer cells use to survive. For example, most cells in the body are programmed to die if their DNA is damaged beyond repair. This is called programmed cell death or apoptosis. But cancer cells block this process so they don't die even when they are supposed to. Some gene therapy strategies aim to reverse this blockage. Doctors hope that these new types of treatment will make the cancer cells die.

Some viruses infect and kill cells. Researchers are working on ways to change these viruses so that they only target and kill cancer cells, leaving healthy cells alone. This sort of treatment uses the viruses to kill cancer cells directly rather than to deliver genes. So it is not cancer gene therapy in the true sense of the word. But doctors sometimes refer to it as gene therapy.

One example of this type of research uses the cold sore virus (herpes simplex virus). The changed virus is called Oncovex. It has been tested in early clinical trials for advanced melanoma, pancreatic cancer and head and neck cancers.

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Gene therapy | Cancer Research UK

06.10.14 11:38 Dow Jones Newswires

DJ Therapy decision in breast cancer: EndoPredict(R) predicts benefit from chemotherapy - Independent research group confirms chemo benefit for high-risk patients

(DGAP-Media / 06.10.2014 / 11:37)

Patients for whom EndoPredict(R) predicts a high risk of relapse have much greater benefit from chemotherapy than low-risk patients. This is the result published by an independent research group after investigating gene expression profiles and histological data of 553 patients. All patients were suffering from hormone receptor-positive and HER2/neu-negative breast cancer.

The authors conclude that a low risk EndoPredict result is a convincing reason not to give chemotherapy to a breast cancer patient. On the other hand, a high risk result is an indicator that the patient will benefit from chemotherapy.

"EndoPredict is the first test of its kind for which exclusively hormone receptor-positive and HER2/neu-negative tumours were already used during its development", says Dr. Christoph Petry, CEO of the developer company Sividon Diagnostics. "It is therefore the first gene expression test really tailor-made for those breast cancer patients who need it. We are pleased that independent scientists have now confirmed that patients for whom EndoPredict has predicted a high metastatic risk are in fact helped more by chemotherapy."

Low-risk patients according to EndoPredict have a very good prognosis, and hardly benefit from chemotherapy. "The treatment would be an unnecessary burden for the patients", explains Petry. "High-risk patients frequently have a genuine benefit from chemotherapy, as has been shown again in the recent study."

The study results are published in the Cancer Letters and can be downloadad at http://www.cancerletters.info/article/S0304-3835(14)00513-8/abstract. Franois Bertucci, Pascal Finetti, Patrice Viens, Daniel Birnbaum, EndoPredict Predicts for the response to neoadjuvant chemotherapy in ER-positive, HER2-negative breast cancer, Cancer Letters (2014), http://dx.doi.org/doi: 10.1016/j.canlet.2014.09.014

Sividon Diagnostics GmbH was founded in July 2010 as a management buyout from Siemens Healthcare Diagnostics Products in Cologne, Germany. The company aims to sustainably increase the quality of therapy-accompanying diagnostics in oncology. EndoPredict(R), the first diagnostic test from Sividon, has been available since 2011. The breast cancer prognostic test helps in deciding for which patient a chemotherapy is indicated Further information is available at http://www.sividon.com or http://www.endopredict.com.

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DJ Therapy decision in breast cancer: EndoPredict(R) predicts benefit from chemotherapy - Independent research group ...

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25-Sep-2014

Contact: Leslie Ridgeway lridgewa@usc.edu 323-442-2823 University of Southern California - Health Sciences

LOS ANGELES Keck Medicine of USC scientists have discovered new clues about a drug instrumental in treating a certain blood cancer that may provide important targets for researchers searching for cures.

The team investigated whether demethylation of gene bodies induced by the drug 5-Aza-CdR (decitabine), which is used to treat pre-leukemia, could alter gene expression and possibly be a therapeutic target in cancer.

"When we put the drug in cancer cells, we found it not only reactivated some tumor suppressor genes, but it down-regulated the overexpressed oncogene (cancer gene)," said Gangning Liang, Ph.D., associate professor of research, Keck School of Medicine of USC Department of Urology, who is corresponding author on the research. "Overexpression is what turns cancer 'on.' The mechanism by which the drug accomplishes this dual action is by removing DNA methylation in the gene body, which we didn't expect."

DNA methylation is an epigenetic signaling tool used by cells use to turn genes off. DNA methylation is an important component in many cellular processes, including embryonic development. Mistakes in methylation are linked to several human diseases, including cancer.

The research builds upon past research by Peter Jones, Ph.D., D.Sc., former director of the USC Norris Comprehensive Cancer Center, Distinguished Professor of Urology and Biochemistry & Molecular Biology, and now director of research at the Van Andel Institute.

"The beginnings of epigenetic therapy, which is now the standard of care for myelodysplastic syndrome, can be traced back to the discovery of the DNA demethylating effects of 5-Azacytidine at Children's Hospital Los Angeles in 1980," Jones said. "Since that time we have always assumed that the drugs act by switching genes on, thus reapplying the 'brakes' to cancer cells. In this paper we show that they may also work by turning down the levels of genes, which have become overexpressed in cancer. In other words, they may also decrease the 'gasoline' and this two pronged mechanism, which was entirely unexpected, may help explain why patients respond to epigenetic therapy."

The research, "Gene body methylation can alter gene expression and is a therapeutic target in cancer," was published online Sept. 25, 2014 in Cancer Cell.

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USC researchers discover dual purpose of cancer drug in regulating expression of genes