Archive for the ‘Gene Therapy Research’ Category

Adult Stem Cell Therapy for COPD: Stage-4 patient
Stage 4 COPD patient Ron Delkie arrived at the Regenerative Medicine Institute at Hospital Angeles Tijuana by ambulance, with very little hope of long term s...

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Goodie Mob Performs "Cell Therapy" at House of Blues For "Age Against The Machine"
Here #39;s more of that Good ole Goodie Mob, gettin down with yet another classic of that "Soul Food" Album. "Cell Therapy" was actually the first official singl...

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Goodie Mob Performs "Cell Therapy" at House of Blues For "Age Against The Machine" - Video

Stem Cell Therapy Day 0 -1 🙂
Montana starting to build new hips.

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Stem Cell Therapy Day 0 -1 🙂 - Video

DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/d8mv33/rna_mirna_rnai) has announced the addition of the "RNA (miRNA, RNAi & siRNA) Therapy in Oncology Drug Pipeline Update 2013" report to their offering.

RNA (miRNA, RNAi & siRNA) Therapy in Oncology Drug Pipeline Update 2013

Potentially any disease-causing gene, cell type or tissue can be targeted with miRNA, RNAi or siRNA, including those not 'druggable' with small molecules or protein-based therapies.

There are today 53 companies plus partners developing 79 RNA (miRNA, RNAi & siRNA) drugs in 110 developmental projects in cancer. In addition, the accumulated number of ceased drugs over the last years amount to another 25 drugs. Rna (Mirna, Rnai & Sirna) Therapy In Oncology Drug Pipeline Update lists all drugs and gives you a progress analysis on each one of them. Identified drugs are linked to 56 different targets. All included targets have been cross-referenced for the presence of mutations associated with human cancer. To date 41 out of the 41 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 drugs targets are further categorized on in the software application by 24 classifications of molecular function and with pathway referrals to BioCarta, KEGG, NCI-Nature and NetPath.

How May Drug Pipeline Update Be of Use?

- Show investors/board/management that you are right on top of drug development progress in your therapeutic area.

- Find competitors, collaborations partners, M&A candidates etc.

- Jump start competitive drug intelligence operations

- Excellent starting point for world wide benchmarking

- Compare portfolio and therapy focus with your peers

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Research and Markets: RNA (miRNA, RNAi & siRNA) Therapy in Oncology Drug Pipeline Update 2013 Out Now for Review

Ive been selecting columns for a book of my favorites, written over the past 22 years for the editorial page of the Kennebec Journal and Waterville Morning Sentinel. I just came across this column about Gene Letourneau, published shortly after his funeral in 1998. I still miss Gene. How about you?

Gene Letourneaus last cast

The warm breeze wafting through Sacred Heart Church in Waterville last Thursday surely must have carried Gene Letourneaus spirit to the great hunting ground beyond our imagination.

As expected, Letourneaus funeral featured stories about fly fishing and hunting dogs, but we also heard about his musical talent, faith in God, and devotion to his family, especially his lovely wife Lou who died in 1996. The light went out for Gene then, and he began his own hike to join Lou. Hes there now, sipping that Heavenly cold clear spring water.

We are bereft, for the irreplaceable outdoor writer has left behind his canoe without so much as a wake we can ride for even a short way. His paddle dipped too quietly, I guess, for his bosses to realize the impact he was having on the people of Maine, his devoted readers and correspondents.

Genes daily outdoors column, titled Sportsmen Say, that started my day as a kid, is gone, found in none of todays daily newspapers, still wondering why theyre losing subscribers.

I was privileged to appear in a video of reverence and praise presented to Gene at his spectacular retirement party at the Augusta Civic Center a few years ago. My remarks focused on the real magic of his newspaper column, in the section called Chips from the blazed trail.

It was here that Gene presented first-hand reports he received in letters and phone calls from sportsmen throughout the state, accounts of their exciting adventures, latest fishing successes, hunting prowess or simple observations of wild birds and animals.

I always felt this was the secret of his column: he let the voices of his readers and fans be heard. Sportsmen Say. Indeed.

Gene connected with people, whether or not they shared his passion for hunting and fishing, because he presented Maines outdoor traditions in their own words, kept them informed, shared his extensive knowledge (while keeping some secret ponds to himself to protect those precious resources), always in simple, clear prose.

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Gene Letourneau’s Last Cast

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Time to stop fighting GMO labeling?

Advanced Genetics and Team Green Athlete Josee Gallant
Contest Prep, Rep #39;n Advanced Genetics and Team Green I do not own the rights to the music heard in this video. It belongs to the respective copyright owners....

