Archive for the ‘Gene Therapy Research’ Category

Laking pasasalamat ni Albert Martinez at ng kanyang pamilya na cleared na sa breast cancer ang asawa niyang si Liezl Martinez.

Kuwento ni Albert sa PEP.ph (Philippine Entertainment Portal), sa tulong daw ng stem cell therapy ay naging cancer-free ang asawa.

Ok naman, so far with Gods blessing,. Its all cleared. So, were very, very happy.

Dealing with cancer is a struggle from day one, and Im really thankful that theres such a thing as stem cell na naging solusyon sa recovery ni Liezl."

EXTENSIVE RESEARCH. Ayon kay Albert ay pinag-aralan niya ang lahat ng puwedeng maging solusyon sa sakit ng asawa.

Pero sa huli, ang stem cell pa rin ang naging solusyon.

Marami akong pinagdaanan kasi ni-research ko lahat yan.

Kailangang alamin mo kung ano ba ang mga dapat gawin. Lahat pinag-aralan natin

And nag-end up talaga, na ang pinakamaganda is stem cell.

I took the risk, researched on the possibilities of doing it, availability niya.

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Albert Martinez claims stem cell therapy cleared wife Liezl Martinez of breast cancer

06-09-2012 07:59 Frank Sinatra, Stephen Hawking, Marie Curie and Stephen Fry all owe their blue eyes to a genetic mutation that likely occurred between 6000 and 10000 years ago, researchers say. Scientists believe they have tracked down the cause of the eye colour of all blue-eyed humans on the planet today. WHERE IS THE BLACK SEA - TODAY'S INTRO BY TheGreatAussieMate - CHECK OUT THE FEATURED YOUTUBERS WEBSITE @ Support Me On GOOGLE+ - FACEBOOKFANPAGE - TWITTER - BLOGSPOT - ADD ME ON SKYPE dibberdelboy When you reguest contacts link please provide youtube name or i wont add you DIBBERS OTHER CHANNELS Intro & Outro Music By

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Blue Eyes Result Of Ancient Genetic Mutation - Video

06-09-2012 03:56 The GENCODE Consortium has found 50% more genes than previously thought.Among their discoveries, the team describe more than 10000 novel genes, identify genes that have 'died' and others that are being resurrected. The GENCODE Consortium has developed a reference gene catalogue that has been the underpinning of the larger ENCODE Project, a large collaborative project aimed to find and describe all functional elements of our genome.The GENCODE Consortium is part of the ENCODE Project that on 5 September 2012, publishes 30 research papers describing findings from their nearly decade-long effort

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GENCODE: An encyclopaedia of genes and gene variants - Video

By Amanda Gardner HealthDay Reporter Latest Prevention & Wellness News

WEDNESDAY, Sept. 5 (HealthDay News) -- Scientists' understanding of what causes human disease -- genetically speaking -- just got a bit clearer and infinitely more complicated all at the same time.

A study appearing online Sept. 5 in the journal Science provides a blueprint not only of genes that are involved in different disorders, but also of the "switches" that control those genes and how these two elements interact.

The "map" could substantially alter how scientists approach the genetics of diseases and, eventually, how treatments and cures are devised, the researchers said.

"This is a paradigm shift in terms of how we look at the genetic basis for disease," said study senior author Dr. John Stamatoyannopoulos, an associate professor in the departments of genome sciences and medicine at the University of Washington in Seattle. "I think it's going to change considerably how people use the genome to identify targets for pharmaceuticals."

Previous genetics research had focused mainly on finding a specific gene or gene variant for a particular disease, the conventional wisdom being that specific variants in that gene would affect protein sequences, and the altered protein sequences would determine if a person was healthy or if he or she developed a disease.

The problem is that many of these studies fingered regions of the human genome that don't actually contain genes, Stamatoyannopoulos explained. In fact, genes constitute only 2 percent of the human genome.

"Hidden in the remaining 98 percent are instructions that basically tell the genes how to switch on and off," he explained.

Stamatoyannopoulos and his colleagues analyzed 349 tissue samples from adults, and then cross-referenced the results with existing genetic data on more than 400 diseases and physical traits, such as height.

The result: a clearer picture of what's inside that mysterious 98 percent of the genome.

