Archive for the ‘Gene Therapy Research’ Category

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Newswise A novel study by the Cancer Science Institute of Singapore (CSI Singapore) at the National University of Singapore (NUS) found that an increase in a gene known as Leo1 affects other genes that are directly implicated in acute myelogenous leukaemia (AML), increasing the incidence of cancer.

Led by Associate Professor Chng Wee Joo, Deputy Director and Senior Principal Investigator at CSI Singapore and Director of the National University Cancer Institute, Singapore, the scientists discovered that inhibition of Leo1 and Leo1 downstream signalling pathways provide an avenue for targeted treatment of AML. The findings were recently published in Cancer Research, the official journal of the American Association of Cancer Research.

In addition, this is the first study to suggest that the protein PRL-3 plays a role in the regulation of ribonucleic acid (RNA) related processes, a finding which advances the understanding of how the protein contributes to cancer progression. The teams work represents the first large-scale quantitative survey of proteins regulated by PRL-3 in leukaemia.

The elevated expression of PRL-3 has been implicated in the progression and metastasis of an array of cancer types, including gastric, ovarian, cervical, lung, liver, and breast. In particular, the protein PRL-3 is overexpressed in about half of AML patients and associated with poor survival. Assoc Prof Chng and his team were the first to report that elevated PRL-3 protein expression occurs in about 47 per cent of AML cases while being absent from normal myeloid cells in bone marrow. As a result, PRL-3 is deemed as an attractive therapeutic target that spares normal tissues.

Previously, knowledge of the mechanisms of PRL-3 was limited. In this study, the researchers used a new, advanced SILAC-based mass spectrometry to identify all the protein changes induced by PRL-3 in a comprehensive manner. Using this approach, they discovered that the gene Leo1 serves as a novel target of PRL-3 phosphatase, and inhibition of Leo1 as well as Leo1 downstream signalling pathways provide an avenue for PRL-3 targeted therapy for AML patients.

In the next phase of research, the team is validating several important proteins directly downstream of Leo1 that can possibly be used as biomarkers and drug targets to improve treatment for leukaemia with PRL-3 overexpression.

Assoc Prof Chng said, Our previous studies showed that PRL-3 is clinical and biologically important in acute myelogenous leukaemia, and may therefore be a useful treatment target. In the current study, we have taken the work further by understanding how PRL-3 confers cancer properties to the leukaemia cells. This now provides a framework for rational design of a treatment based on mechanistic understanding. In the process, we will also develop biomarkers to better select patients for the treatment and hence, progress towards personalising treatment for leukaemia patients.

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Singapore Researchers Discover a Gene That Increases Incidence of Acute Myelogenous Leukaemia

Released: 17-Sep-2014 10:00 AM EDT Source Newsroom: Alzforum Contact Information

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Newswise Rising to fame from humble beginnings in bacteria, the CRISPR gene editing system has taken the biomedical field by storm. Using an ever-evolving genetic toolbox to change, turn off, turn on, add, or delete genes, scientists can tinker with any region of the genome. The technology may one day allow clinicians to correct mutations in genetic diseases or deliver therapeutic genes to mend damaged cells or kill cancerous ones, scientists predict.

Neuroscientists have adopted CRISPR to neurons, both in a dish and in living animals, and they have set their sights on using the technology to treat neurodegenerative diseases such as ALS, Parkinsons, Huntingtons, and Alzheimers. In a two-part series, Alzforum explains the CRISPR technology and its first neuroscience forays (Part I), and delves into how scientists will engineer transgenic animals and perhaps treat disease through CRISPR gene therapy (Part II).

About Alzforum: Founded in 1996, Alzforum is a dynamic, Web-based scientific community dedicated to understanding Alzheimer's disease and related disorders. Access to the website is free to all. Our editorial priorities are as diverse as the needs of the research community. The website reports on the latest scientific findings from basic research to clinical trials, creates and maintains public databases of essential research data and reagents, and produces discussion forums to promote debate, speed the dissemination of new ideas, and break down barriers across the numerous disciplines that can contribute to the global effort to cure Alzheimer's disease. The Alzforum team of professional science writers and editors, information technology experts, Web developers, and producers all work closely with our distinguished Advisory Board to ensure a high quality of information and services. We very much welcome our readers' participation in all aspects of the website.

Alzforum is developed and operated by the Biomedical Research Forum (BRF) LLC. BRF is a wholly owned subsidiary of FMR LLC. FMR LLC and its affiliates invest broadly in many companies, including life sciences and pharmaceutical companies. The Alzforum website does not endorse any specific product or scientific approach.

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Neuroscientists Get Busy in the CRISPR Kitchen--What Will They Cook Up?

18 hours ago by Stuart Mason Dambrot The T4 capsid-derived specific exogenous DNA plus protein packaging and eukaryotic cell delivery scheme. (A) DNA encoding a 10-amino acid N-terminal CTS peptide fused to the phage P1 Cre allows synthesis of CTS-Cre and targeting of the enzyme into the early core-scaffold of the T4 procapsid in vivo. Procapsid assembly and maturation-specific viral protease stabilize the procapsid, remove most of the scaffolding core as peptides, and remove the CTS peptide from Cre. Mutations in the viral terminase block DNA packaging and allow a mature but DNA-empty large Cre-containing procapsid to be highly purified from viral-infected bacteria. (B) In vitro packaging into the mature capsid of plasmid DNA containing mCherry driven by a CMV promoter and two loxP sites flanking an SfiI restriction enzyme site that allow the linearization required for packaging. The DNA is packaged into the procapsid by the ATP-driven terminase motor protein (gp17) with high efficiency. (C) The packaged Cre enzyme recircularizes the packaged linear plasmid DNA between the two loxP sites. The DNA-containing capsid is taken up by eukaryotic cells, here without displaying a specific peptide target, or into eukaryotic cells specifically using Soc and Hoc displayed peptides that have high affinity for the RP1 and RP2 receptors, respectively. Credit: Liu JL, et al. (Published online before print August 26, 2014) Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression. Credit: Liu JL, et al. (2014) Viral nanoparticle-encapsidated enzyme and restructured DNA for cell delivery and gene expression. Proc Natl Acad Sci USA Published online before print August 26, 2014. doi:10.1073/pnas.1321940111

(Phys.org) By loading any specific protein and nucleic acid into an icosahedral phage T4 capsid-based nanoparticle, the resulting cell delivery vehicle's ligands can bind to the surface of specific target tissues to deliver the protein/DNA cargo. (Icosahedral viral nanoparticles are evolutionary protein shells assembled in a hierarchical order that results in a stable protein layer and an inner space for accommodating nucleic acids and proteins; a capsid is the protein shell of a virus.) The technique has drug- and gene-delivery applications in human diseases, diagnostic and cellular imaging, and other medical areas. Recently, scientists at US Naval Research Laboratory, Washington, DC and University of Maryland at Baltimore packaged T4 nanoparticles in vivo with active cyclic recombination, or Cre, recombinase (a genetic recombination enzyme used to manipulate genome structure and control gene expression) and in vitro with fluorescent mCherry (a fluorescent protein used as a marker when tagged to molecules and cell components) expression plasmid DNA, and delivered these nanoparticles into cancer cells: When released into cells in the presence of both DNA and protein, the recombinase enhances mCherry expression by circularization (that is, changing the packaged linear DNA into a circular loop). The researchers state that this efficient and specific packaging into capsids and the unpackaging of both DNA and protein with release of the enzymatically altered protein/DNA complexes from the nanoparticles into cells have potential in numerous downstream applications such as genetic and cancer therapeutics.

Dr. Jinny L. Liu discussed the paper that she, Prof. Lindsay W. Black and their co-authors published in Proceedings of the National Academy of Sciences USA. "Icosahedral viral nanoparticles are essentially 100 nm by 80 nm nanocontainers that allow exogenous genetic material to be packaged in vitro through nucleic acid machinery that generally only allows linear DNA/RNA to be packaged through a portal channel," Liu tells Phys.org. "However, in vitro protein packaging is generally impossible, because for most viral nanoparticles there is no protein packaging machinery comparable to nucleic acid packaging machinery." While protein may be chemically cross-linked to the capsid inner surface, this is expected to lead to protein denaturation and loss of enzymatic activity.

