Archive for the ‘Gene Therapy Research’ Category

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Battle Purple Tomato: Genetically Engineered vs. Non-GMO

In5D Radio - Jordan Maxwell - Do Your Homework! Episode 16 | In5D.com
Gregg Prescott and Kendra Gilbert interviewed our returning guest, Jordan Maxwell, on In5D Radio as we covered a wide range of topics including astrotheology...

By: in5d

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In5D Radio - Jordan Maxwell - Do Your Homework! Episode 16 | In5D.com - Video

Using a novel form of immune-genetic therapy, researchers from Yale School of Medicine and the Jagiellonian University College of Medicine in Poland have successfully inhibited a strong immune allergic inflammatory response in the skin of mice. The results suggest the technique could be used to combat a variety of diseases.

We use an antibody coating we chose to deliver therapeutic genetic material we selected to target cells, said Dr. Philip Askenase, professor of medicine and senior author of the study published July8 in the Journal of Allergy and Clinical Immunology.

The delivery system consists of naturally occurring nanoparticles called exosomes that are about one thousandth the size of donor cells that release them. These tiny vesicles were once thought to contain only unneeded cellular debris. However, in the last decade, scientists have shown that there are billions of exosomes in the circulation and that they carry genetic instructions in the form of micro-RNAs (miRNA) to regulate the functions of nearby and distant cells.

Askenase and colleagues found that exosomes could be coated with an antibody of their choosing. These nanovesicles were able to deliver therapeutic miRNA to specific cells targeted by the antibody. In the current study, the coated exosomes delivered their miRNA cargo to immune system cells, inhibiting an active allergic disease response in the skin of mice.

These natural nanoparticles are present throughout the body, said Dr. Krzysztof Bryniarski of Jagiellonian University and lead author of the paper. They seem to be a superior delivery system compared to artificial nanoparticles currently in use, which often are eliminated from the body because they are sensed as artificial.

In theory, the researchers said, the natural nanoparticles coated with chosen specific antibodies and loaded with selected miRNAs could be used to specifically target and then genetically alter crucial cells involved in allergic conditions such as asthma, autoimmune responses, and potentially even cancers and neurological diseases.

The research was funded by the National Institute of Allergy and Infectious Diseases at the NIH (AI-00714 and 076366), and the Polish Ministry of Science and Higher Education (N401 092 31/2176 and K/ZDS/001429).

(Image via Shutterstock)

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Nature’s own nanoparticles harnessed to target disease

Awareness of genetics, ovarian cancer link could be lifesaver
Angelina Jolie #39;s double mastectomy made genetics and breast cancer a hot topic. But women also need to focus on the links between genetics and the "below-the-belt cancers."

By: wbal

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Awareness of genetics, ovarian cancer link could be lifesaver - Video

Newswise BETHESDA, MD July 9, 2013 Six graduate students and one undergraduate were named as recipients of Genetics Society of America (GSA) poster awards at the 19th International C. elegans Meeting, held June 2630 on the campus of the University of California, Los Angeles. More than 1,750 scientists attended the worlds largest gathering of those conducting research using the nematode worm, Caenorhabditis elegans, a model organism that lends itself to easy investigation where findings can easily be translated to humans.

Recipients were selected from almost 400 eligible posters presented at the meeting. The 93 faculty who judged the poster presenters had quite a challenge to select the best ones, because there were so many excellent posters. They did a great job judging and selecting the finalists, said Tina L. Gumienny, PhD, co-chair of the poster competition. The caliber of science at this year's poster session was amazing and bodes well for the future of C. elegans research, added Erin J. Cram, PhD, poster co-chair.

One winner was selected in each topic area:

Cell Biology Tisha E. Bohr University of California, Santa Cruz, CA Spindle assembly checkpoint proteins regulate and monitor meiotic synapsis in C. elegans

Development and Evolution Tulsi Patel Columbia University Medical Center, New York, NY Cell Fate Restriction and Reprogramming in C. elegans

Gene Regulation and Genomics Ashlyn D. Ritter University of Massachusetts Medical School, Worcester, MA Complex expression dynamics and robustness in C. elegans insulin networks

Methods and Technology Valeriya Laskova Samuel Lunenfeld Research Institute, Toronto, ON, Canada Mapping the entire connectome of C. elegans L1 larvae

Neurobiology Julie E. Grimm Technion Institute of Technology, Haifa, Israel How to Fix a Broken Neuron

Physiology Kurt J. Warnhoff Washington University, St. Louis, MO natc-1 mediates stress resistance and dauer formation as a downstream effector of the insulin/IGF-1 signaling pathway

Undergraduate Michael James Hoy College of the Holy Cross, Worcester, MA The C. elegans Insulin Signaling Response to Glucose Stress Requires Unique Regulators

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Seven Receive Genetics Society of America Poster Awards at Worm Meeting

Home Mail News Sports Finance Weather Games Groups Answers Flickr More omg! Shine Movies Music TV Health Shopping Travel Autos Homes Search News Search Web Sign In Mail Help Account Info Help Suggestions Yahoo! Home Video Photos GMA Year in Review LiveRoom Odd Comics Travel Opinion Trending Now Who Knew? Weather The Upbeat U.S. U.S. Video GMA Education Religion Crimes and Trials The Lookout Local Contributor Network Year In Review World World Video Middle East Europe Latin America Africa Asia Canada Australia/Antarctica Business Video Exclusives Today's Markets Stocks Personal Finance Marketplace Entertainment Video Clinton Concert Celebrity TV Movies Music Fashion Books Arts Theater Dear Abby Comics Odd News Sports Video NFL MLB NBA NCAAF NCAAB Soccer Cycling NHL Tennis Golf Boxing Motor Sports MMA Olympics Tech Gadgets Wireless Apple Social Media Security Open Source Gaming Apps This Could Be Big Upgrade Your Life Politics Remake America The Issues Women and Politics Press Releases Video Science Science Video Weather News Space / Astronomy Pets Dinosaurs / Fossils Biotech Energy Green Health Video Weight Loss Cancer Sexual Health Medications/Drugs Parenting/Kids Seniors/Aging Diseases/Conditions Blogs The Lookout The Sideshow Around the World Katie's Take Power Players This Could Be Big Newsmakers Trending Now Just Explain It The Upbeat Local Popular Search Keyword News Search Featured Videos Photos Just Explain It Katie's Take Weather The Upbeat Newsmakers