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Scientists Inch Closer to Genetic Blueprint of Diseases

by Ken Alltucker - Sept. 6, 2012 11:15 PM The Republic | azcentral.com

The right drug for the right person at the right time.

The pioneers of medicine's Genetic Age have long predicted that personalized drug treatments are inevitable as technology improves and costs plummet.

Although genetic medicine so far has produced more hype than substance, there are signs that medical treatments based on an individual's genes are tantalizingly close for some.

Pharmaceutical companies are investing tens of millions of dollars on developing tailored tests and drugs that identify and attack malfunctioning genes. High-powered machines can sequence an entire genome in a day, compared with earlier models that took weeks or even months to map out all of a person's genetic information.

In Arizona, nowhere is the prospect of personalized medicine more evident than at Mayo Clinic in Scottsdale and the Translational Genomics Research Institute in Phoenix.

Mayo Clinic, with an assist from TGen, is among the first wave of U.S. medical and research institutions offering people the potential of life-extending treatments based on a unique examination of their genes.

Those who are willing to spend $50,000 to $75,000 can have billions of their own genes decoded, sequenced and analyzed in an effort to figure out a personalized remedy to treat cancer or other life-threatening diseases.

Using a technology called whole-genome sequencing, Mayo and TGen researchers compare the DNA and other key genetic information from an individual's healthy cells and tumor cells to find a single gene or group of genes responsible for a cancer's growth.

Mayo's new initiative, part of its Center for Individualized Medicine, has attracted more than a dozen patients to metro Phoenix from places such as Houston and Canada and as far away as Russia in the hopes that technology can deliver a medical miracle.

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Mayo, TGen close in on personalized gene therapy

Public release date: 6-Sep-2012 [ | E-mail | Share ]

Contact: Gracie Gutierrez ggutierr@bcm.edu 713-798-4710 Baylor College of Medicine

HOUSTON - (Sept. 7, 2012) When mice are born lacking the master gene Atoh1, none breathe well and all die in the newborn period. Why and how this occurs could provide new answers about sudden infant death syndrome (SIDS), but the solution has remained elusive until now.

Research led by Baylor College of Medicine and the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital demonstrates that when the gene is lacking in a special population of neurons called RTN (retrotrapezoid nucleus), roughly half the young mice die at birth. Those who survive are less likely to respond to excess levels of carbon dioxide as adults. A report of their work appears online in the journal Neuron.

"The death of mice at birth clued us in that Atoh1 must be needed for the function of some neurons critical for neonatal breathing, so we set out to define these neurons," said Dr. Huda Zoghbi, senior author of the report and director of the Neurological Research Institute and a professor of molecular and human genetics, neuroscience, neurology and pediatrics at BCM. Zoghbi is also a Howard Hughes Medical Institute investigator.

"We took a genetic approach to find the critical neurons," said Wei-Hsiang Huang, a graduate student in the Program in Developmental Biology at BCM who works in Zoghbi's laboratory. With careful studies to "knockout" the activity of the gene in a narrower and narrower area in the brain, they slowly eliminated possible neurons to determine that loss of Atoh1 in the RTN neurons was the source of the problem.

"Discovering that Atoh1 is indeed critical for the RTN neurons to take their right place in the brainstem and connect with the breathing center helped us uncover why they are important for neonatal breathing," said Zoghbi.

"This population of neurons resides in the ventral brainstem," said Huang. "When there is a change in the makeup of the blood (lack of oxygen or buildup of carbon dioxide), the RTN neurons sense that and tell the body to change the way it breathes." A defect in these neurons can disrupt this response.

"Without Atoh1 the mice have significant breathing problems because they do not automatically adjust their breathing to decrease carbon dioxide and oxygenate the blood," he said.

It turns out the findings from this mouse study are relevant to human studies.