That being said, nature has evolved solutions to this protein packaging conundrum. During in vivo viral capsid assembly, Liu explains, some bacterial viruses, or bacteriophages, target proteins within the procapsids before the nucleic acid is packaged so as to eject the proteins with the nucleic acid, thereby facilitating infection in conjunction with the nucleic acid. (A procapsid, or prohead, is an immature viral capsid structure formed in the early stages of self-assembly of some bacteriophages. Production and assembly of stable proheads is an essential precursor to bacteriophage genome packaging.) Only a few phages have well-characterized in vivo protein packaging systems, and phage T4 is the best characterized. "Prof. Black's lab at UMB and my lab at NRL have demonstrated that not only can a specific foreign enzyme cyclic recombination (Cre) recombinase be packaged into the capsid in vivo, but also that it is active within the capsid." This activity was demonstrated by showing the religation (the rejoining of two DNA strands or other molecules by a phosphate ester linkage) of packaged linear DNA flanked with two Cre recombination sites.

The paper shows that the substantial space within a T4 nanocontainer accommodates the active Cre enzyme along with exogenous DNA. "For potential applications, T4 can package up to 50 kb exogenous linear DNA containing full-length desired genes along with recombinases, either Cre or -red proteins, for specific homologous recombination within the chromosome," Liu notes. (Homologous recombination is a type of genetic recombination in which nucleotide sequences are exchanged between two similar or identical molecules of DNA.) "We expect that the cas9 enzyme could be encapsidated in a comparable way and in fact, at least eight different proteins have been encapsidated in this manner. Through homologous recombination, our system can allow the corrected gene to replace the mutated gene in its original location within the chromosome or by precisely knocking out the overactive genes in stem cells." Liu points out that the T4 delivery vector is safer and better controlled than other viral delivery gene therapy, such as those delivering genes using infectious animal viral vectors to randomly insert the gene within the chromosome.

In their paper, the authors report that the T4 capsid NP gene expression and protein delivery system may be complementary to or used in conjunction with gene therapy based on RNA Cas and taran nuclease. (Cas genes code for proteins related to DNA loci containing short repetitions of base sequences known as Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPRs.) "The T4 nanoparticle expression system can easily complement Cas9 and taran nuclease-based recombination by packaging the linear cas9, target-sgRNA plasmid DNA, and Cre recombinase or even ligase, an enzyme that facilitates the joining of DNA strands and deliver the resulting T4 nanoparticles into the recipient eukaryotic cells with high specificity employing SOC and HOC," Liu tells Phys.org. (SOC and HOC are dispensable T4 capsid proteins.) "By displaying the targeting ligands (binding molecules) onto the surface, the T4 capsid gene expression and protein system will be able to efficiently deliver the Cas9 and sgRNA plasmids together into the desired recipient cells. Relevant enzymatically-active proteins Cas9, lambda exonuclease, lambda beta protein and others can be delivered directly at the same time from the T4 nanoparticle."

Liu adds that her lab has also been studying cell imaging and drug/gene delivery to eukaryotic cells using T4 tailless nanoparticles, which the researchers demonstrated can enter the eukaryotic cells without causing cell death.

A specific example of potential downstream drug and gene therapeutic applications resulting from the new approach is delivery of the toxic protein and linear plasmid that produces neutralizing peptides or antibodies into targeted cancer cells displaying specific cancer markers using high affinity SOC + HOC marker binding proteins on the surface of the capsids, while another example is to use the system for HIV gene therapy.

Liu adds that there are several pathways to use this system for gene therapy:

In addition to diagnostic and cellular imaging, the T4 nanoparticle gene-protein system can deliver repaired genes to correct human genetic diseases for example, reversing adenosine deaminase (ADA) deficiency by introducing the protein-DNA complex to express ADA in stem cells. Other broad areas of research impacted by gene therapy technologies, such as genetic defects, cancer, neurological diseases in adults, and aging itself, may also benefit from this study.

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Nanocontainers for nanocargo: Delivering genes and proteins for cellular imaging, genetic medicine and cancer therapy

TORONTO Twenty-five years ago this month, the medical world was turned on its ear with the isolation of the gene that causes cystic fibrosis, a devastating inherited disease that usually killed children by their late teens.

At the helm of the research was Lap-Chee Tsui, who led the team at Torontos Hospital for Sick Children that made the seminal discovery in collaboration with scientists at the University of Michigan.

The science of human genetics was still in its infancy at that time. Pinpointing the mutated CFTR gene came about through painstaking mapping of bits of DNA to locate the root of CF symptoms thick, sticky mucus that clogs the lungs and gums up the gastrointestinal tract, requiring patients to take scores of digestive enzymes a day so they could digest food.

The cystic fibrosis defect is really a very subtle defect, Tsui (pronounced Choy), 63, said Monday during an event at Sick Kids to mark the 1989 discovery. It didnt kill the patients (right away), but the problems accumulated slowly, and at the end the patients succumbed to infection.

Using the same analogy as he used in 1989 to explain CFTRs location on chromosome 7, Tsui said researchers first narrowed it down to somewhere between Halifax and Vancouver, then further pinpointed it in Toronto, and finally zeroed in to a certain street and then the actual house that represented the defective gene.

In the ensuing years, researchers have determined there is not only one mutation in the CF gene, but about 1,900 different defects that cause greater or lesser severity of symptoms in individual patients a scientific process Tsui likened to going into the house and turning on all the lights and taps to see which ones are faulty.

The celebrated geneticist, who left Toronto 12 years ago to become vice-chancellor and president of the University of Hong Kong, from which he just retired, called progress in understanding and treating CF since the gene was isolated very exciting.

Within two years of that discovery, other Sick Kids researchers had determined that a protein that keeps epithelial cells lining the lungs, airways and digestive system nice and moist was faulty, causing the buildup of mucus that clogs the lungs and disables the digestive system.

I think the expectation when the gene was first discovered was that it would be easy to fix because the disease was caused by a single gene, and if you replaced that gene through gene therapy, then you would be able to completely reverse the consequences of the disease, said senior scientist Christine Bear, who led that team.

And it may be that gene therapy will be part of that future therapy in CF, but right now we havent developed safe ways to do that.

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Canadian researchers mark 25 years since CF gene found

Stacey Burling, Inquirer Staff Writer Posted: Sunday, September 14, 2014, 3:51 AM

It's getting harder to find the line between science and science fiction.

One of the hot research techniques these days, "optogenetics," uses gene therapy to deliver light-sensitive proteins to specific cells. Then researchers use light to control the cells. The field got its start in the brain, where scientists have demonstrated the technique by making contented mice fly into a rage - a remarkable, if slightly creepy, achievement.

Brian Chow, a University of Pennsylvania bioengineer, has bigger ambitions than that.

He wants to develop optogenetic tools that help scientists unlock the secrets of all kinds of cells by triggering discrete cellular activities on demand, say the expression of a gene or the activation of a protein.

Scientists have never had that kind of control over specific cell functions before. Drugs affect large numbers of different kinds of cells. Electricity can be used in a small region, but not just one cell type. Brain imaging studies have let scientists see which parts of the brain were active during certain activities, but they couldn't tell what role they played.

Optogenetics - the combination of optics and genetics - lets researchers see exactly what specific cells do, and control when they do it.

"It just fundamentally allows us to answer questions we have not been able to answer in the past," Chow said.

"The promise of it is demonstrating causality as opposed to correlation."

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Illuminating technology

AAP A breast cancer patient says she's stunned that human genes can be patented by corporations.

Corporations can continue owning the rights to human genes thanks to a federal court ruling but patients at the heart of the legal battle say the practice is immoral.

Central to the test case, between support group Cancer Voices Australia and US biotech company Myriad Genetics, is the susceptibility gene known as BRCA1.

The validity of Myriad's patent over the so-called "cancer gene" in its isolated state has been challenged, on the basis that under Australian law, patents cannot be granted over products of nature, as opposed to inventions.

But on Friday, the full bench of the federal court upheld the rights of Myriad, the patent owner.

Maurice Blackburn lawyer Rebecca Gilsenan feared the gene patent could stifle research.

"It places limits on genetic testing, genetic research and the development of treatments and cures for genetically associated disease," she said.

The case began in 2010 on behalf of Queensland cancer patient Yvonne D'Arcy, 68, who was devastated by Friday's outcome.