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Advanced Genetics and Team Green Athlete Josee Gallant - Video

The Sims 3: Perfect Genetics Challenge: Episode 4
Baljeet.

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The Sims 3: Perfect Genetics Challenge: Episode 4 - Video

The Sims 3: Perfect Genetics Challenge: Episode 5
Im lazy as hell.

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The Sims 3: Perfect Genetics Challenge: Episode 5 - Video

Pitch Nim Genetics SSIS 2012
Presentación Nim Genetics durante la edición 2012 de Spain Startup Investor Summit.

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Pitch Nim Genetics SSIS 2012 - Video

RACING PIGEON TIPS 2 ....... TRICKS OF THE TRADE
AFTER MY FIRST VIDEO OF THE SECRECTS CHAMPIONS KNOW ,THERE WAS A VERY BIG POSITIVE RESPONSE AND I DID A FOLLOW UP .. SEE superacepigeon.weebly.com . WHAT TO ...

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RACING PIGEON TIPS 2 ....... TRICKS OF THE TRADE - Video

Genetics and Care for Children with Congenital Heart Defects
The "Genetics and Care for Children with Congenital Heart Defects" seminar was presented by Dr. John Belmont and Susan Fernbach, RN, BSN on Aug. 1, 2013. This is part of the Evenings with Genetics...

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Genetics and Care for Children with Congenital Heart Defects - Video

Genetics 080913

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Genetics 080913 - Video

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/pnxjqz/drug_delivery_in) has announced the addition of Jain PharmaBiotech's new report "Drug Delivery in Central Nervous System Diseases - Technologies, Markets and Companies" to their offering.

The delivery of drugs to central nervous system (CNS) is a challenge in the treatment of neurological disorders. Drugs may be administered directly into the CNS or administered systematically (e.g., by intravenous injection) for targeted action in the CNS. The major challenge to CNS drug delivery is the blood-brain barrier (BBB), which limits the access of drugs to the brain substance.

Advances in understanding of the cell biology of the BBB have opened new avenues and possibilities for improved drug delivery to the CNS. Several carrier or transport systems, enzymes, and receptors that control the penetration of molecules have been identified in the BBB endothelium. Receptor-mediated transcytosis can transport peptides and proteins across the BBB. Methods are available to assess the BBB permeability of drugs at the discovery stage to avoid development of drugs that fail to reach their target site of action in the CNS.

Various strategies that have been used for manipulating the blood-brain barrier for drug delivery to the brain include osmotic and chemical opening of the blood-brain barrier as well as the use of transport/carrier systems. Other strategies for drug delivery to the brain involve bypassing the BBB. Various pharmacological agents have been used to open the BBB and direct invasive methods can introduce therapeutic agents into the brain substance. It is important to consider not only the net delivery of the agent to the CNS, but also the ability of the agent to access the relevant target site within the CNS. Various routes of administration as well as conjugations of drugs, e.g., with liposomes and nanoparticles, are considered. Some routes of direct administration to the brain are non-invasive such as transnasal route whereas others involve entry into the CNS by devices and needles such as in case of intrathecal and intracerebroventricular delivery. Systemic therapy by oral and parenteral routes is considered along with sustained and controlled release to optimize the CNS action of drugs. Among the three main approaches to drug delivery to the CNS - systemic administration, injection into CSF pathways, and direct injection into the brain - the greatest developments is anticipated to occur in the area of targeted delivery by systemic administration.

Many of the new developments in the treatment of neurological disorders will be biological therapies and these will require innovative methods for delivery. Cell, gene and antisense therapies are not only innovative treatments for CNS disorders but also involve sophisticated delivery methods. RNA interference (RNAi) as a form of antisense therapy is also described.

The role of drug delivery is depicted in the background of various therapies for neurological diseases including drugs in development and the role of special delivery preparations. Pain is included as it is considered to be a neurological disorder. A special chapter is devoted to drug delivery for brain tumors. Cell and gene therapies will play an important role in the treatment of neurological disorders in the future.

The method of delivery of a drug to the CNS has an impact on the drug's commercial potential. The market for CNS drug delivery technologies is directly linked to the CNS drug market. Values are calculated for the total CNS market and the share of drug delivery technologies. Starting with the market values for the year 2012, projections are made to the years 2017 and 2022. The markets values are tabulated according to therapeutic areas, technologies and geographical areas. Unmet needs for further development in CNS drug delivery technologies are identified according to the important methods of delivery of therapeutic substances to the CNS. Finally suggestions are made for strategies to expand CNS delivery markets. Besides development of new products, these include application of innovative methods of delivery to older drugs to improve their action and extend their patent life.