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Master gene affects neurons that govern breathing at birth and in adulthood

Public release date: 6-Sep-2012 [ | E-mail | Share ]

Contact: Cathia Falvey cfalvey@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 6, 2012The naturally occurring cytokine interleukin-18, or IL-18, plays a key role in inflammation and has been implicated in serious inflammatory diseases for which the prognosis is poor and there are currently limited treatment options. Therapies targeting IL-18 could prove effective against inflammatory diseases of the lung including bronchial asthma and chronic obstructive pulmonary disease (COPD), as described in a review article published in Journal of Interferon & Cytokine Research (http://www.liebertpub.com/jir), a peer-reviewed publication from Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com). The article is available free online at the Journal of Interferon & Cytokine Research website. (http://www.liebertpub.com/jir)

Tomotaka Kawayama and coauthors from Kurume University School of Medicine, Fukuoka, Japan, University of Ryukyus, Okinawa, Japan, and Frederick National Laboratory for Cancer Research, Frederick, MD, review the growing evidence to support the important role IL-18 has in inflammation and how it may help to initiate and worsen inflammatory disorders such as arthritis, dermatitis and inflammatory diseases of the bowel and immune system. In the article "Interleukin-18 in Pulmonary Inflammatory Diseases" (http://online.liebertpub.com/doi/full/10.1089/jir.2012.0029) they describe the potential benefits of therapies aimed at blocking the activity of IL-18 to treat inflammatory lung disease.

"This review provides an interesting and thorough summary of the biology and potential application of IL-18 in the setting of inflammatory pulmonary disease," says Co-Editor-in-Chief Thomas A. Hamilton, PhD, Chairman, Department of Immunology, Cleveland Clinic Foundation.

###

About the Journal Journal of Interferon & Cytokine Research (http://www.liebertpub.com/jir), led by Co-Editors-in-Chief Ganes C. Sen, PhD, Chairman, Department of Molecular Genetics, Cleveland Clinic Foundation, and Thomas A. Hamilton, PhD, is an authoritative peer-reviewed journal published monthly in print and online that covers all aspects of interferons and cytokines from basic science to clinical applications. Journal of Interferon & Cytokine Research is the official journal of the International Society for Interferon and Cytokine Research. Complete tables of content and a sample issue (http://online.liebertpub.com/toc/jir/31/6) may be viewed online at the Journal of Interferon & Cytokine Research website. (http://www.liebertpub.com/jir)

About the Publisher

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

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Promising new drug target for inflammatory lung diseases

NEW YORK (Reuters Health) - Counseling people about their personal risk of diabetes based on their genes may not motivate them to take steps to prevent the blood sugar disease, a new study suggests.

Overweight and obese research participants lost the same amount of weight and were similarly dedicated to a diabetes-prevention program whether they learned their genes put them at high or low risk - or when they hadn't been counseled at all.

"It's very, very hard to change behavior," said lead researcher Dr. Richard Grant, now at Kaiser Permanente Northern California in Oakland.

"The idea that a number, whether it's a genetic number or anything, will have a big impact on changing people's behavior - it just won't work."

Researchers have predicted that people who learn they are at extra-high risk of diabetes might be more motivated to change their lifestyle, and patient surveys support that idea.

But there is also the concern that those at low genetic risk will get a false sense of reassurance and believe that eating a healthy diet and exercising aren't important for them.

To examine those theories, Grant and his colleagues tested middle-aged adults at Massachusetts General Hospital in Boston for 36 genetic markers known to be linked to type 2 diabetes. Forty-two people with the highest genetic risk and 32 with the lowest risk then went to a brief counseling session about those findings.

After counseling, the researchers started people on both ends of the risk spectrum on a 12-week group diet and exercise education program, along with another 34 people who had never had their genes tested.

Over the course of the program, participants lost an average of 8.5 pounds, and just under one-third of them lost at least five percent of their initial body weight. There was no difference in weight loss among people with high or low genetic risk - nor was there a difference in participants' motivation to prevent diabetes or how closely they stuck to the program schedule.

"Even though people have the intent and they think genetic information will have an impact, when you actually look at their behavior to reduce their risk of diabetes we didn't see any differences," Grant told Reuters Health.

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Study: Genetic counseling about diabetes risk doesn’t change behavior

Public release date: 6-Sep-2012 [ | E-mail | Share ]

Contact: Patrick Dion patrick.a.dion@umontreal.ca 514-246-0126 University of Montreal

This press release is available in French.