"To me now, they've made it personal," she told reporters in Brisbane.

"I don't think it's right ... I don't think any private company should own part of a human body."

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Gene decision fuels calls for legal change

Yvonne D'Arcy, who fought to have the breast cancer gene patent overturned, outside the Federal Court in February last year. Photo: Peter Rae

Cancer survivors and advocates are devastated at a decision by the full bench of the Federal Court that private companies have the right to control human genes.

They fear the decision in the so-called "breast cancer gene" case, which found a company could patent mutations in the gene BRCA1, will lead to higher costs for patients in need of potentially life-saving tests.

They have called on the federal government to intervene to change the laws, andMaurice Blackburn, the lawyers that brought the case, have vowed to fight it "to the end", flagging a potential appeal to the High Court of Australia.

The structure of the protein produced by the BRCA1 gene. Photo: Supplied

However, patent lawyers say the laws are a fair reflection of the work done by the biotechnology industry, and the decision may draw business to Australia.

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Sally Crossing, from Cancer Voices Australia, said the Australian community had made it clear the patenting of human genes was "offensive and counter-intuitive."

"This news is not good for cancer research, especially in the promising field of targeted therapies, or for people affected by any cancer," she said.

Director of Advocacy at Cancer Council Australia, Paul Grogan, said that, if the ruling was an interpretation of the law, then the law needed to change.

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Mutation of breast cancer gene can be patented, says Federal Court

Yvonne D'Arcy, who fought to have the breast cancer gene patent overturned, outside the Federal Court in February last year. Photo: Peter Rae

Cancer survivors and advocates are devastated at a decision by the full bench of the Federal Court that private companies have the right to control human genes.

They fear the decision in the so-called "breast cancer gene" case, which found a company could patent mutations in the gene BRCA1, will lead to higher costs for patients in need of potentially life-saving tests.

They have called on the federal government to intervene to change the laws, andMaurice Blackburn, the lawyers that brought the case, have vowed to fight it "to the end", flagging a potential appeal to the High Court of Australia.

The structure of the protein produced by the BRCA1 gene. Photo: Supplied

However, patent lawyers say the laws are a fair reflection of the work done by the biotechnology industry, and the decision may draw business to Australia.

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Sally Crossing, from Cancer Voices Australia, said the Australian community had made it clear the patenting of human genes was "offensive and counter-intuitive."

"This news is not good for cancer research, especially in the promising field of targeted therapies, or for people affected by any cancer," she said.

Director of Advocacy at Cancer Council Australia, Paul Grogan, said that, if the ruling was an interpretation of the law, then the law needed to change.

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Cancer shock over BRCA1 gene patent

27.08.2014 - (idw) Max-Delbrck-Centrum fr Molekulare Medizin (MDC) Berlin-Buch

Stem cells are essential for the repair of muscle damage, but all attempts to manipulate human muscle stem cells for therapy have thus far failed. Now Dr. Andreas Marg and Prof. Simone Spuler of the Experimental and Clinical Research Center (ECRC), a joint cooperation between the Max Delbrck Center (MDC) and the Charit, have shown how this might work. They developed a method in which they did not isolate the muscle stem cells, but rather cultivated, proliferated and transplanted them along with their muscle fibers. Using this method in mice, they were able to successfully regenerate muscle tissue. Thus they have opened the door for the use of muscle stem cells to treat muscle diseases.* "Muscle stem cells, which we also refer to as satellite cells, can awaken in their stem cell niche after decades of quiescence and can then repair damaged muscle tissue," Professor Spuler explained. At the ECRC in Berlin-Buch, the neurologist heads the University Outpatient Clinic for Muscle Disorders and the Department of Muscle Sciences. She and her team are exploring the causes of muscle diseases. Evidence shows that satellite cells are active in people with severe muscle diseases such as Duchenne muscular dystrophy, a severe genetic disease in which the muscles degenerate. "But at some point, she added, the reservoir is depleted of muscle stem cells and muscle wasting cannot be stopped."

All attempts to rebuild muscle tissue by transplanting satellite cells in patients with Duchenne muscular dystrophy have failed. The transplanted cells are not viable. Furthermore, the use of other cells with potential to regenerate muscle cells has shown little success. These cells have only limited potential to regenerate muscle. But how is it possible to nevertheless use the bodys own self-renewal potential and the reconstruction potential of satellite cells?

The offer of developmental biologist Professor Carmen Birchmeier (MDC) to participate in the network project on satellite cells (SatNet) of the Federal Ministry of Education and Research pointed Professor Spuler and her co-workers in the right direction. One of the topics of the project was to elucidate why satellite cells rapidly lose their regeneration potential if they are kept in a cell culture. This led to the idea to cultivate the satellite cells together with the surrounding muscle tissue to see whether the cells, if they remain in their accustomed milieu, might possibly regenerate better.

Muscle biopsy specimens from young and old donors After due approval and written, informed consent, Professor Spuler and Dr. Marg obtained specimens of fresh thigh muscle tissue from patients between 20 and 80 years of age from neurosurgeons of Helios Klinikum Berlin-Buch, which like the MDC is located close to the ECRC.

From the biopsy specimens, Professor Spuler and her co-workers dissected more than 1000 muscle fiber fragments, each about 2-3 millimeters long. Remarkably, the researchers found the number of stem cells in the individual tissue specimens to be independent of the age of the donor and that thousands of myoblasts developed from a small number of satellite cells. After further developmental steps, these fuse into muscle fibers.

Dr. Marg: Satellite cells need to have their local milieu around them Professor Spuler and her co-workers cultivated the muscle fiber fragments with the satellite cells, initially for up to three weeks. During this time, the satellite cells increased by 20- to 50-fold, but numerous connective tissue cells also developed in these cultures. To prevent this, the researchers concurrently subjected the muscle fragments to oxygen deprivation (hypoxia) and to cooling (hypothermia) at 4 degrees Celsius. Under these conditions, only satellite cells are able to survive in their stem cell niche, in contrast to the connective tissue cells. Apparently, the satellite cells receive the proper nutrients in their own local milieu, Dr. Marg said.

First success in mice The ECRC researchers then tried out their therapy approach in mice in which muscle regeneration had been inhibited by irradiation. They grafted the muscle fragments containing the satellite cells, which following the hypothermia had been kept for two weeks in culture dishes, into the tibalis anterior muscle. The researchers found that the muscles of animals that had been treated with these fiber fragments regenerated particularly well.

Objective: to couple satellite cells with gene therapy However, a genetic muscle disease cannot be successfully treated alone by transplanting muscle fragments. Professor Spuler: The idea is therefore to equip the satellite cells additionally with a healthy gene that repairs the defective gene and then to transfect it with the aid of a non-viral gene taxi into the muscles to be treated. In a first experiment with a reporter gene in the Petri dish, Professor Spuler and her co-workers proved that this is possible in principle. The reporter gene fluoresces green when it is transfected into the satellite cell. As gene taxi the researchers use the Sleeping Beauty transposon a jumping gene that can change its position in the genome. This transposon technique was developed several years ago by Dr. Zsuzsanna Izsvk (MDC) and Dr. Zoltn Ivics (Paul Ehrlich Institute, Frankfurt) and is considered to be a very promising delivery vehicle (vector) for gene therapy.

Before the method developed by Professor Spuler and her group can be used to benefit patients, some hurdles remain to be taken. So far, the transplantation has succeeded in small mice muscles. In clinical trials, the scientists and physicians want to determine whether this technique can be used in large human thigh muscles, which may be severely altered due to a muscular disease.

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New Research Method Opens Door to Therapy with Human Muscle Stem Cells Promising Method Developed

Toronto, ON (PRWEB) August 26, 2014

Bel Marra Health, who offers high-quality, specially formulated vitamins and nutritional supplements, has reported on new research that has shown the success of the first ever biological pacemaker that could put an end to invasive surgeries.

As Bel Marra Health reports in its article, (http://www.belmarrahealth.com/heart-health/heart-patients-to-live-longer-thanks-to-new-gene-therapy/), the study was conducted by Los Angeles Cedars-Sinai Heart Institute and published in Science Translational Medicine in July. For this study, 12 pigs with heart block a condition where the electrical signal is slowed or disrupted as it moves through the heart were injected with either the single gene, called TBX18, to reprogram cells, or a fluorescent green protein acting as a placebo.