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Myriad sues competitor over cancer gene test

Home Mail News Sports Finance Weather Games Groups Answers Flickr More omg! Shine Movies Music TV Health Shopping Travel Autos Homes Search News Search Web Sign In Mail Help Account Info Help Suggestions Yahoo! Home Video Photos GMA Year in Review LiveRoom Odd Comics Travel Opinion Trending Now Who Knew? Weather The Upbeat U.S. U.S. Video GMA Education Religion Crimes and Trials The Lookout Local Contributor Network Year In Review World World Video Middle East Europe Latin America Africa Asia Canada Australia/Antarctica Business Video Exclusives Today's Markets Stocks Personal Finance Marketplace Entertainment Video Clinton Concert Celebrity TV Movies Music Fashion Books Arts Theater Dear Abby Comics Odd News Sports Video NFL MLB NBA NCAAF NCAAB Soccer Cycling NHL Tennis Golf Boxing Motor Sports MMA Olympics Tech Gadgets Wireless Apple Social Media Security Open Source Gaming Apps This Could Be Big Upgrade Your Life Politics Remake America The Issues Women and Politics Press Releases Video Science Science Video Weather News Space / Astronomy Pets Dinosaurs / Fossils Biotech Energy Green Health Video Weight Loss Cancer Sexual Health Medications/Drugs Parenting/Kids Seniors/Aging Diseases/Conditions Blogs The Lookout The Sideshow Around the World Katie's Take Power Players This Could Be Big Newsmakers Trending Now Just Explain It The Upbeat Local Popular Search Keyword News Search Featured Videos Photos Just Explain It Katie's Take Weather The Upbeat Newsmakers

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Myriad sues competitor overs cancer gene test

ALISO VIEJO, Calif.--(BUSINESS WIRE)--

Ambry Genetics announced today that it intends to vigorously defend itself against the patent infringement suit filed yesterday in the United States District Court for the District of Utah by Myriad Genetics, the University of Utah Research Foundation, the Trustees of the University of Pennsylvania, HSC Research and Development LP, and Endorecherche, Inc. The complaint alleges that Ambry Genetics infringes on certain Myriad BRCA1 and BRCA2 patents by offering diagnostic BRCA1 and BRCA2 testing to women.

Ambry Genetics began offering BRCA1 and BRCA2 diagnostic testing following the United States Supreme Courts landmark June 13, 2013 decision in AMP et al v. Myriad Genetics, et al. That lawsuit was brought by a coalition of patients, physicians and health care groups to challenge Myriads patents directed at BRCA1 and BRCA2 genes. As alleged in that lawsuit, Myriad maintained a monopoly over diagnostic testing of BRCA1 and BRCA2 genes under the Myriad patents and threatened legal actions against entities that wished to provide BRCA1 and BRCA2 gene testing to women. Following a multi-year legal battle, the unanimous Supreme Court decision held that genomic sequences, whether isolated or not, may not be patented and declared invalid the Myriad BRCA1 and BRCA2 patent claims challenged in that suit.

Ambry Genetics supports the Supreme Courts decision and will vigorously defend its position, said Charles Dunlop, Chief Executive Officer of Ambry Genetics. We have had an overwhelming response from our clients seeking an alternative laboratory to perform BRCA testing and Ambry is fully committed to supporting our clients and patients moving forward.

Ambry Genetics is the leader in hereditary cancer diagnostics. With the addition of BRCA1 and BRCA2 testing to its menu, Ambry Genetics now offers the most comprehensive germline cancer-testing menu in the industry, providing patients with the most accurate diagnosis currently available. Menu highlights include: BRCAplus, BreastNext, OvaNext, ColoNext, CancerNext and coming soon multi-gene NGS panels aimed to analyze genes implicated in hereditary renal, pancreatic and PGL-PCC cancers.

About Ambry Genetics

Ambry Genetics is a College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified commercial clinical laboratory with headquarters in Aliso Viejo, Orange County, California. Since its founding in 1999, it has become a leader in providing genetic services focused on clinical diagnostics and genomic services, particularly in sequencing and array services. Ambry has established a reputation for unparalleled service and for over a decade has been at the forefront of applying new technologies to the clinical molecular diagnostics market and to the advancement of disease research. To learn more about testing and services available through Ambry Genetics, visit http://www.ambrygen.com.

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Ambry Genetics Will Vigorously Defend Against BRCA1 and BRCA2 Gene Patent Infringement Suit

Scientists have created genetically-engineered mice with artificial human chromosomes in every cell of their bodies, as part of a series of studies showing that it may be possible to treat genetic diseases with a radically new form of gene therapy.

Click image above to enlarge graphic

In one of the unpublished studies, researchers made a human artificial chromosome in the laboratory from chemical building blocks rather than chipping away at an existing human chromosome, indicating the increasingly powerful technology behind the new field of synthetic biology.

The development comes as the Government announces today that it will invest tens of millions of pounds in synthetic biology research in Britain, including an international project to construct all the 16 individual chromosomes of the yeast fungus in order to produce the first synthetic organism with a complex genome.