Profiles of 75 companies involved in drug delivery for CNS disorders are presented along with their technologies, products and 75 collaborations. These include pharmaceutical companies that develop CNS drugs and biotechnology companies that provide technologies for drug delivery. A number of cell and gene therapy companies with products in development for CNS disorders are included. References contains over 400 publications that are cited in the report. The report is supplemented with 51 tables and 11 figures.

Key Topics Covered:

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Research and Markets: Drug Delivery in Central Nervous System Diseases - Technologies, Markets and 75 Company Profiles ...

Public release date: 29-Aug-2013 [ | E-mail | Share ]

Contact: NHLBI Communications NHLBI_news@nhlbi.nih.gov 301-496-4236 NIH/National Heart, Lung and Blood Institute

By lowering the expression of a single gene, researchers at the National Institutes of Health have extended the average lifespan of a group of mice by about 20 percent -- the equivalent of raising the average human lifespan by 16 years, from 79 to 95. The research team targeted a gene called mTOR, which is involved in metabolism and energy balance, and may be connected with the increased lifespan associated with caloric restriction.

A detailed study of these mice revealed that gene-influenced lifespan extension did not affect every tissue and organ the same way. For example, the mice retained better memory and balance as they aged, but their bones deteriorated more quickly than normal.

This study appears in the Aug. 29 edition of Cell Reports.

"While the high extension in lifespan is noteworthy, this study reinforces an important facet of aging; it is not uniform," said lead researcher Toren Finkel, M.D., Ph.D., at NIH's National Heart, Lung, and Blood Institute (NHLBI). "Rather, similar to circadian rhythms, an animal might have several organ-specific aging clocks that generally work together to govern the aging of the whole organism."

Finkel, who heads the NHLBI's Laboratory of Molecular Biology in the Division of Intramural Research, noted that these results may help guide therapies for aging-related diseases that target specific organs, like Alzheimer's. However, further studies in these mice as well as human cells are needed to identify exactly how aging in these different tissues is connected at the molecular level.

The researchers engineered mice that produce about 25 percent of the normal amount of the mTOR protein, or about the minimum needed for survival. The engineered mTOR mice were a bit smaller than average, but they otherwise appeared normal.

The median lifespan for the mTOR mice was 28.0 months for males and 31.5 months for females, compared to 22.9 months and 26.5 months for normal males and females, respectively. The mTOR mice also had a longer maximal lifespan; seven of the eight longest-lived mice in this study were mTOR mice. This lifespan increase is one of the largest observed in mice so far.

While the genetically modified mTOR mice aged better overall, they showed only selective improvement in specific organs. They generally outperformed normal mice of equivalent age in maze and balance tests, indicating better retention of memory and coordination. Older mTOR mice also retained more muscle strength and posture. However, mTOR mice had a greater loss in bone volume as they aged, and they were more susceptible to infections at old age, suggesting a loss of immune function.

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Single gene change increases mouse lifespan by 20 percent

Aug. 29, 2013 Researchers have identified a novel disease gene in which mutations cause rare but devastating genetic diseases known as mitochondrial disorders.

Nine rare, disease-causing mutations of the gene, FBXL4, were found in nine affected children in seven families, including three siblings from the same family. An international team of researchers report the discovery in the American Journal of Human Genetics.

The lead author is Xiaowu Gai, PhD, director of the Center for Biomedical Informatics at Loyola University Chicago Stritch School of Medicine.

Mitochondrial diseases are caused by defects in mitochondria, the cell's energy plants. Malfunctions in mitochondria lead to multi-systemic defects in the brain, heart, muscles, kidney and endocrine and respiratory systems. The many possible clinical symptoms include loss of motor control, muscle weakness, heart disease, diabetes, respiratory problems, seizures, vision and hearing problems, diabetes and developmental delays.

Mitochondrial diseases are caused by mutations in either mitochondrial DNA or in genes in the nucleus that encode for proteins that function in the mitochondria. Mitochondrial DNA is inherited from the mother. Thus, a child can inherit a mitochondrial disease either from the mother alone or from both parents carrying mutations in the same nuclear gene. Mitochondrial diseases affect between 1 in 4,000 and 1 in 5,000 people.

FBXL4 is a nuclear gene that encodes for a protein called F-Box and Leucine-Rich Repeat Protein 4. The study found that mutations of this gene lead to either truncated or altered forms of the protein. This results in cells having less mitochondrial DNA, decreased mitochondrial membrane potential and a faulty process in cell metabolism called oxidative phosphorylation. The study also proved that the FBXL4 protein is located exclusively in mitochondria, which was previously unrecognized.