The patch of peppercorn-sized cells acted as a pacemaker for a two-week period, performing the function of a conventional one. During this same period, cardiologists looked at the average heart rate of the pigs in the morning when they ate and at night when they slept.

They found that the gene therapy was fast-acting, reprogramming enough muscle cells to effectively regulate heart rate within 24 to 48 hours. After eight days of testing, the average heart rate was much higher in the pigs that received the therapy than ones that did not.

This biological pacemaker, as its been dubbed by researchers, could be useful for certain patients, such as those who develop infections from electronic pacemakers and need to have the devices temporarily removed, or fetuses with life-threatening heart disorders who cannot have an electronic pacemaker implanted.

Spokesperson for Bel Marra Health, Dr. Victor Marchione, says, Since the early 1960s, pacemakers have been widely available, and theyve constantly improved, becoming more safe and sophisticated.

Conventional pacemakers are electronic, implanted into the chest to control an abnormal heartbeat. Electronic pacemakers restore regular function to slowing and arrhythmic hearts by using electricity to stimulate heartbeats. Thats a function usually performed by a cluster of thousands of cardiac cells that tell the heart to pump at a regular rate.

These mechanisms are lifesaving for many people with abnormal or slow heart rhythms. But they require an invasive surgery to be installed. So scientists have been waiting for the day when an implant is no longer needed by patients.

Of course, the applications of this new research are still a long way off. And the benefits of a pacemaker usually outweigh the risks. Still, pig hearts are similar to human hearts in their size and the way they work, so theres good reason to think that the new findings could translate to humans.

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Bel Marra Health Reports on the Success of New Gene Therapy for Heart Patients

Scientists have corrected an irregular heart beat in pigs using gene therapy, a finding they say may one day offer a replacement for the electronic pacemakers now used in humans.

In the study, a gene known as TBX-18 that expresses a protein normally involved in regulating heart rhythm, was injected into the hearts of seven pigs who carried a medical condition that made their heart beat too slowly or irregularly. The inserted DNA reprogrammed cells in the animals hearts in a way that corrected the beat, according to the report published in the journal Science Translational Method.

The findings, which wore off after about 10 days, offer promise for creating bridge pacemakers for those who get infections or fetuses with congenital heart block, where early surgery isnt an option, said Eduardo Marbn, a study author.

Biology takes over and creates a functioning pacemaker, said Marbn, who is also director of the Cedars-Sinai Heart Institute in Los Angeles, in a telephone interview. Its one of the penultimate steps to getting this to clinical trials in humans.

About 300,000 Americans get electronic pacemakers yearly, according to the research. The devices send electrical shocks that shock the heart to restore regular rhythm.

About 2 percent of people with pacemakers get infections during the process and need to get the devices removed, according to Marbn. The gene was injected using a catheter, and researchers are continuing to study whether the procedure can have longer-lasting results, he said.

Human trials are at least 2 to 3 years away, Marban said.

The research offers an intriguing proof of concept that should be pursued, said Nikhil Munshi, a cardiovascular researcher at the University of Texas Southwestern Medical Center in Dallas, in a telephone interview.

Its exciting, said Munshi, who wrote an accompanying commentary in the journal. Its a step in the right direction toward developing a biological pacemaker to complement existing pacemakers.

He identified other methods being studied that included the use of stem cells that help rebuild the hearts ability to operate normally, and manipulating currents in the hearts ion channels, pores within the heart that open or close in response to chemical signals, creating electrical charges.

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Biological Pacemaker That Works in Pigs Offers Promise

PUBLIC RELEASE DATE:

16-Jul-2014

Contact: Trish Reynolds reynoldsp2@mail.nih.gov 301-496-8190 NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases

Investigators have identified a gene that underlies a very rare but devastating autoinflammatory condition in children. Several existing drugs have shown therapeutic potential in laboratory studies, and one is currently being studied in children with the disease, which the researchers named STING-associated vasculopathy with onset in infancy (SAVI). The findings appeared online today in the New England Journal of Medicine. The research was done at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the National Institutes of Health.

"Not only do these discoveries have profound implications for children with SAVI, but they could have a broader impact by helping us to understand other, more common inflammatory conditions," said NIAMS Director Stephen I. Katz, M.D., Ph.D. "Diseases such as lupus share some characteristics with SAVI, so this work may lead to novel insights and possibly new treatments for these debilitating conditions, as well."

The senior author of the study, Raphaela Goldbach-Mansky, M.D., and the co-lead authors, Yin Liu, M.D., Ph.D., Adriana A. Jesus, M.D., Ph.D., and Bernadette Marrero, Ph.D., are in the NIAMS Translational Autoinflammatory Disease Section.

Autoinflammatory diseases are a class of conditions in which the immune system, seemingly unprovoked, becomes activated and triggers inflammation. Normally, the inflammatory response helps quell infections, but the prolonged inflammation that occurs in these diseases can damage the body.

In 2004, Dr. Goldbach-Mansky was called upon to advise on a patient with a baffling problema 10-year-old girl with signs of systemic inflammation, especially in the blood vessels, who had not responded to any of the medications her doctors had used to treat her.

She had blistering rashes on her fingers, toes, ears, nose and cheeks, and had lost parts of her fingers to the disease. The child also had severe scarring in her lungs and was having trouble breathing. She had shown signs of the disease as an infant and had progressively worsened. She died a few years later.

By 2010, Dr. Goldbach-Mansky had seen two other patients with the same symptoms. She suspected that all three had the same disease, and that it was caused by a genetic defect that arose in the children themselves, rather than having been inherited from their parents, who were not affected. Her hunch suggested a strategy for identifying the genetic defect. By comparing the DNA of an affected child with the DNA of the child's parents, scientists would be able to spot the differences and possibly identify the disease-causing mutation.

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NIH scientists identify gene linked to fatal inflammatory disease in children

JUDY WOODRUFF: Theres been a big disappointment in the hope to find a cure for AIDS. It involves a young child who was thought to have been cured of HIV as a baby.

JEFFREY BROWN: It was in March of last year that doctors thought they might have made a breakthrough in the goal of finding a cure for AIDS, treating a baby girl in Mississippi with early and unusually aggressive drug therapy.

The mother had HIV and had not been treated during pregnancy. But the girl was treated within 30 hours of her birth and was free of the virus for two years. Doctors allowed her to stay off therapy and, still, there were no signs of HIV returning.

But, yesterday, officials announced that the girl, now almost 4, had tested positive for HIV during a follow-up visit last week.

Dr. Anthony Fauci of the National Institute of Allergy and Infectious Diseases joins me now.

And welcome back to you.

This is something that you and I talked about when the news came out last year. So, remind us first why this seemed so hopeful, how this early and aggressive treatment promised such a difference.

DR. ANTHONY FAUCI, National Institute of Allergy and Infectious Diseases: Well, it promised such a difference because what happened with this particular baby was an unusual situation, that the baby had been on therapy, this aggressive form that you correctly described, for about 18 months, but then was lost to follow-up.

And the mother discontinued the therapy because she just dropped out of the out of the health care system, came back five months later, and when the physicians examined the baby, they found out that they couldnt find the virus anywhere by the standard methods of looking for virus, no virus in the plasma and no virus in the cells in the blood.

So they decided that this possibly could have been a cure related to the fact that the baby was treated very early, as you mentioned, within 30 hours, and aggressively. As it turned out, they followed the baby very, very carefully, and over a period of 27 months without any therapy at all, there was no indication at all of any virus in the baby. There was no plasma viremia, as we say, namely virus in the blood.

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Newswise LA JOLLA-The ability to switch out one gene for another in a line of living stem cells has only crossed from science fiction to reality within this decade. As with any new technology, it brings with it both promise--the hope of fixing disease-causing genes in humans, for example--as well as questions and safety concerns. Now, Salk scientists have put one of those concerns to rest: using gene-editing techniques on stem cells doesn't increase the overall occurrence of mutations in the cells. The new results were published July 3 in the journal Cell Stem Cell.

"The ability to precisely modify the DNA of stem cells has greatly accelerated research on human diseases and cell therapy," says senior author Juan Carlos Izpisua Belmonte, professor in Salk's Gene Expression Laboratory. "To successfully translate this technology into the clinic, we first need to scrutinize the safety of these modified stem cells, such as their genome stability and mutational load."