A synthetic yeast with man-made chromosomes could eventually be used as a platform for making new kinds of biological materials, such as antibiotics or vaccines, while human artificial chromosomes could be used to introduce healthy copies of genes into the diseased organs or tissues of people with genetic illnesses, scientists said.

Researchers involved in the synthetic yeast project emphasised at a briefing in London earlier this week that there are no plans to build human chromosomes and create synthetic human cells in the same way as the artificial yeast project. A project to build human artificial chromosomes is unlikely to win ethical approval in the UK, they said.

However, researchers in the US and Japan are already well advanced in making mini human chromosomes called HACs (human artificial chromosomes), by either paring down an existing human chromosome or making them de novo in the lab from smaller chemical building blocks.

Natalay Kouprina of the US National Cancer Institute in Bethesda, Maryland, is part of the team that has successfully produced genetically engineered mice with an extra human artificial chromosome in their cells. It is the first time such an advanced form of a synthetic human chromosome made from scratch has been shown to work in an animal model, Dr Kouprina said.

The purpose of developing the human artificial chromosome project is to create a shuttle vector for gene delivery into human cells to study gene function in human cells, she told The Independent. Potentially it has applications for gene therapy, for correction of gene deficiency in humans. It is known that there are lots of hereditary diseases due to the mutation of certain genes.

Synthetic biology is loosely defined as designing new kinds of life-forms or making new genetic arrangements that do not exist in nature, which could provide practical benefits to society, notably in medicine, manufacturing or environmental monitoring.

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Exclusive: Scientists create human chromosome in breakthrough that could revolutionise medicine

DANVERS, Mass.--(BUSINESS WIRE)--

Cell Signaling Technology, Inc. (CST) announced today the issuance of a key US patent, 8,481,279, which relates to methods for inhibiting the progression of lung cancers that express the EML4-ALK fusion gene. The ALK fusion gene is an alteration or defect in a normal gene called anaplastic lymphoma kinase (ALK) that is thought to play a critical role in the growth of some non-small cell lung cancers (NSCLCs) and has been central to the development of several promising new, targeted cancer therapeutics.

CST has quietly pursued an aggressive research agenda over the last decade to understand the underlying mechanisms of disease progression, with an intense focus on cancer for the drug discovery industry and our customers, said Michael J. Comb, Ph.D., President and CEO of CST. We have assembled world-class scientists, detection systems and bioinformatics capabilities, investing over 20% of revenue to further research and development into the biochemical aberrations that underlie disease and the companion diagnostics and therapies to combat it.

CST has previously been issued patents (8,377,642, 8,288,102, 8,232,060, 8,168,383, and 7,700,339) covering methods for detecting the EML4-ALK fusion, which has been reported in a subset of patients with breast, colon and non-small cell lung cancer (NSCLC). CST now has a dominant IP position for EML4-ALK in NSCLC that spans from research through diagnosis and therapy.

CST was one of two groups that independently discovered that rare lung adenocarcinomas contain rearrangements of theALK gene that result in the pathologic expression of a fusion protein, most commonly EML4-ALK. CST is unique in the industry as its research agenda is completely self-funded. Privately held and scientist led, CST leverages what is widely regarded as the highest quality antibody portfolio commercially available, a byproduct of the companys research, to further its disease research agenda. The company regularly shares the results of its findings with the research community via high-impact journal publications (Cell, Nature Biotechnology et al.) and its ongoing, deep collaborations with academia and industry.

CST is proud to be part of one of the most elegant examples of translational research and personalized medicine that I have seen: a discovery going from a patient to the lab and back to patients, all within the space of two years, according to Mary Pinder-Schenck, M.D., in an article about the ALK story in Cancer GRACE.

Lung cancer is the worlds leading cause of cancer death, with more than 1.8 million new cases diagnosed each year, 85% of which are NSCLC. Approximately 3-5% of NSCLC patients have tumors positive for the ALK fusion gene. Patients are usually diagnosed with advanced disease and have a very low survival rate. Recently Pfizers Xalkori (crizotinib) was approved in the US for treatment that targets patients with the ALK fusion and several companies have additional therapeutics in various clinical trial stages.

About Cell Signaling Technology, Inc.

Founded by research scientists in 1999, Cell Signaling Technology (CST) is a private, family-owned company with over 400 employees worldwide. Active in the field of applied systems biology research, particularly as it relates to cancer, CST understands the importance of using antibodies with high levels of specificity and lot-to-lot consistency. Its why we produce all of our antibodies in house, and perform painstaking validations for multiple applications. And the same CST scientists who produce our antibodies also provide technical support for customers, helping them design experiments, troubleshoot, and achieve reliable results. We do this because thats what wed want if we were in the lab. Because, actually, we are.

For more information visit cellsignal.com.

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Cell Signaling Technology Awarded Patents Critical to Lung Cancer Therapy

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/d2p5hw/neuroprotection) has announced the addition of Jain PharmaBiotech's new report "Neuroprotection - Drugs, Markets and Companies" to their offering.

This updated report now contains even more company profiles than the previous edition!

This report describes the role of neuroprotection in acute disorders such as stroke and injuries of the nervous system as well as in chronic diseases such as neurodegenerative disorders because many of the underlying mechanisms of damage to neural tissues are similar in all these conditions and several products are used in more than one disorder. Over 500 products have been investigated for neuroprotective effects including those from the categories of free radical scavengers, anti-excitotoxic agents, apoptosis (programmed cell death) inhibitors, anti-inflammatory agents, neurotrophic factors, metal ion chelators, ion channel modulators and gene therapy. Some of the agents are old established pharmaceuticals whereas others are new biotechnology products.