While mutations in more than 100 genes have been linked to mitochondrial diseases, the new discovery adds another novel disease gene to the list. Consequently, genetic testing will enable more parents to discover the cause of their childrens' mitochondrial diseases. "This knowledge will help give them the peace of mind that it was not something they did to cause the disease," Gai said. More importantly, the discovery also will improve scientists' understanding of mitochondrial diseases, and potentially lead to new drugs to treat the disorders, Gai said.

The discovery began with an 8-year-old girl who had a mitochondrial disease known as Leigh syndrome. She has been seen by Dr. Marni J. Falk of the Children's Hospital of Philadelphia, who is a co-senior author of the study. A battery of genetic tests of the girl and her parents over the years all had failed to find any of the gene mutations previously known to cause mitochondrial diseases.

Gai and Falk used the high-performance computer cluster at Loyola's Center for Biomedical Informatics to analyze billions of DNA sequences to identify the gene mutation in the child and her parents. The research team then reached out to other collaborators to see if any of their patients also had the FBXL4 mutation. Eight additional affected children in six unrelated families were found to also have disease-causing mutations in this gene.

The discovery is an example of how Loyola's Center for Biomedical Informatics is using computational approaches to address basic biomedical questions. Isolating an unknown mutation can involve sequencing and analyzing a patient's entire genome, containing 6 billion base pairs (DNA letters). Powerful computational approaches and infrastructure are required to read and compare sequences of billions of DNA base pairs.

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Gene that causes devastating mitochondrial diseases identified

Aug. 29, 2013 Investigators at The Feinstein Institute for Medical Research have discovered a new way to measure the progression of Huntington's disease, using positron emission tomography (PET) to scan the brains of carriers of the gene. The findings are published in the September issue of The Journal of Clinical Investigation.

Huntington's disease causes the progressive breakdown of nerve cells in the brain, which leads to impairments in movement, thinking and emotions. Most people with Huntington's disease develop signs and symptoms in their 40s or 50s, but the onset of disease may be earlier or later in life. Medications are available to help manage the symptoms of Huntington's disease, but treatments do not prevent the physical, mental and behavioral decline associated with the condition.

Huntington's disease is an inherited disease, passed from parent to child through a mutation in the normal gene. Each child of a parent with Huntington's disease has a 50/50 chance of inheriting the Huntington's disease gene, and a child who inherits the gene will eventually develop the disease. Genetic testing for Huntington's disease can be performed to determine whether a person carries the gene and is developing the disease even before symptoms appear. Having this ability provides an opportunity for scientists to study how the disease first develops and how it progresses in its early, presymptomatic stages. Even though a carrier of the Huntington's disease gene may not have experienced symptoms, changes in the brain have already taken place, which ultimately lead to severe disability. Brain imaging is one tool that could be used to track how quickly Huntington's disease progresses in gene carriers. Having a better way to track the disease at its earliest stages will make it easier to test drugs designed to delay or even prevent the onset of symptoms.

Researchers at the Feinstein Institute used PET scanning to map changes in brain metabolism in 12 people with the Huntington's disease gene who had not developed clinical signs of the illness. The researchers scanned the subjects repeatedly over a seven-year period and found a characteristic set (network) of abnormalities in their brains. The network was used to measure the rate of disease progression in the study participants. The Feinstein Institute investigators then confirmed the progression rate through independent measurements in scans from a separate group of Huntington's disease gene carriers who were studied in the Netherlands. The investigators believe that progression networks similar to the one identified in Huntington's disease carriers will have an important role in evaluating new drugs for degenerative brain disorders.

"Huntington's disease is an extremely debilitating disease. The findings make it possible to evaluate the effects of new drugs on disease progression before symptoms actually appear. This is a major advance in the field," said David Eidelberg, MD, Susan and Leonard Feinstein Professor and head of the Center for Neurosciences at the Feinstein Institute.

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Researchers track Huntington's disease progression using PET scans

Aug. 29, 2013 Biologists at the University of Fribourg have been looking at a threadworm gene which also occurs in humans. This gene could be central to a genetic system which is responsible for development, reproduction and the ageing process.

Ageing involves a deterioration in physiological functions which inevitably leads to death. The risk of contracting age-related diseases such as cancer, diabetes and cardiovascular and neurodegenerative disorders is increased by the body's deterioration. Latest advances in research permit the isolation of genetic factors which control not only ageing but also the occurrence of age-related diseases.