When scientists want to change the sequence of a stretch of DNA inside cells--either for research purposes or to fix a genetic mutation for therapeutic purposes--they have their choice of two methods. They can use an engineered virus to deliver the new gene to a cell; the cell then integrates the new DNA sequence in place of the old one. Or scientists can use what's known as custom targeted nucleases, such as TALEN proteins, which cut DNA at any desired location. Researchers can use the proteins to cut a gene they want to replace, then add a new gene to the mix. The cell's natural repair mechanisms will paste the new gene in place.

Previously, Belmonte's lab had pioneered the use of modified viruses, called helper-dependent adenoviral vectors (HDAdVs) to correct the gene mutation that causes sickle cell disease, one of the most severe blood diseases in the world. He and his collaborators used HDAdVs to replace the mutated gene in a line of stem cells with a mutant-free version, creating stem cells that could theoretically be infused into patients' bone marrow so that their bodies create healthy blood cells.

Before such technologies are applied to humans, though, researchers like Belmonte wanted to know whether there were risks of editing the genes in stem cells. Even though both common gene-editing techniques have been shown to be accurate at altering the right stretch of DNA, scientists worried that the process could make the cells more unstable and prone to mutations in unrelated genes--such as those that could cause cancer.

"As cells are being reprogrammed into stem cells, they tend to accumulate many mutations," says Mo Li, a postdoctoral fellow in Belmonte's lab and an author of the new paper. "So people naturally worry that any process you perform with these cells in vitro--including gene editing--might generate even more mutations."

To find out whether this was the case, Belmonte's group, in collaboration with BGI and the Institute of Biophysics, Chinese Academy of Sciences in China, turned to a line of stem cells containing the mutated gene that causes sickle cell disease. They edited the genes of some cells using one of two HDAdV designs, edited others using one of two TALEN proteins, and kept the rest of the cells in culture without editing them. Then, they fully sequenced the entire genome of each cell from the four edits and control experiment.

While all of the cells gained a low level of random gene mutations during the experiments, the cells that had undergone gene-editing--whether through HDAdV- or TALEN-based approaches--had no more mutations than the cells kept in culture.

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7-Jul-2014

Contact: Steve Graff stephen.graff@uphs.upenn.edu 215-349-5653 University of Pennsylvania School of Medicine

PHILADELPHIA A University of Pennsylvania-developed personalized immunotherapy has been awarded the U.S. Food and Drug Administration's Breakthrough Therapy designation for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL). The investigational therapy, known as CTL019, is the first personalized cellular therapy for the treatment of cancer to receive this important classification.

In early-stage clinical trials at the Hospital of the University of Pennsylvania and the Children's Hospital of Philadelphia, 89 percent of ALL patients who were not responding to conventional therapies went into complete remission after receiving CTL019.

"Our early findings reveal tremendous promise for a desperate group of patients, many of whom have been able to return to their normal lives at school and work after receiving this new, personalized immunotherapy," said the Penn research team's leader, Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine and director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania. "Receiving the FDA's Breakthrough Designation is an essential step in our work with Novartis to expand this therapy to patients across the world who desperately need new options to help them fight this disease."

The FDA's Breakthrough Therapy designation, created in 2012, is intended to expedite the development and review of new medicines both drugs and biologic agents that treat serious or life-threatening conditions, if the therapy has demonstrated substantial improvement over available therapies. The FDA has previously granted Breakthrough Therapy to only four other biologic agents.

In August 2012, Penn announced an exclusive global research and licensing agreement with Novartis to further study, develop and commercialize personalized chimeric antigen receptor (CAR) T cell therapies for the treatment of cancers. Trials employing CTL019 began in the summer of 2010 in patients with relapsed and refractory chronic lymphocytic leukemia (CLL), and are now underway for adult and pediatric patients with ALL, and patients with non-Hodgkin lymphoma and myeloma. Penn and Novartis are also investigating the next generation of CAR therapies, with trials for mesothelioma, ovarian, breast and pancreatic cancer now in early stages.

During the 2013 annual meeting of the American Society of Hematology, the Penn research team announced study results of the first 27 ALL patients(22 children and five adults) treated with CTL019: 89 percent of the patients had a complete response to the therapy. The first pediatric ALL patient to receive the Penn therapy celebrated the second anniversary of her cancer remission in May, and the first adult patient remains in remission one year after receiving the therapy.

The investigational treatment pioneered by the Penn team begins by removing patients' T cells via an apheresis process similar to blood donation, then genetically reprogramming them in Penn's Clinical Cell and Vaccine Production Facility. After being infused back into patients' bodies, these newly built "hunter" cells both multiply and attack, targeting tumor cells that express a protein called CD19. Tests reveal that the army of hunter cells can grow to more than 10,000 new cells for each single engineered cell patients receive.

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Penn's immunotherapy for leukemia receives FDA's Breakthrough Therapy designation

Gene Therapy Program > Home

Providing a foundation for basic research necessary to assure the success of gene therapy.

Given all the developments in molecular genetics, the isolation and cloning of genes is now a relatively common procedure. Research now centers on somatic gene therapy, referring to the techniques used to insert a functioning gene into the somatic (non-reproductive) cells of a patient to correct an inborn genetic error or to provide a new function to the cell. Having individual genes available opens the way for gene therapy to take place. And yet, after an initial period of about six years of preclinical work and another thirteen years involving clinical trials, effective gene delivery still remains one of the central challenges in the field.

The Gene Therapy Program of the University of Pennsylvania comprises basic scientific research and core lab research services. Our focus is on developing effective gene vectors derived from recombinant viruses. Much of our current effort is in the development of new adeno-associated virus (AAV) vectors, although some of our research involves both adenoviruses and lentiviruses. Several basic science core laboratories work together to support the development of new vectors.

Contact: Gene Therapy Program Suite 2000, Translational Research Laboratories (TRL) 125 S. 31st Street Philadelphia, PA 19104-3403 Phone: 215-898-0226 Fax: 215-494-5444 GTP@mail.med.upenn.edu

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University of Pennsylvania || Gene Therapy Program