Pathomechanisms of diseases are described with steps at which neuroprotective therapies are directed. Diseases covered include cerebrovascular disorders, traumatic brain injury, spinal cord injury, Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, multiple sclerosis, epilepsy and ischemic optic neuropathy as well as retinal degeneration. Although anesthetics such as propofol are neuroprotective as well, neuroprotection during surgery and anesthesia is discussed with the aim of preventing and treating complications that result in CNS damage.

The report contains profiles of 137 companies that have a neuroprotective product or products along with 121 collaborations. Some of the products in development at academic institutions that do not have a commercial sponsor are also included. Although an up-to-date search of the literature was performed and selected 1,000 references are included. Clinical trials of various neuroprotective agents are described and failures of trials are analyzed with suggestions for improving the selection of drugs and design of trials. The report is supplemented with 67 tables and 13 figures.

Market analysis of currently used products that have a neuroprotective effect are analyzed for the year 2012. Some of these products are approved for other indications but are known to have a neuroprotective effect. With the approval of new products and takeover of markets for obsolete symptomatic therapies, the neuroprotection market value will rise by the year 2017 when it will constitute a major and important component of the CNS market. Forecasts are made until 2022. By that time neuroprotection will be an established part of the neurological practice and measures will be available to achieve this effectively.

Key Topics Covered:

Part I: Drugs & Markets

Executive Summary

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Research and Markets: Neuroprotection - Drugs, Markets and Companies - 2013-2022

Public release date: 9-Jul-2013 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY -- Next-generation hydrogels can form synthetic scaffolds to support the formation of replacement tissues and organs in the emerging area of regenerative medicine. Embedding peptides into the hydrogels stimulates the growth of essential microvascular networks to ensure a good blood supply. Novel, cutting-edge technology in which hydrogels functionalized with laminin-derived peptides were transplanted in a mouse cornea and were shown to support cell growth and blood vessel formation is described in an article in BioResearch Open Access, a peer-reviewed open access journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResarch Open Access website.

Saniya Ali and coauthors from Rice University and Baylor College of Medicine, Houston, TX, and Duke University, Durham, NC, created a biodegradable hydrogel-based scaffold containing laminins. These peptides are key components of cells' extracellular matrix and play a critical role in the attachment, movement, and organization of endothelial cells, which form the lining of tubules such as blood vessels. Stimulating and controlling the formation and growth of these tubule-like, cell-lined structures is essential for ensuring sufficient blood supply to support large complex tissues or organs. The authors present their work and the results of animal studies in the article "Immobilization of Cell-Adhesive Laminin Peptides in Degradable PEGDA Hydrogels Influences Endothelial Cell Tubulogenesis."

"Enhancing vascularization in synthetic scaffolds is essential to support the formation of blood vessels in engineered tissues," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "The work in this study demonstrates that laminin-derived peptide sequences immobilized in synthetic scaffolds can be used to regulate the formation of microvasculature in tissue-engineered constructs."

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available on the BioResarch Open Access website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including, DNA and Cell Biology, Tissue Engineering, Stem Cells and Development, Human Gene Therapy, and AIDS Research and Human Retroviruses. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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Method to improve blood supply to engineered replacement tissues

Arcadia, CA (PRWEB) July 09, 2013

Retina Institute (RIC) now offers RetnaGene, a genetic test to evaluate the risk of a patient with early age-related macular degeneration (AMD) progressing to advanced choroidal neovascular disease within 2, 5, and 10 years. Efficient monitoring of this chronic disease greatly reduces the chances of partial or complete loss of vision in patients.

"RetnaGene testing represents the leading edge of clinically impactful genetic medicine in ophthalmology, stated RIC surgeon, Joshua Hedaya, MD. This technology will fundamentally improve our ability to treat and prevent vision loss from AMD. It is a very exciting time for doctors and patients alike."

The reported risk scores for developing CNV are based on four risk factors including genotype, phenotype, age and environmental factors such as smoking.

With the information provided by the RetnaGene test, RIC can customize an approach to monitoring and treatment for each individual patient based on their reported risk scores for disease progression. This provides a tremendous advantage when managing a condition such as AMD, in which loss of vision is often severe, unpredictable, and variably responsive to treatment.

With this test, RIC can safely reduce the screening burden for lower risk patients, many of whom may have difficulty complying with frequent office visits. This additional genetic data point allows for a more informed decision regarding initiation of treatment in patients with equivocal findings on standard diagnostic tests.

For further information about RetnaGene or to refer a patient to RIC for AMD screening, please contact RIC at 800-898-2020. Testing is available at all RIC locations, and is performed at no or minimal cost to patients.

About Retina Institute The Retina Institute was founded on a model of exceptional patient care and clinical excellence and is recognized as one of the nations leading practices specializing in vitreoretinal diseases. With 15 highly-trained retina surgeons in 24 centers, Retina Institute is committed to innovation, compassion and superior results, and works in close partnership with each other and with referring doctors to achieve the best possible outcome for every patient. For more information visit retina2020.com.

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Retina Institute of California Advances Genetic Diagnostics for Treatment of Age-related Macular Degeneration (AMD)

NEW YORK & LOS ANGELES--(BUSINESS WIRE)--

Scil is launching a service, the first of its kind, that allows individuals to benefit from 2012 Nobel Prize-winning medical research to store a backup of their adult selves literally freezing their healthy cells in time. Scils world-leading experts use cutting-edge techniques to take cells from a small sample of an individuals skin and reboot them at the "Day Zero state". These skin cells are processed into stem cells (iPS) that are stored long-term for potential future uses, which could repair damaged organs, rebuild tissue or fight disease, as personalised genetic-based medicine continues to develop.