Researchers from the Guy Rouleau Laboratory affiliated with the CHUM Research Centre and the CHUSainte-Justine Research Centre have discovered the genetic cause of a rare disease reported only in patients originating from Newfoundland: hereditary spastic ataxia (HSA).

This condition is characterized by lower-limb spasticity (or stiffness) and ataxia (lack of coordination), the latter leading to speech and swallowing problems, and eye movement abnormalities. The disease is not deadly, but people start developing gait problems between 10 to 20 years of age, walk with a cane in their 30s, and in the most severe cases, are wheel-chair bound in their 50s. It has been shown that HSA is transmitted from the affected parent to the child in a dominant fashion, which means there is a 50% chance of the child having the mutation.

History of a discovery: collaboration between the University of Montreal and Memorial University

Researchers and clinicians from Memorial University (St. John's, Newfoundland) contacted Dr. Rouleau, who is also a professor of medicine at the University of Montreal, over a decade ago to investigate the genetics behind this disorder occurring in three large Newfoundland families. Dr. Inge Meijer, a former doctoral candidate in the Rouleau Laboratory, discovered that these families were ancestrally related, and in 2002, identified the locus (DNA region) containing the mutation causing HSA.

A few years later, Cynthia Bourassa, lead author of the study, took over Meijer's project. "I reexamined some unresolved details using newer and more advanced methods," explains Bourassa, who is a master's student in the Faculty of Medicine at the University of Montreal. She then teamed up with Dr. Nancy Merner, who after obtaining her Ph.D. at Memorial University moved to Montreal to further her career in genetic research. "It is an honour to be a part of this study and impact the lives of my fellow Newfoundlanders. I knew coming into the Rouleau Laboratory that the genetic factors of the HAS families had not yet been identified. In fact, I asked about them on my first day of work, shortly after which I teamed up with Cynthia and we found the gene!"

Scientific explanation:

The gene harbouring the mutation is VAMP1, encoding the synaptobrevin protein. "Not only was the mutation present in all patients and absent from all population controls, but also, synaptobrevin is a key player in neurotransmitter release, which made sense at the functional level as well," says Bourassa. In fact, the authors believe that this mutation in the VAMP1 gene may affect neurotransmission in areas of the nervous system where the synaptobrevin protein is located, causing the unique symptoms of HSA. In other words, there are not enough messengers released, so nerves cannot function optimally.

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Genetic discovery in Montreal for a rare disease in Newfoundland

THE Newman government has announced a major refocus on genetics and disease resistance research to improve winter cereal varieties across the state.

Using the appropriate backdrop of the Toowoomba Ag Show, Premier Campbell Newman and Agriculture Minister John McVeigh announced, in a first for winter grains research in Queensland, a "genetic platform" for the research and development of cereal crops, which would see a renewed focus on genetics and pre-breeding at the Hermitage Research Station, Warwick.

Mr McVeigh said the refocus would boost barley breeding across the state through improved programs and a better product.

"It establishes a solid relationship between our researchers, who will concentrate on early stages of breeding, and InterGrain Pty Ltd, which will breed finished varieties for growers," he said.

"This new 'genetics platform' will have a strong disease-resistance focus.

"It's about delivering improved winter cereal varieties by the commercial sector to growers."

InterGrain is an Australian crop-breeding company with a highly successful barley-breeding program that targets the major cereal growing areas of Australia.

Mr Newman and Mr McVeigh were joined by the Grains Research and Development Corporation chairman Keith Perrett for the announcement, who said the organisation would co-invest in the platform with $1.4 million and a total of $11.2m over seven years, across all programs.

"This investment will support the transition of barley breeding to the commercial sector, underpin pre-breeding, and develop capacity in other key RD&E areas such as summer crop agronomy, genetics and pathology, farming systems research and production research," Mr Perrett said.

"This deal delivers 11 positions and this capacity will service high-priority areas identified by growers."

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Govt to focus on winter cereal research

SILVER SPRING, Md. and SINGAPORE, Singapore, Sept. 6, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced today its wholly-owned subsidiary, Austrianova Singapore Pte Ltd (ASPL) has just received notice that the U.S. based Personal Care Products Council (PCPC) has assigned the International Nomenclature Cosmetic Ingredient (INCI) name Gel8 (pronounced gelate), to the company's proprietary cell encapsulation materials.