Prof. Fritz Mller, Dr. Chantal Wicky and their research team have highlighted the importance of the gene let-418/Mi2 in the Caenorhabditis elegans worm because it regulates ageing and stress resistance as well as being essential for development and reproduction. The researchers have discovered that when the gene is deactivated in adult worms in the laboratory, they live longer and are considerably more resistant to stress. The gene forms part of a genetic system which plays a key beneficial role in growth and reproduction. But as soon as these stages are over, the effects become harmful.

Thanks to their collaboration with Prof. Simon Sprecher's recently formed research team at the University of Fribourg, the researchers were able to establish that this gene also operates as an ageing and stress regulator in the case of flies and plants. This indicates that the mechanism of action of this gene has been preserved over the course of evolution and may function similarly in humans. Deactivating the gene after the reproductive stage is over would enable the human body to enjoy a significant increase in life expectancy since its level of resilience would rise and the occurrence of age-related illnesses would diminish. The study of such factors -- which have negative as well as positive effects according to the stage of life -- represents a huge potential for human medicine.

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Biologists may have identified gene central to development, reproduction and aging

Public release date: 27-Aug-2013 [ | E-mail | Share ]

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

New Rochelle, NY, August 26, 2013New classes of therapeutic antisense oligonucleotides can have toxic effects on the liver. A novel machine learning-based approach used to predict the hepatotoxic potential of an antisense drug based on its chemical sequence is presented in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. The article is available on the Nucleic Acid Therapeutics website.

Peter Hagedorn and coauthors from Santaris Pharma, Hrsholm, Denmark, and University of Copenhagen, Denmark, describe the use of machine learning techniques to develop a method of classifying therapeutic oligonucleotides based on their DNA sequence and modification patterns. Computers create a classification scheme linking this variation to a compound's potential to cause liver toxicity.

In the article "Hepatotoxic Potential of Therapeutic Oligonucleotides Can Be Predicted from Their Sequence and Modification Pattern," the authors demonstrate the use of this approach to predict the hepatotoxicity of a validation set of oligonucleotides with 74% accuracy. They also use the classifier scheme to identify a therapeutic oligonucleotide with high potential liver toxicity and show how the drug could be redesigned to reduce its potential toxicity.

"As is true of all potential therapeutic entities, whether small molecules or nucleic acid-based drugs, it is critical to understand as much as possible about possible toxic effects before proceeding to clinical trials," says Executive Editor Fintan Steele, PhD, SomaLogic, Inc., Boulder, CO. "The approach described by these authors holds great promise for maximizing the safety of in vivo testing and, we hope, the eventual clinical use of these new antisense-based compounds."

###

Nucleic Acid Therapeutics is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC, and C.A. Stein, MD, PhD, City of Hope National Medical Center, Duarte, CA; and Executive Editor Fintan Steele, PhD (SomaLogic, Boulder, CO).

About the Journal

Nucleic Acid Therapeutics is an authoritative, peer-reviewed journal published bimonthly in print and online that focuses on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. Nucleic Acid Therapeutics is the official journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a free sample issue may be viewed on the Nucleic Acid Therapeutics website.

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Can toxicity of a DNA drug be predicted and minimized?

By Eric Langer, president and managing partner, BioPlan Associates, Inc.

Technological advances in genetic engineering, particularly expression systems (the genetically modified cells that express desired proteins), process design, and equipment continue to be combined such that the same amount of drug product can be manufactured at a much smaller scale. Today, smaller-scale, less-expensive equipment is permitting more rapid drug development and production in smaller facilities.

This years 10th Annual Report and Survey of Biopharmaceutical Manufacturing Capacity and Production continues to show overall increased productivity and efficiency in biomanufacturing. The average expression yield (amount of protein produced in a fixed bioreactor fluid volume), exemplified by mammalian cell culture production of monoclonal antibodies, is now reported to be 2.68 grams/Liter for late-stage clinical supplies manufacturing and 2.29 grams/Liter for commercial-scale manufacturing. These production yields have been increasing since 2008 at an average annual growth rate of 9.8 percent. This almost exclusively involves cell culture using Chinese hamster ovary (CHO) cells. With CHO serving very well and major changes generally avoided in this highly regulated industry, adoption of other and improved expression systems, despite offering further advantages, remains slow.

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Innovation In Expression Systems Yields Increase Productivity

The personalised medicine diagnostics market has been forecast to increase at a compound annual growth rate (CAGR) of 4.43% through 2016, driven by the increasing demand for personalised medicine for treating cancer, and the increase in the number of industrial activities.