Andr Choulika, CEO of Scil, said: Scil offers people the best possible chance in the future. People should be able to live young, no matter how old they grow. Scil gives them the opportunity to take advantage of the wave of regenerative medicine. Were offering the potential for people to use their cells for their cure as soon as regenerative medicine treatments become available.

iPS (induced pluripotent stem cells) are capable of developing into any cell type. They can be derived from adult cells at any time of life, but given the fast rate at which cell DNA degenerates (the human body accumulates about 1.8 million DNA mutations per second), Scils experts recommend taking this step sooner rather than later to ensure the healthiest possible cells are stored.

With the Scil service, the iPS cells are stored at -180C for as long as needed for potential future use as soon as regenerative medicine treatments become available. It is the first time ever that such a service has been made available to individuals commercially.

Regenerative medicine is the process of replacing or regenerating human cells, tissues and organs to restore or establish normal function or, by stimulating the body's own repair mechanisms, to heal previously irreparable tissues or organs.

The service is very simple and painless for the individual. A skin tissue sample is taken under local anaesthetic and by arrangement at your convenience. The skin cells are then rebooted into iPS cells by Scils world-leading biotechnology teams; and finally they are stored by Scil for a lifetime in cutting-edge laboratory conditions until they are needed for future medical applications as soon as they become available.

You can read more about Scil, the benefits of stem cell banking, and the leading role its parent company Cellectis plays in genetic research at http://www.sceil.com

About Scil

Scil, part of the Cellectis Group, delivers a service that allows individuals to freeze their adult stem cells and harness the future potential of regenerative medicine. Based on Nobel Prize-winning research, Scils world-leading experts use cutting-edge techniques to take cells from a small sample of an individuals skin and reset them at the Day Zero state. These skin cells are processed into induce pluripotent stem cells (iPS) that are stored long-term for potential future needs to repair damaged organs, rebuild tissue and fight disease as personalised genetic-based medicine continues to develop. For further information about Scil, please visit http://www.sceil.com

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Scéil Launches World’s First iPS Stem Cell Back-up Service

An in vitro fertilization (IVF) milestone has been announced by British researchers. For the first time, a baby was born using a new embryo screening technique combs through genetic data looking for risk for diseases and other abnormalities. The researchers say the technique, known as "next generation sequencing" will revolutionize embryo selection for families turning to IVF.

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"In the past few years, results from randomized clinical trials have suggested that most IVF patients would benefit from embryo chromosome screening, with some studies reporting a 50 percent boost in pregnancy rates. However, the costs of these genetic tests are relatively high, putting them beyond the reach of many patients," lead researcher Dr. Dagan Wells, a scientist at the NIHR Biomedical Research Centre at the University of Oxford in the U.K., said in a statement. "Next generation sequencing is a way which could make chromosome testing more widely available to a greater number of patients, improving access by cutting the costs."

Wells presented the case study of the first birth Monday at the European Society of Human Reproduction and Embryology in London.

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Next generation DNA-sequencing technology is used in other areas of medicine, such as in research of differences between normal cells and cancer cells, according to the Columbia Genome Center in New York City. For the process, DNA is broken into small fragments which are turned into strings of genetic code which are then sequenced in hundreds of millions parallel reactions.

This testing can also reveal information on the chances for inheriting genetic disorders, chromosome abnormalities and mitochondrial disease, mutations within a cell's nucleus that could lead to conditions including heart disease, motor disorders, diabetes, respiratory problems, seizures, and vision and hearing problems.

Wells said since the technology is already revolutionizing diagnostic medicine, if it's applied to embryo selection for IVF, it can provide "an unprecedented insight into the biology of embryos."

That's important, because only about 30 percent of embryos currently selected for transfer in IVF actually implant in the uterus. The reason for this high failure rate is unknown, according to the researchers, but they suspect hidden genetic mutations and abnormalities may be at play.

To ensure the new technique's accuracy, the researchers sequenced cells from 45 embryos that had previously been shown to be abnormal by a different testing technique. In a blind comparison of the two techniques, high accuracy was established.

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IVF first: Baby born with embryo selection from DNA screening

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/96mtm2/personalized) has announced the addition of Jain PharmaBiotech's new report "Personalized Medicine - Scientific and Commercial Aspects" to their offering.

This updated 2013 report now features even more company profiles and collaborations!

The aim of personalized medicine or individualized treatment is to match the right drug to the right patient and, in some cases, even to design the appropriate treatment for a patient according to his/her genotype. This report describes the latest concepts of development of personalized medicine based on pharmacogenomics, pharmacogenetics,pharmacoproteomics, and metabolomics. Basic technologies of molecular diagnostics play an important role, particularly those for single nucleotide polymorphism (SNP) genotyping. Diagnosis is integrated with therapy for selection of the treatment as well for monitoring the results. Biochip/microarray technologies are also important and finally bioinformatics is needed to analyze the immense amount of data generated by various technologies.

Pharmacogenetics, the study of influence of genetic factors on drug action and metabolism, is used for predicting adverse reactions of drugs. Several enzymes are involved in drug metabolism of which the most important ones are those belonging to the family of cytochrome P450. The knowledge of the effects of polymorphisms of genes for the enzymes is applied in drug discovery and development as well as in clinical use of drugs. Cost-effective methods for genotyping are being developed and it would be desirable to include this information in the patient's record for the guidance of the physician to individualize the treatment. Pharmacogenomics, a term that overlaps with pharmacogenetics but is distinct, deals with the application of genomics to drug discovery and development. It involves the mechanism of action of drugs on cells as revealed by gene expression patterns. Pharmacoproteomics is an important contribution to personalized medicine as it is a more functional representation of patient-to-patient variation than that provided by genotyping. A 'pharmacometabonomic' approach to personalizing drug treatment is also described.