Genetic disorders have been plaguing people for ages and causing fatalities. However, with new information and research, and something called gene therapy, hope now exists for these unfortunate individuals. Gene therapy is a technique for correcting defective genes responsible for disease development. It has been around for a while now and is getting more advanced with time. Experimentation is an ongoing process with gene therapy. Ethical issues are something that has been accompanying the procedure since it has been used. New facts on gene therapy continue to be uncovered as we speak. To start off, an overview of why people need gene therapy should be covered. Each of us carries about half a dozen defective genes. However, we remain ignorant to this fact unless we are among the millions of people who have a genetic disorder. About one in ten people has, or will develop some time later in life, an inherited genetic abnormality. And approximately two thousand eight hundred specific conditions are known to be caused by defects in just one of the patients genes. Some single gene disorders are pretty common, such as cystic fibrosis. Most people do not suffer harmful effects from our defective genes because we carry two copies of nearly all genes. One is inherited from our mother and the other from our father. The only exceptions to this rule are the genes found on the male sex chromosomes. Males have one X and one Y chromosome, the former from the mother and the latter from the father. So each cell has only one copy of the genes on these chromosomes. In the majority of cases, one normal gene is enough to avoid all the symptoms of disease. If the gene that may be harmful is recessive, then its normal counterpart will carry out all the tasks assigned to both. A disease will develop only if someone inherits two copies of the recessive gene from their parents. (Web source #3) In other terms, if the gene is dominant, it alone can produce the disease, even if the counterpart is normal. Finally, there are the X chromosome-linked genetic diseases. Because males have only one copy of the genes from this chromosome, there are no others available to fulfill the defective genes function. Hemophilia is a common result of this. To continue, how gene therapy works, should be explained. There are several different approaches scientists may use to correct faulty genes with therapy. One method is that a normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This happens to be the most common approach utilized. Another method is an abnormal gene could be exchanged for a normal gene through homologous recombination. (Web source #2) Also, the abnormal gene could be repaired through selective reverse mutation, which would restore the gene to its original function. Finally, the regulation or degree to which a gene is turned on or off, of a particular gene could be altered. In the majority of gene therapy studies, a normal gene is inserted into the genome to replace an abnormal, disease-causing gene. A carrier molecule called a vector must be used to deliver the therapeutic gene to the patients target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease causing agents and insert therapeutic genes. There are a series of different types of viruses used as gene therapy vectors. One is adenoviruses. These are a class of viruses with double-stranded DNA genomes that cause respiratory, intestinal and eye infections in humans. The virus that causes the common cold is an adenovirus. Another type is retroviruses. This is a class that can create double-stranded DNA copies of their RNA genomes. These copies of its genome can be integrated into the chromosomes of host cells. HIV is a retrovirus. A third type is adeno-associated viruses, which are a class of small, single-stranded DNA viruses that can insert their genetic material at a specific site on chromosome nineteen. One more type is herpes simplex viruses. This is a class of double-stranded DNA viruses that infect particular cell type, neurons. Herpes simplex virus type one is a common human pathogen that causes cold sores. Besides, virus mediated gene-delivery systems, there are several nonviral options for gene delivery. The simplest method is the direct introduction of therapeutic DNA into target cells. Another nonviral approach involves the creation of an artificial lipid sphere with an aqueous core. (Web source #2) Today, the current status of gene therapy is unofficial. In fact the Food and Drug Administration has not yet approved any human gene therapy product for sale. Gene therapy is currently experimental and has not quite been proven completely successful in clinical trials. For example, little forward progress has been achieved since the first gene therapy experimental trial began in 1990. A major setback was suffered in 1999 when the death of an 18-year-old named Jesses Gelsinger occurred. He was participating in a clinical trial for ornithine transcarboxylase deficiency, or OTCD. Jesses death is believed to have been triggered by a severe immune response to the adenovirus carrier. FDAs Biological Response Modifiers Advisory Committee met at the end of February 2003 to discuss possible measures that could allow a number of retroviral gene therapy trials for treatment of life-threatening diseases to proceed with appropriate guide lines. (Web source #2) There are certain factors that have kept gene therapy from becoming an effective treatment for genetic diseases. One is the short-lived nature of gene therapy. Before gene therapy can become a permanent cure for any condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be long-lived and stable. (Web source #2) Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells prevent gene therapy from achieving any long-term benefits. Patients will have to undergo numerous rounds of gene therapy. Another factor is immune response. Anytime a foreign object is introduced into human tissues, the immune system is designed to attack the invader. The risk of stimulating the immune system in a way that reduces gene therapy effectiveness is always a potential risk. (Web source #2) Furthermore, the immune systems enhanced response to invaders it has seen before makes it difficult for gene therapy to be repeated in patients. Another setback is problems with viral vectors. Viruses, while the carrier of choice in most gene therapy studies, present a variety of potential problems to the patient which include toxicity, immune and inflammatory responses, and gene control and targeting issues. In addition, there is always the fear that the viral vector, once inside the patient, may recover its ability to cause disease. One more problem is multigene disorders. Conditions or disorders that arise from mutations in a single gene are the best candidates for gene therapy. Unfortunately, some of the most commonly occurring disorders, such as heart disease, high blood pressure, arthritis, and diabetes are caused by the combined effects of variations in many genes. Multigene or multifactoral disorders such as these would be especially difficult to treat effectively using gene therapy. (Web source #2) There is current progress being made with gene therapy, however. In reference to the Human Genome Project, the mapping out of the makeup of all human genes, another gene has recently been successfully analyzed. Scientists in Britain have finished work on human chromosome six which contains genes linked to the bodys immune response against bacteria and viruses. Comprising nearly six percent of the entire human genome, it is the largest of the twenty three pairs of human chromosomes completed so far. (Web source #1) Chromosome six is very concentrated in immune genes. These are the genes that give us protection from pathogens. With this new knowledge, there is now a boost for organ transplants These immune genes are particularly important for transplant medicine because doctors will be better able to match donor organs and recipients. About 1,557 genes on chromosome six are thought to be functional. Roughly 130 genes have been identified that somehow cause or predispose humans to certain diseases. (Web source #1) Knowing all of this information will help scientists in the future to learn how to altar these genes in order to prevent these diseases from taking place. In addition, ethical issues are coincidently always brought up with gene therapy. A common question is what is normal and what is a disability or disorder, and who decides? This is a very pressing question that will remain as long as gene therapy does. Whether it is the parents, the governments or the patients right to decide what goes forth is a tough concept. Another ethical issue is preliminary attempts at gene therapy are extremely expensive. Who will have access to these therapies and who will pay for their use? This is a very difficult question to derive an answer to. A fairness issue arises when only the very wealthy are the ones able to receive clinical trials of the medicines used in gene therapy. Also, the question of are disabilities diseases and do they need to be cured or prevented comes up. I believe it is up to the individual person to decide whether or not treatment is necessary but there may be exceptions. One more popular issue brought up with gene therapy is does searching for a cure demean the lives of individuals presently affected by disabilities? This is a question that can only be solved through personal research with actual patients or individuals. (Web source #2) In conclusion, although currently an unofficial form of treatment, gene therapy holds promise for the future. The Human Genome continues to be mapped and the more that is analyzed, the further research can be taken. Progress is being made, and despite setbacks along the way, the achievements seem to outweigh the failures. Progress will hopefully continue so that genetic disorders may someday be unheard of with the help of gene therapy and a better understanding of human genetics.

Works Cited 1.http://my.aol.com/news.news_stories 2.http://www.ornl.gov/techresources/humangenome/medicine/genetherapy.html 3. http://www.accessexcellence.org/AB/IWT/gene_therapy.overview.html

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Dallas, TX (PRWEB) July 02, 2014

Cell Culture Market by Equipment (Bioreactor, Incubator, Centrifuge), by Reagent (Media, Sera, Growth Factors, Serum Free Media), by Application (Cancer Research, Gene Therapy, Drug Development, Vaccine Production, Toxicity Testing) Global Forecast to 2018 research report segments the cell culture market into two distinct market segments, namely, the cell culture equipment market and the cell culture media, sera, and reagents market. The segments of the cell culture equipment market included in this report are bio-safety cabinets, consumables, lab equipment, sterilization equipment, and storage equipment. The lab equipment segment consists of four subsegments, namely, cell counters, centrifuges, fermentors and bioreactors, and incubators. The storage equipment segment consists of two subsegments, namely, cryogenic storage and refrigerators and freezers.

The segments of the cell culture media, sera, and reagents market included in this report (http://www.lifescienceindustryresearch.com/cell-culture-market-by-equipment-bioreactor-incubator-centrifuge-by-reagent-media-sera-growth-factors-serum-free-media-by-application-cancer-research-gene-therapy-drug-development-vacci.html ) are contamination detection kits, cryoprotective agents, lab reagents, media, serum, and other reagents. The lab reagents segment consists of four subsegments, namely, balanced salt solutions, buffers and chemicals, cell dissociation reagents, and supplements and growth factors. The media segment consists of three subsegments, namely, basal media, reduced serum media, and serum-free media.

Cell culture has its applications in a large number of fields. On the basis of application, the report is segmented into biopharmaceutical production, cancer research, drug screening and development, gene therapy, tissue culture and engineering, toxicity testing, vaccine production, and other applications. The geographic segments included in this report are North America, Europe, Asia-Pacific, and Rest of the World.

The key drivers for this market are rapid increase in the demand for biopharmaceuticals and increased adoption of single-use technology. According to IMS Health, biopharmaceutical is expected to be the fastest growing pharmaceutical product segment by2017. Biopharmaceuticals are pharmaceutical products isolated from living organisms. Cell culture is one of the most important and widely used techniques for biopharmaceutical production. It was also the largest application segment of the cell culture market in 2013. Growth in the biopharmaceutical market will drive the growth of the cell culture market.

Companies profiled in this cell culture market research report include Becton, Dickinson and Company, Corning, Inc., Eppendorf AG, Lonza Group Ltd., Merck Kgaa, Sartorius AG, Sigma-Aldrich Corporation, Thermo Fisher Scientific, Inc. and Promocell GMBH. Order a copy of this report at http://www.lifescienceindustryresearch.com/purchase?rname=15929 .

Secondary information was used to identify the overall revenues, geographic reach, and product portfolios of the market players. Estimates of their cell culture segment revenues were validated through primary interviews. Primary interviews with key opinion leaders were also used to determine percentage shares of each subsegment and the relative differences in the growth rates.