One of the main trends witnessed in the personalised medicine diagnostics market is the development of innovative tools and technologies. For instance, AssureRx Health Inc. launched GeneSightRx ADHD in May 2012, a personalised medicine test for the growing number of children and adults diagnosed with ADHD.

Moreover, Bristol-Myers Squibb Co. collaborated with MMRF in December 2012 for advanced personalised medicine for multiple Myeloma. Alacris Theranostics GmbH entered into an agreement with GlaxoSmithKline plc in November 2012 to use ModCell system for early stage cancer research.

Furthermore, AltheaDx Inc. launched FLT3 Mutation Assay in October 2012. Agendia N.V. launched ColoPrint microarray-based 18-gene expression signature in June 2012 to predict the risk of distant recurrence for stage II colon cancer patients. The European Medicines Agency approved Zelboraf, manufactured by F. Hoffmann-La Roche Ltd., in December 2011.

Personalised medicine is a medical model that proposes the customization of healthcare - with medical decisions, practices, and/or products being tailored to the individual patient. The use of genetic information has played a major role in certain aspects of personalised medicine, and the term was even first coined in the context of genetics.

To distinguish from the sense in which medicine has always been inherently "personal" to each patient, personalised medicine commonly denotes the use of some kind of technology or discovery enabling a level of personalisation not previously feasible or practical.

One of the main challenges the industry faces is the low awareness regarding personalised medicine diagnostics in developing countries. The majority of patients in developing countries such as India and China are not fully aware of the recent developments in personalised medicine diagnostics. The high cost of personalised medicine diagnostics in certain treatments is another major challenge for the market in these countries.

Key players currently dominating the personalised medicine diagnostics market include Abbott Laboratories, Agilent Technologies Inc., Becton Dickinson and Company, bioMerieux SA, Illumina Inc., and Roche Holding AG.

For more information on the personalised medicine diagnostics market, see the latest research: Personalised Medicine Diagnostics Market

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Personalised medicine diagnostics market to increase at a CAGR of 4.4%

Aug. 29, 2013 A Cleveland Clinic researcher has discovered a genetic mutation in a drug-resistant -- and often deadly -- form of prostate cancer.

The mutation occurs in the androgen-synthesizing enzyme 3HSD1 in castration-resistant prostate cancer (CRPC), according to research published online today in Cell. This mutation enables the tumor to make its own supply of androgens, a hormone that fuels the growth of the prostate cancer.

Prostate cancer requires a constant supply of androgens in order to sustain itself. The current standard of care for patients with metastatic prostate cancer is medical castration, the ability to interfere with the body's production of testosterone (androgens) using medications that disrupt the process. Oftentimes, metastatic prostate cancer flourishes despite the lack of testosterone in the bloodstream, creating CRPC. These tumors are able to exist without the body's supply of testosterone by creating androgens within the tumor cell; however, increased androgen synthesis has not yet been attributable to any known mutations. The Cleveland Clinic discovery shows that the 3HSD1 mutation makes this enzyme hyperactive to create androgens.

"This discovery gives us the ability to identify molecular subtypes of prostate cancer known to resist treatment. By finding the mutated enzyme, we can now investigate treatments that block it. This kind of strategy is the crux of personalized medicine which is currently used as the standard of care for some forms of lung cancer and melanoma," said Nima Sharifi, MD, Kendrick Family Chair for Prostate Cancer Research at Cleveland Clinic, who led the research.

The 3HSD1 mutation can occur within CRPC tumors and it can also come from germline DNA, which is inherited from maternal and paternal sources.

The research found that laboratory models of human prostate cancer fall into two categories of androgen synthesis: those that make androgens slowly and those that do so rapidly. Next, they found that the 3HSD1 mutation explains the difference between these two categories and that DNA from some patient tumors also contains this mutation. The mutation works by opening the floodgates to androgen synthesis, essentially throwing fuel on the fire that promotes tumor progression.

In an era of personalized cancer care, there is increased focus on defining and treating cancer by its genetic abnormalities. Tumor-promoting enzyme mutations in several cancers have been identified and, subsequently, have led to the development of targeted drug therapies, improving outcomes for patients.

"The past decade has seen an explosion of molecularly targeted therapies that are matched to specific mutations in a given patient's tumor," says Dr. Sharifi. "However, no drug-targeting based on enzyme mutations exists for the standard treatment of metastatic CRPC. With this finding, we have the opportunity for matching a mutant disease-driving biomarker with a pharmacologic inhibitor."

Prostate cancer is the most common cancer in men, with nearly 240,000 new cases diagnosed each year in the United States. According to the American Cancer Society, there will be an estimated 30,000 deaths due to prostate cancer in 2013. Almost every man who dies of prostate cancer dies with castration-resistant prostate cancer.