Biological therapies such as those which use patient's own cells are considered to be personalized medicines. Vaccines are prepared from individual patient's tumor cells. Individualized therapeutic strategies using monoclonal bodies can be directed at specific genetic and immunologic targets. Ex vivo gene therapy involves the genetic modification of the patient's cells in vitro, prior to reimplantation of these cells in the patient's body.

Increase in efficacy and safety of treatment by individualizing it has benefits in financial terms. Information is presented to show that personalized medicine will be cost-effective in healthcare systems. For the pharmaceutical companies, segmentation of the market may not leave room for conventional blockbusters but smaller and exclusive markets for personalized medicines would be profitable. Marketing opportunities for such a system are described with market estimates from 2012-2022.

Profiles of 283 companies involved in developing technologies for personalized medicines, along with 504 collaborations are included in the part II of the report. Finally the bibliography contains over 650 selected publications cited in the report. The report is supplemented by 65 tables and 18 figures.

Key Topics Covered:

Part I: Scientific & Commercial Aspects

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Research and Markets: Personalized Medicine Report - Scientific and Commercial Aspects - 2013-2022

LIVINGSTON, NJ--(Marketwired - Jul 9, 2013) - Reprogenetics, one of the largest providers of preimplantation genetic diagnosis (PGD) services announced today that one of their researchers Dr. Dagan Wells of the NIHR Biomedical Research Centre at the University of Oxford, UK, Reprogenetics UK and Reprogenetics LLC, has been acknowledged by the European Society of Human Reproduction and Embryology (ESHRE), in London, UK for his work in the area of Next Generation Sequencing (NGS). His research led to the first ever successful birth of a baby through this unique science revealing extensive genetic information derived from human embryos. This impressive science can significantly impact the process for, and success rate of, embryo selection for in vitro fertilization in the future.

"Reprogenetics congratulates Dr. Wells on his outstanding accomplishment to genetic analysis and achieving this monumental goal in the reproductive space," said Dr. Jacques Cohen, Reprogenetics Scientific Director. "We are proud of the quality team we have established including Dr. Wells who all play an integral role in us reaching the goal of predicting viability of embryos with our NGS technique, and what it can ultimately mean for patients."

About the studyTo test the accuracy and predictability of NGS, the international study conducted in the UK in collaboration with IVF clinics in the USA used a technique that has never before been applied in the screening of human embryos. The study looked at multiple cells from cell lines known to have chromosomal abnormalities, genetic defects (cystic fibrosis), or other mutations (n=30). Further, in a blinded portion of the study, investigators used the new NGS technique to re-examine 45 embryos in which abnormalities had previously been identified by other methods. Once the accuracy of the new technique was demonstrated, it was applied clinically, with cells sampled from seven five-day old embryos produced by two couples undergoing IVF. The mothers were 35 and 39 years old, and one had a history of miscarriage (total 82 embryos).

The outcome validated the NGS technique, with 100% of samples yielding results (82/82). NGS accurately identified abnormalities in those cells that had been previously identified as having them. And, in the clinical setting, NGS revealed 3/5 viable embryos from the first couple and 2/2 from the second. Single embryo transfers based on these results led to healthy pregnancies for both couples. The first pregnancy ended with the delivery of a healthy boy, Connor Levy, last month and the other is ongoing.

Dr. Wells noted that the cost of NGS, which enables simultaneous testing for a variety of abnormalities, is significantly lower than that of existing screening methods, suggesting that this technique may ultimately bring genetic analysis within reach for a much larger number of patients.

"We greatly admire the vision, drive and perseverance demonstrated by Dr. Wells and his team. This study validates an important new approach to genetic analysis, and we look forward to working with them and pursuing this research further," said Dr. Jamie Grifo, of NYU and a participant in the study.

About Embryo ScreeningAccording to ESHRE, on average, only about 30% of embryos selected for transfer actually result in a viable pregnancy. Although the precise reason is unknown, researchers believe that unidentified genetic or chromosomal abnormalities are in part responsible for this low success rate. Genetic screening methods introduced in recent years are helpful, but still have multiple drawbacks when used in a clinical setting.

About PGS In PGS, embryos created through in-vitro fertilization are tested for chromosomal abnormalities prior to replacement in a woman's uterus. This process allows the reproductive endocrinologist to select only chromosomally healthy embryos for replacement with the goal of increasing the chance of successful implantation, reducing spontaneous abortion, reducing the chance for a fetus to have a chromosomal abnormality and improving delivery rates for assisted reproduction.

Chromosome abnormalities are the primary cause of miscarriage and failure to implant. This percentage increases with maternal age, and studies have shown that 82% of embryos from women 40 years and older will be chromosomally abnormal. However, once normal (euploid) embryos are selected, they implant equally well at any age up to 42 years of age.

About ReprogeneticsReprogenetics is a genetics laboratory specialized in Reproductive Medicine and a pioneer and Preimplantation Genetic Diagnosis (PGD). Dr. Santiago Munn and Dr. Jacques Cohen founded Reprogenetics in the year 2000 after extensive experience in PGD and IVF, with more than 24,000 PGD procedures performed so far, and with branches in 7 countries. Reprogenetics offers a comprehensive and personalized service to its referring IVF centers and their patients. Genetic counselors are intricately involved in the process and interact routinely with the patients pursuing all PGD tests.

Originally posted here:
First Ever Baby Born Through Genetic Derived Human Embryos

LOS ANGELES--(BUSINESS WIRE)--

The HIV gene medicines company Calimmune announced today that the first patient has begun treatment in a Phase I/II clinical trial designed to determine whether a pioneering genetic medicine approach can help to protect individuals infected with HIV from the effects of the virus. The study, Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection, utilizes a gene medicine called Cal-1, developed in the lab of Nobel Laureate Dr. David Baltimore and by Calimmune.