The report provides qualitative insights about key market shares, growth rates, and market drivers for all important subsegments. It maps the market sizes and growth rates of each subsegment and identifies the segments poised for rapid growth in each of the geographic segments. The report also includes company profiles of the market leaders. These company profiles include financial performances, product portfolios, and market developments for each company. The report also provides a competitive landscape of the cell culture market. The competitive landscape covers the growth strategies adopted by the industry players over the last three years. It also includes analysis of industry developments like mergers and acquisitions, agreements and partnerships, and new product launches.

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The report will enable both established firms and new entrants to gauge the pulse of the market and help them make important strategic growth decisions. The report provides insights on the following:

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10.71% CAGR for Cell Culture Market Forecast to 2018 in a New Global Research Report Available at ...

DALLAS, July 1, 2014 /PRNewswire/ --

The report "Cell Culture Marketby Equipment (Bioreactor, Incubator, Centrifuge), by Reagent (Media, Sera, Growth Factors, Serum Free Media), by Application (Cancer Research, Gene Therapy, Drug Development, Vaccine Production, Toxicity Testing) - Global Forecast to 2018" published by MarketsandMarkets, provides a detailed overview of the major drivers, restraints, challenges, opportunities, current market trends, and strategies impacting the global Cell Culture Market along with the estimates and forecasts of the revenue and market share analysis.

Browse 91 market data tables and 13 figures spread through 210 Pages and in-depth TOC on "Cell Culture Market"

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The global Cell Culture Market was valued at an estimated $14,772 million in 2013. This market is expected to grow at a CAGR of 10.71% between 2013 and 2018, to reach $24,574 million in 2018.

The Cell Culture Market is segmented on the basis of cell culture equipment and cell culture media, sera, and reagents. Each of these two market segments is further divided into multiple product segments and subsegments. The cell culture equipment market consists of five segments, namely, bio-safety cabinets, consumables, lab equipment, sterilization equipment, and storage equipment. Of these, the lab equipment product segment had the largest share of the cell culture equipment market in 2013, whereas the consumables product segment is expected to grow at the highest CAGR between 2013 and 2018. The subsegments of the lab equipment segment are cell counters, centrifuges fermentors & bioreactors, and incubators. The subsegments of the storage equipment segments are cryogenic storage and refrigerators and freezers.

The cell culture media, sera, and reagents market consists of six segments, namely, contamination detection kits, cryoprotective agents, lab reagents, media, serum, and other reagents. Of these, the serum product segment had the largest share of the cell culture media, sera, and reagents market in 2013, whereas the media product segment is expected to grow at the highest CAGR between 2013 and 2018. The subsegments of the lab reagents segment are balanced salt solutions, buffers and chemicals, cell dissociation reagents, and supplements and growth factors. The subsegments of the media segment are basal media, reduced serum media, and serum-free media.

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The application segments included in this report are biopharmaceutical production, cancer research, drug screening and development, gene therapy, tissue culture and engineering, toxicity testing, vaccine production, and other applications. The biopharmaceutical production application segment had the largest share of the cell culture equipment market in 2013, whereas the vaccine production application segment is expected to grow at the highest CAGR between 2013 and 2018. The geographic segments included in this report are North America, Europe, Asia-Pacific, and Rest of the World.

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Cell Culture Market worth $24,574 Million by 2018

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19-Jun-2014

Contact: Sophie Mohin Smohin@liebertpub.com 914-740-2254 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, June 19, 2014An innovative system using automated 3D printing technology and advanced digital tools to create customized, prefabricated ceramic building blocks, called PolyBricks, is enabling the construction of mortar-less brick building assemblies at much greater scales than was previously possible. The new techniques that use 3D printers to produce modular ceramic bricks from a single material that then interlock and assemble easily into larger units for architectural applications are described in an article in 3D Printing and Additive Manufacturing (3DP), a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free online on the 3D Printing and Additive Manufacturing website.

Jenny Sabin, Martin Miller, Nicholas Cassab, and Andrew Lucia, of Sabin Design Lab, Cornell University (Ithaca, NY) and Jenny Sabin Studio (Philadelphia, PA), provide a detailed description of the computational design techniques they developed for the digital fabrication and production of ceramic PolyBrick components. The authors explain how they used available 3D printing technology to produce mass customized components in the article PolyBrick: Variegated Additive Ceramic Component Manufacturing (ACCM)"

"This work offers an exciting new alternative approach for 3D printing at architectural scales, without requiring the large infrastructure that most current methods require. It could open the door to many new applications" says Editor-in-Chief Hod Lipson, PhD, Director of Cornell University's Creative Machines Lab at the Sibley School of Mechanical and Aerospace Engineering, Ithaca, NY.

###

About the Journal

3D Printing and Additive Manufacturing (3DP) is a peer-reviewed journal published quarterly in print and online. The Journal facilitates and supports the efforts of engineers, software developers, architects, lawyers, Deans and academic chairpersons of engineering and business schools, technology transfer specialists, chief research officers and vice presidents of research in government, industry, and academia, medical professionals, venture capitalists, and entrepreneurs. Spanning a broad array of disciplines focusing on novel 3D printing and rapid prototyping technologies, policies, and innovations, the Journal brings together the community to address the challenges and discover new breakthroughs and trends living within this groundbreaking technology. Complete tables of content and a sample issue may be viewed at the 3D Printing and Additive Manufacturing (3DP) website.

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New digital fabrication technique creates interlocking 3D-printed ceramic PolyBricks

New jab would act like a one-off 'vaccination' against heart attack It works by 'knocking out' gene which raises levels of cholesterol in blood Scientists say it could cut the risk of heart attack by 90 per cent Still in development, but after tests it could be available in a decade

By Richard Spillett

Published: 01:06 EST, 11 June 2014 | Updated: 02:58 EST, 11 June 2014

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A single injection that slashes the risk of heart attack by up to 90% could be available within 10 years, scientists say.

The jab uses DNA-editing technology to 'knock out' a liver gene believed to raise levels of cholesterol in the bloodstream.

The one-off treatment has already been tested successfully on mice, reducing the blood concentrations of cholesterol in the animals by 35 to 40 per cent within days.

A new injection which cuts the risk of heart attack could be used as 'a vaccination' within 10 years

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A single injection to stop heart attacks? Jab that slashes risk by 90% by 'knocking out' gene linked with cholesterol ...

In January, Jordan passed a law to control research and therapy using human stem cells derived from embryos the first such regulation in the Arab and Islamic region. I was part of the group headed by Abdalla Awidi Abbadi, director of the Cell Therapy Center at the University of Jordan in Amman, that initiated the call for the law and later drafted it. Stem-cell research is a hot topic for Jordan because of the kingdoms status as a health-care hub that draws patients from abroad. It is already one of few countries in the Middle East with regulations for protecting people who participate in clinical trials. This latest law should serve as an example to other countries in the region.

The new rules ban private companies from using human embryonic stem (ES) cells in research or therapies. Such work will be allowed only in government organizations or publicly funded academic institutions in Jordan, which have higher levels of transparency than private firms and are supervised by the health ministry and a specialized committee. The law also bans payment for donations of stem cells and eggs, and says that modified and manipulated cells are not to be used for human reproduction. There is no current research on human ES cells in Jordan; this is a pre-emptive step.

Much of the controversy and disagreement over work on stem cells worldwide arises from the different views of the major religions on the earliest stages of life. Although the use of human ES cells is opposed by the Roman Catholic Church and some Protestant denominations, it is generally supported by the Jewish community and accepted in many Muslim countries. There is no consensus on when human embryonic life begins, but the majority of Muslim scholars consider it to start 40120 days after conception and therefore hold the view that a fertilized egg up to 5days old has no soul it is not human life but biological life. So for many, there is no ethical problem in the Islamic faith with using an early embryo to produce stem cells.

All our discussions in Jordan have concluded that stem-cell research is permissible in Islam.

Such conclusions are not easy to reach. Many Muslim countries consider legislation and bioethics principles to be based on three pillars of Islamic law. The first is the Quran. The second is Sunnah, or the legislative decisions of the Prophet Muhammad. The third is ijmaa the consensus of Muslim scholars and ijtihad, the concept that every adequately qualified scholar has the right to independently solve problems. On the basis of these pillars, Iran, Saudi Arabia and Tunisia have drawn up guidelines on stem-cell research, but they are not legally binding.