This research was funded by Prostate Cancer Foundation, American Cancer Society, Department of Defense, Howard Hughes Medical Institute and National Cancer Institute.

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Genetic mutation found in castration-resistant prostate cancer

Aug. 25, 2013 Of the over 1,900 errors already reported in the gene responsible for cystic fibrosis (CF), it is unclear how many of them actually contribute to the inherited disease. Now a team of researchers reports significant headway in figuring out which mutations are benign and which are deleterious. In so doing, they have increased the number of known CF-causing mutations from 22 to 127, accounting for 95 percent of the variations found in patients with CF.

In a summary of their research to be published online in Nature Genetics Aug. 25, the scientists say that characterizing those additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene will not only bring certainty to families about a CF diagnosis or carrier status, but will also accelerate the design process for new drugs tailored to a particular mutation. There already is one such individualized drug on the market.

"Since not all mutations cause disease, sequencing the DNA in both copies of your CFTR gene and finding an abnormality in one wouldn't tell us if you are a carrier for CF unless we knew if that abnormality causes CF," says Garry Cutting, M.D., professor of pediatrics in the McKusick-Nathans Institute of Genetic Medicine at the Johns Hopkins University School of Medicine. "Until this new work, more than a quarter of couples in which both partners were found to carry a CFTR mutation were left wondering if their mutations were going to affect their offspring. Now it's down to 9 percent," he says.

CF is the most common, lethal, recessive genetic disease affecting Caucasians, with approximately 70,000 to 80,000 cases worldwide. When two copies of a defective CFTR gene are inherited, one from each parent, a child's body will not be able to create working CFTR proteins, resulting in the production of thickened mucus, which clogs the lungs and digestive system. Modern treatments to unclog the lungs and address other symptoms have allowed patients to survive into adulthood, but most will still die prematurely of lung disease.

One in 30 Caucasians in the United States is a "carrier" of the disease, meaning their genomes include one abnormal copy of the CFTR gene but they experience no symptoms of the disease, and as many as a million Americans are tested each year for carrier status. If two carriers have children together, each child has a 1-in-4 chance of inheriting two bad copies of CFTR and suffering from the disease. The severity of the disease will depend on which particular gene variations are inherited and how they affect the functioning of the CFTR protein.

In 2012, a drug (ivacaftor) that enhances the function of one specific mutant form of the protein became available. Although the particular mutation targeted is only found in 4 percent of patients with CF, drug companies are already working on drugs to target mutant proteins resulting from other mutations.

"There is very important information in each of these naturally occurring mutations that teaches us more and more about the disease," says Patrick Sosnay, M.D., assistant professor of pulmonary and critical care medicine. "We want to get to a point where we can say, 'This is your mutation, this is what it means and this is how you can treat it.'"

The team began its study with a database containing the genetic information of nearly 40,000 patients with CF. It then examined the 159 mutations that occurred in the database at a frequency of at least 0.01 percent. (Most of the more than 1,900 known mutations are even more rare than that.) The research team analyzed each of these mutations to determine its clinical relevance and its effect on the work of the CFTR protein.

The impact of each mutation on patients' health was assessed by first examining data on the salt concentrations in the sweat of patients bearing each particular mutation. CF causes unusually high amounts of salt to appear in sweat, so a mutation was deemed clinically significant if patients carrying that mutation had high reported salt concentrations.

The team then looked at how each genetic error affected the protein made by the CFTR gene. Eighty of the mutations would prevent the production of any CFTR protein based on the location of the mutation. These were classified as disease-causing, Cutting said. The remaining 77 mutations were tested biochemically in cells to determine the amount of damage sustained by the CFTR protein in each case.

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Scientists pinpoint 105 additional genetic errors that cause cystic fibrosis

Public release date: 27-Aug-2013 [ | E-mail | Share ]

Contact: Todd Datz tdatz@hsph.harvard.edu 617-432-8413 Harvard School of Public Health

Boston, MA A newly discovered genetic variant may increase the risk of heart disease in people with type 2 diabetes by more than a third, according to a study led by researchers at Harvard School of Public Health (HSPH) and Joslin Diabetes Center. It is the first genome-wide association study (GWAS) to identify a novel genetic variant associated with coronary heart disease (CHD) in people with type 2 diabetes, who have a two- to four-fold higher risk of heart disease compared with those without diabetes. The finding could lead to new interventions aimed at preventing or treating CHD among patients with type 2 diabetes.