In the study, 12 HIV-positive participants will be infused with their own T cells and stem cells (hematopoietic stem cells, HSC), which have been modified to block the HIV receptor CCR5, and to prevent HIV fusion. The procedure is designed to prevent the virus from entering and damaging protected cells. The dual approach used in the study is designed to reduce the possibility that HIV can develop resistance to the procedure.

The goal of the study is to assess the safety, feasibility and tolerability of Cal-1 in HIV-infected individuals who have previously been on highly active antiretroviral therapy (HAART) but are not currently taking any antiretroviral agent. In addition to routine clinical and laboratory assessments to monitor general health and HIV infection, the study will monitor the presence of Cal-1 protected cells in various cell types in the blood and lymphoid tissue. Other analyses will monitor the safety of Cal-1. The first patient was treated in the study in late June. Data from this study are expected in 2015.

All participants in the studys three arms will receive the Cal-1 gene transfer. Participants in two of the three study arms will also receive different doses of a preconditioning drug known as busulfan, which may make the therapy more effective.

This study is an early but important step in an emerging area of scientific exploration, representing the culmination of more than a decade of research and development, said Calimmune Chief Executive Officer Louis Breton. We are optimistic that what we learn from this study may bring us closer to the day when a one-time treatment could provide an alternative to a lifetime of antiretroviral therapy.

The study has been partially funded by the California Institute for Regenerative Medicine (CIRM). The study will take place at clinical trial sites in Los Angeles and San Francisco, Calif., under the direction of Principal Investigators Ron Mitsayasu, M.D., of UCLA and Jacob P. Lalezari, M.D., of Quest Clinical Research.

For more information, visit http://www.clinicaltrials.gov.

About Calimmune

Calimmune is a clinical-stage HIV gene medicines company focused on developing innovative cell-based therapies for HIV. The companys stem cell technology was discovered in the labs of Nobel Laureate Dr. David Baltimore (Caltech) and Dr. Irvin Chen (UCLA AIDS Institute). Calimmune is also developing a rich product candidate pipeline to address the needs of different types of individuals at different states of HIV infection and with different levels of treatment experience.

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Calimmune Initiates HIV Stem Cell Study at Two California Research Sites

Shotgun Flash - Apex Genetics on Esamir
A good run on my shotgun flash -- http://www.twitch.tv/lepalose/c/2512624 utm_campaign=archive_export utm_source=lepalose utm_medium=youtube.

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Shotgun Flash - Apex Genetics on Esamir - Video

ATV Motocross @ Apex Genetics
Some shotgun flashing among the buildings at Apex Genetics -- http://www.twitch.tv/lepalose/c/2512633 utm_campaign=archive_export utm_source=lepalose utm_medium=youtube.

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Shaman Genetics

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AMSTERDAM, THE NETHERLANDS--(Marketwired - Jul 9, 2013) - uniQure B.V., a leader in human gene therapy, today announced it has signed collaboration agreements with Chiesi Famaceutici SpA for the commercialization of Glybera, the first gene therapy to receive regulatory approval in Europe, as well as the co-development of a gene therapy for hemophilia B. In connection with this transaction, uniQure has received EUR 17 million (USD 21.8 million) in collaboration financing and EUR 14 million (USD 18 million) in equity financing from Chiesi, and has converted into equity the previously announced EUR 14.1 million (USD 18.1 million) investment led by Coller Capital (London, UK) with participation by existing investors.

uniQure's agreement with Chiesi, an international company headquartered in Parma, Italy, gives Chiesi exclusive rights to commercialize Glybera, the first gene therapy product approved in the European Union for the treatment of the orphan disease lipoprotein lipase deficiency (LPLD) for which there is currently no treatment, as well as for uniQure's pipeline product for hemophilia B, in Europe and selected other countries (Brazil, Mexico, Pakistan, Turkey, Russia, and the CIS countries, plus China for Glybera only). Commercial rights for the US, Japan, and parts of Latin America and Asia, and Australasia remain with uniQure. In exchange, uniQure stands to receive net royalties that range from 20 to 30 percent over time on sales of both products. Furthermore, Chiesi will fund half of the remaining development costs for uniQure's hemophilia B program, as well as take an equity stake in uniQure.

The investment by Chiesi will also result in the conversion into new uniQure shares of the EUR 14.1 million (USD 18.1 million) in convertible debt the Company recently raised from Coller Capital, a leading global private equity investor, and existing investors Forbion Capital Partners, Gilde Healthcare Partners, Lupus Alpha, Grupo Netco and others.

"The agreement with Chiesi is a key component of our strategy to rapidly develop and commercialize multiple gene therapy based treatments as well as being a validation of our technology platform," said Jrn Aldag, CEO of uniQure. "With its focus on innovative therapeutics, Chiesi is a strong partner for the commercialization of Glybera in Europe. The investment from Coller Capital, supplemented by our existing investors, allows us to continue apace with the development of what we believe is the richest and most advanced gene therapy pipeline in the industry. In the next 12-18 months, we anticipate clarifying the path toward an FDA filing for Glybera in the US, reporting results from a Phase I/II study in acute intermittent porphyria, and starting at least two Phase I/II studies for additional pipeline programs."

About uniQure uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. http://www.uniqure.com.

About Chiesi Farmaceutici Founded in 1935 in Parma, Italy, Chiesi Farmaceutici currently has 25 affiliates worldwide and markets its therapeutics in over 60 countries. Chiesi's manufacturing plants in Parma, Blois (France) and Santana de Parnaiba (Brazil), and R&D centers in Parma, Paris, Rockville (USA) and Chippenham (UK) integrate their efforts to advance the Group's pre-clinical, clinical and registration programs. At the end of 2012, the Chiesi Group's total staff stood at over 3,800 people, more than 350 of whom are dedicated to R&D. The main areas of activity are in respiratory therapeutics and specialist medicine areas.