Jordans stem-cell law is the product of years of discussions by committees comprising scientists, physicians, Arabic-language experts, lawyers and Muslim and Christian theologians. The issues that arose confusion between stem cells and embryonic stem cells, for instance were discussed and resolved. We consulted with both the National Committee for Science and Technology Ethics and the education ministry. The final law was approved by the council of Muslim scholars, the Majlis Al-Iftaa.

The council agreed with a 2003 decision (fatwa) by Muslim scholars that allows the use of human ES cells from permissible sources including legally produced excess fertilized eggs from invitro fertilization. The decision to ban private companies from using these cells was driven by concerns that the work would encourage termination of pregnancies, which is illegal in Jordan unless the mothers life or health is at risk. The council was clear that the new law must forbid human reproductive cloning and should not allow embryos to be created from the sperm and eggs of unmarried couples.

The distinction drawn between the various sources of stem cells earlier in the discussion process allowed the Majlis Al-Iftaa to take a more permissive approach to techniques using stem cells that are not derived from human embryos. For example, somatic-cell nuclear transfer (in which a patients DNA is transplanted into an unfertilized human egg that has no nucleus) and induced pluripotent stem cells, which are made from adult cells, can be worked on by the private sector under the new rules.

The therapeutic use of bone-marrow transplantation including transplants of blood-forming stem cells is well established in Jordan. Such procedures are already regulated by existing laws on medical practice, so the new law makes a clear distinction between these techniques and human ES-cell therapy.

The legislation not only covers all current aspects of stem-cell research and use, but also leaves room for later modification. It mandates the creation of a national committee that, among other things, will take responsibility for laying out specific regulations for stem-cell banking in accordance with international standards.

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Jordans stem-cell law can guide the Middle East

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Newswise New York, New York Research to Prevent Blindness (RPB), the leading eye research foundation, is providing $500,000 to accelerate the development of treatments for Retinitis Pigmentosa (RP) -- a family of retinal diseases that progressively create extreme tunnel vision, loss of night vision and leave affected patients legally blind by the age of 40. The funds are being awarded to five nationally prominent RP investigators.

"Recent breakthroughs in gene therapy that reverse vision loss in certain RP patients have given us all hope that treatments for other forms of RP are within reach," says Diane S. Swift, Chair of RPBs Board of Trustees. While we have funded RP research for decades, the restoration of sight in these young people is so dramatic, and the developments in gene therapy so promising, that we felt focused investments in key RP researchers at this crucial time could hasten life-changing treatments."

The five recipients of the RPB Nelson Trust Award for Retinitis Pigmentosa were selected after rigorous review by multiple RPB advisory panels comprised of outstanding scientists and chairs of departments of ophthalmology from across the country. The award was originally conceived to produce at least one grant annually for the next eight to nine years, says Brian F. Hofland, PhD, RPB President, but we realized that the exceptional pool of applicants truly some of the nation's foremost retina investigators created an opportunity for us to make an immediate impact. It's an unusual application of endowed funds for us, but we are seizing the moment to push this promising science toward the goal."

Each of the RPB Nelson Trust Award awardees will receive $100K in flexible support to pursue novel projects.

Vadim Arshavsky, PhD, Duke University has made seminal discoveries related to the process by which light is converted into electrical signals in the photosensitive cells of the retina. He will focus on a newly discovered common risk factor for RP and the development of a possible treatment for multiple forms of RP that arise from different mutations. (Visit the Arshavsky Lab.) (More about Dr. Arshavsky.)

Wolfgang Baehr, PhD, University of Utah is frequently described as the leading retinal biochemist and molecular biologist of our era. His multifaceted studies will apply cutting edge genetic tools to address broad mechanisms shared by many forms of RP, creating new models for therapeutic study and developing new treatments. (Visit the Baehr Lab.) (More about Dr. Baehr.)

David M. Gamm, MD, PhD, University of Wisconsin is one of the undisputed leaders in stem cell research relevant to age-related macular degeneration. He intends to develop a rod-replacement-therapy for RP using human induced pluripotent stem cells, which can be created from a patients own skin cells. (More on Dr. Gamm and stem cell research video.)

Eric A. Pierce, MD, PhD, Harvard University is one of the leading investigators in RP research and has recently identified a possible genetic cause of RP. Dr. Pierces studies will provide a path to developing a gene therapy for diseases caused by mutations in this gene. (Visit the Pierce lab.)

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Top Retinitis Pigmentosa Scientists Earn Awards for Further Research

Dr. Mark Penn, founder and CMO of Juventas Therapeutics

While the optimal treatment for heart failure was provided to a group of patients, they were still having symptoms. However when a new drug therapy based in regenerative medicine was given to these patients they showed clinically meaningful improvements in end systolic volumes, end diastolic volumes, ejection fraction and NTproBNP levels.

The drug, produced by Cleveland, Ohio-based Juventas Therapeutics, called JVS-100, is a non-viral gene therapy that expresses SDF-1 and promotes endogenous stem cell repair of the heart in patients with severe heart failure.

"What was remarkable about the improvement is that this drug was given to patients who had heart failures stemming from heart attacks that occurred - on average- about eleven years ago," said Dr. Mark Penn, founder and CMO of Juventas Therapeutics, and director of Cardiovascular Research at Summa Health System in Akron, Ohio.

Penn presented phase II clinical data last month at the European Society of Cardiology- Heart Failure Congress in Athens, Greece.

The field of regenerative medicine, which is the process of replacing, engineering or regenerating human cells, tissues or organs to restore or establish normal function, has come a long way. Penn explained that 14 years ago that stem cell based repairs lacked molecular signals that orchestrated the repairs. "Doing research we asked what drives stem cell repair? We saw that newly injured tissue was sending some signal to ask for it to be repaired. And in 2000 we discovered SDF-1 could aid that signal. Now our theory is validated that the gene therapy is a key factor for recruiting stem cells to the site for any injured tissue."

The therapy showed an 80% chance of a significant decrease in mortality for high risk heart failure patients.

With this success, Penn hopes to start next summer of 2015 on a larger trial of 300-400 patients. When that trial is initiated the company will have to move from manufacturing the drug for clinic studies to a commercial scale. Once the drug has regulatory approval the company will decide where to manufacture.

When asked about the reason for the success of the company, Penn says that "the company has always been driven by data. We had no preconceived ideas that this should work. We designed good trials, looked at the data and that told us where to go."

With regard to the financial side of the business, the company has worked with venture investors. And they have formed partnerships. The company has on-going collaborative research programs with Cleveland Clinic, Center for Stem Cell & Regenerative Medicine, Global Cardiovascular Innovation Center and Summa Health System.

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New Drug-Based Approach to Regenerative Medicine for Heart Failure

Family Centers President Bob Arnold and Barbara Netter.

At its annual luncheon in Darien on April 23, the Center for HOPE recognized Greenwich resident Barbara Netter for her many contributions toward the advancement of gene therapy-based cancer treatments.

As the President of the Alliance for Cancer Gene Therapy (ACGT) a nonprofit organization she founded with her late husband, Edward, in 2001 Ms. Netter has raised nearly $22.5 million for numerous gene therapy research initiatives around the world. Over the years, ACGTs support has been instrumental in launching 17 human clinical trials addressing lung, ovarian, prostate and breast cancer, as well as lymphoma and leukemia.

For her work providing millions of cancer patients with a renewed sense of optimism, Ms. Netter was presented with the Ray of HOPE Award. The Ray of HOPE is the Center for HOPEs highest honor that highlights a member of the community whose efforts assist those coping with a loss, critical illness or life-altering circumstance.

We are delighted to recognize Barbara with a Ray of HOPE Award, shinning the light on her body of work in the area of critical illness and bereavement, said Bob Arnold, Family Centers president. Barbara truly inspires us, as her bountiful efforts continue to fill our world with hope and compassion.

In addition to her work with ACGT, Ms. Netter has been involved for many years with The Den for Grieving Kids a Family Centers program providing support to children and families who have lost a loved one.

The Center for HOPE Luncheon also featured a keynote address from Huffington Post founder and editor-in-chief Arianna Huffington.

All proceeds from the event benefited the Center for HOPE and The Den for Grieving Kids.

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Center for HOPE honors Netter