"This is a very intriguing finding because this variant was not found in previous genome-wide association studies in the general population," said lead author Lu Qi, assistant professor in the HSPH Department of Nutrition and assistant professor at the Channing Division of Network Medicine, Brigham and Women's Hospital. "This means that the genetic risk factors for cardiovascular disease may be different among those with and without diabetes."

"The identification of this genetic variant opens up the possibility of developing treatments that are specifically aimed at breaking the links between diabetes and CHD," said co-lead author Alessandro Doria, associate professor in the Department of Epidemiology at HSPH and a researcher at Joslin Diabetes Center.

The study appears online August 27 and will appear in the August 28, 2013 issue of JAMA (Journal of the American Medical Association).

More than 370 million people worldwide have type 2 diabetes and CHD is the leading cause of death among diabetic patients. Overall CHD-related mortality has been declining in the United States and other industrialized countries over the past few decades. But CHD deaths that are diabetes-related are on the rise because of the increasing prevalence of the latter ailment. Although prior genome-wide studies have found many genetic variants for CHD in people in the general population, no such study had examined genetic determinants for CHD specifically in those with type 2 diabetes.

For their analysis, the researchers used data from several long-term studies: the Nurses' Health Study, the Health Professionals Follow-up Study, the Joslin Heart Study, and two Italian studiesthe Gargano Heart Study and the Catanzaro Study. They looked at 4,188 diabetic patients, including 1,517 with CHD and 2,671 without CHD as a control group.

Testing more than 2.5 million genetic variants, the researchers found that a variant near the GLUL gene, a gene that encodes a key enzyme regulating the conversion of glutamic acid to glutamine, was consistently associated with a 36% increased risk of CHD in people with diabetes. There was no association between this variant and CHD risk in study participants without diabetes.

They also found that the variant may interfere with the expression of a gene that regulates blood levels of amino acids involved in insulin secretion and glucose metabolismkey functions that go awry in those with type 2 diabetes.

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Genetic variant identified that may increase heart disease risk among people with type 2 diabetes

August 29, 2013

redOrbit Staff & Wire Reports Your Universe Online

By creating a model of genetic code evolution, researchers have discovered new information about how RNA signaling could have developed into the near-optimal modern genetic code.

Lead author Justin Jee, a doctoral student at NYU School of Medicine, and colleagues set out to account for the composition of the genetic code, which makes it possible for proteins to be constructed from amino acids with high specificity based on information stored in a RNA or DNA genome.

Our model shows that todays genetic code probably resulted from a combination of selective forces and random chance, Jee said. His teams research, which they say could help explain the complexities of the origins of life, appears in the latest edition of the Journal of the Royal Society Interface.

The translation process between nucleic acids and amino acids is largely universal a phenomenon the researchers refer to as mysterious and remarkable. The same code is shared in all types of organisms, ranging from bacteria to humans, and at the same time it is nearly perfect in terms of how well it is able to select the correct type of amino acids for specific particular nucleic acid sequences.

Ever since the code was first discovered some five decades ago, experts have wondered how a near-optimal code also became so universal in nature. In order to try and discover the answer, Jee and his associates crafted a model of genetic code evolution in which multiple translating RNAs and genomic RNAs competed for survival. The translating RNAs were able to link amino acids together using data stored in the genomic RNA, they explained.

In running computer simulations of RNA interactions, they could see two phenomena. First, it was necessary for the translating and genomic RNAs to organize into cells, which aided the coordination of a code between the translating and genomic RNAs. Second, selective forces led a single set of translating RNAs to dominate the population, the university said. In other words, the emergence of a single, universal, near-optimal code was a natural outcome of the model. Even more remarkably, the results occurred under realistic conditions specifically, they held under parameters such as protein lengths and rates of mutation that likely existed in a natural RNA world.

The most elegant ideas in this paper are rather obvious consequences of a well-studied model based on sender-receiver games, added senior author Bud Mishra of the NYU School of Medicines Sackler Institute of Graduate Biomedical Sciences. Yet the results are still very surprising because they suggest, for example, that proteins, the most prized molecules of biology, might have had their origin as undesirable toxic trash. Other studies based on phylogenomic analysis seem to be coming to similar conclusions independently.

In addition to Jee and Mishra, study co-authors included Andrew Sundstrom of the Courant Institute and Steven Massey of the University of Puerto Ricos Department of Biology. The research was funded by grants from the National Science Foundation (NSF) and a National Defense Science and Engineering Graduate Fellowship from the US Department of Defense.

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Model Of 'Near Optimal' Genetic Code Created By NYU Researchers