DisclaimerThis press release contains forward-looking statements based on uniQure's current expectations. These forward-looking statements include statements regarding the commercialization of Glybera, regulatory approval matters and the development of additional gene therapies. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties regarding further regulatory requirements, the success of further clinical trials, and competitive pressures. uniQure assumes no responsibility to update such forward-looking statements.

Press release (PDF): http://hugin.info/157414/R/1715194/569858.pdf

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uniQure Signs EU Commercialization Agreement With Chiesi Farmaceutici for First Approved Gene Therapy Treatment, and ...

by Buena Bernal Posted on 07/06/2013 4:28 PM |Updated 07/08/2013 2:16 PM

STEM CELLS. Health Secretary Enrique Ona says stem cell therapy is a procedure still under clinical evaluation and study. Photo by Rappler/Naoki Mengua

MANILA, Philippines (UPDATED) Amid increasing interest in and the risks of stem cell treatment in the country, the health secretary said doctors are required to disclose to their potential patients that the procedure is still "investigative" its potential is still being explored, and there's no definite word that it can heal diseases.

This is in line with the ethical standards of medical practice that seek to empower patients with enough information prior to consenting to the treatment.

The patient must exactly know that he is part of an investigative process. And that is what we require as far as the Department of Health is concerned right now, Department of Health (DOH) Secretary Enrique Ona said in an interview with Rappler on Friday, July 5.

Stem cell therapy or regenerative medicine is the use of the bodys repair cells as a substitute to old cells that may cause debilitating diseases.

(READ: 6 things you need to know about stem cell therapy)

The treatment has gained controversy of late, after 3 unnamed high-profile politicians allegedly died due to botched procedures performed abroad. The deaths are currently being investigated by the Philippine Medical Association (PMA).

Not to stifle innovation

So why is the DOH encouraging the development of the science despite the treatment being under clinical evaluation and study?

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DOH: Stem cell therapy not yet proven to be curative

Scientists said Monday they had used a new-generation gene sequencing technique to select a viable embryo for in-vitro fertilisation (IVF) that yielded a healthy baby boy.

IVF, the process whereby a human egg is fertilised with sperm in the laboratory, is a hit-and-miss affair, with only about 30 percent of fertilised embryos resulting in pregnancy after implantation.

The reason for the high failure rate is not clear but genetic defects are the prime suspects, according to the authors of the paper presented Monday at a meeting in London of the European Society of Human Reproduction and Embryology (ESHRE).

The new method, known as next generation sequencing or NGS, uses updated technology to sequence an entire genome -- revealing inherited genetic disorders, chromosome abnormalities and mutations.

Study author Dagan Wells of the University of Oxford's NIHR Biomedical Research Centre said the new technology was "inherently cheaper" and yielded more genetic data than older methods.

It provides millions of fragments of DNA from a single cell which are then sequenced by a computer.

The method has started being used in genetic research and diagnostics, but not yet in embryo screening, according to Wells.

"Many of the embryos produced during infertility treatments have no chance of becoming a baby because they carry lethal genetic abnormalities," he said in a statement.

"Next generation sequencing improves our ability to detect these abnormalities and helps us identify the embryos with the best chances of producing a viable pregnancy."

Current methods of detecting embryonic gene deficiencies add over 2,000 (2,300 euros, $3,000) to a single IVF attempt, said Wells.

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New gene sequencing yields healthy baby

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/8hsggg/analysis_of) has announced the addition of the "Analysis of Global Biochips Industry, 2012-2018" report to their offering.

Global biochips market is forecasted to reach US$11.4 billion by 2018 with a CAGR of 18.6% during 2012-2018; the massive CAGR is primarily supported by Asia-Pacific followed by the European Union. Among the major industry segments, biochips instruments are expected to exert the highest support to the industry with a CAGR of 20% globally.

The evolution of biochips has opened new vistas in the biological systems. In addition, all other sciences are integrated which cumulatively contribute for a big future of biochip industry. The broad life science' division has been witnessing a rapid growth and technological improvements varying from sector to sector since the past 3-5 years. Accelerating growth rate exhibited by the biochips industry, even during the recession years, confirms the positive growth prospects going ahead. The field of drug discovery and development research gets more glamorous with the diagnostics and treatment at cellular level. DNA biochips and lab-on-chips have created revolution enabling the target validation. Genome scan is very soon going to become an ultimate weapon for diagnosis. In no time all the information related to genes will be sequenced, annotated and completed along with the list of diseases which are susceptible. Day to day the researchers are also making an effort to develop medication to control the various diseases, by using biochip technology. As the applications of biochips are wide both in the research and clinical use, a wide potential market is expected. The emergence of biochip technology can be attributed to a decade which has gradually developed into maturity. This industry is expected to bring rapid and significant changes in the life sciences and medicine. Microarray technologies have a great potential and is widely used in DNA and protein analysis.

The report reviews the latest biochips market trends with a perceptive attempt to disclose the near-future growth prospects. An in-depth analysis on a geographic basis provides strategic business intelligence for life science sector investments. The study reveals profitable investment strategies for pharmaceutical manufacturers, biotechnology companies, laboratories, Contract Research Organizations (CROs), government organizations and many more in preferred locations.

The report primarily focuses on:

- Emerging Market Trends

- Advancements in the Technological Space

- Market Demand Of The Segments (By-Region)

- Key Growth Areas and Market Size

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Research and Markets: Analysis of Global Biochips Industry, 2012-2018