The PCPC determined, through the International Nomenclature Committee, to provide the Gel8 name assignment to ASPL. As a result, Gel8 will now be included as a new product in the International Cosmetic Ingredient Dictionary & Handbook as well as in the next update of the Cosmetic industry's International Buyer's Guide. The combined Dictionary/Handbook contains more than 19,100 INCI labeling names for the United States, European Union, Japan and other countries. There are many aspects of cosmetics safety and labeling overseen by the FDA and other regulatory bodies around the world. Receiving the INCI designation allows for greater potential use in cosmetics throughout the US and the world.

Dr. John Dangerfield, Chief Operating Officer of ASPL, said, "We are thrilled that our work has enabled us to receive this designation. Most importantly, holding the INCI name is necessary for product safety and visibility as well as a significant step forward for using the Cell-in-a-Box(R) and Bac-in-a-Box(R) technology throughout the cosmetics industry. We see applications of the technology that expand from the cancer field well into other areas of medicine, cosmetics, and nutrition, among others, and see this designation as one of the first important steps."

The Chief Executive of Nuvilex, Dr. Robert Ryan, stated, "I was extremely impressed by what I saw while visiting ASPL in Singapore last week. I'm certain the effort they put forward to receive assignment of the INCI name Gel8 for our live cell components from our Cell-in-a-Box(R) and Bac-in-a-Box(R) patented encapsulation technology from the Personal Care Products Council is a significant move forward. All of the effort by the lab over the past year will allow us to expand the use of the technology and ultimately be able to produce products for other markets including the cosmetics market. The ability to receive the INCI designation also demonstrates how broad this platform technology we are developing together with ASPL is."

About Nuvilex

Nuvilex, Inc. (NVLX) is an international biotechnology provider of live therapeutically valuable, encapsulated cells and services for research and medicine. A great deal of work is ongoing to move Nuvilex and its Austrianova Singapore subsidiary forward. This was clearly apparent during Dr. Ryan's recent trip to Singapore completed last week. Our company's clinical offerings will include cancer, diabetes and other treatments using the company's cell and gene therapy expertise and live-cell encapsulation technology.

The Nuvilex, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=13494

Safe Harbor Statement

This press release contains forward-looking statements described within the 1995 Private Securities Litigation Reform Act involving risks and uncertainties including product demand, market competition, and meeting current or future plans which may cause actual results, events, and performances, expressed or implied, to vary and/or differ from those contemplated or predicted. Investors should study and understand all risks before making an investment decision. Readers are recommended not to place undue reliance on forward-looking statements or information. Nuvilex is not obliged to publicly release revisions to any forward-looking statement, reflect events or circumstances afterward, or disclose unanticipated occurrences, except as required under applicable laws.

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Nuvilex Cleared to Enter Multi Billion Dollar Cosmetics Industry

05-09-2012 16:14 Hank brings us breaking news from a team of geneticists working on figuring out what all that "junk DNA" in the human genome really is - turns out it's not junk after all. Like SciShow: Follow SciShow: References for this episode can be found in the Google document here: scishow, news, breaking, hank green, gene, genetics, DNA, junk DNA, ENCODE project, human genome project, code, instruction, protein, encyclopedia of DNA elements, biomedical, research, function, regulatory gene, regulator, cell, medicine, tissue sample, disease, autoimmune diseases, type 1 diabetes, illness, learn, discovery, immune system, predict, treat, noncoding DNA

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The Secret of Your "Junk," Revealed! - Video

TUESDAY, Sept. 4 (HealthDay News) -- At least seven antibiotic-resistant genes have recently passed between soil bacteria and bacteria that cause human disease, according to a new study.

Further research is needed to determine how widespread this sharing is, and to what extent it could make disease-causing bacteria harder to control, said the researchers at Washington University School of Medicine in St. Louis.

"It is commonplace for antibiotics to make their way into the environment. Our results suggest that this may enhance drug resistance in soil bacteria in ways that could one day be shared with bacteria that cause human disease," first author and graduate student Kevin Forsberg said in a university news release.

For this study, the researchers analyzed the DNA of bacteria in soil samples collected at various locations in the United States. The findings were published recently in the journal Science.

The researchers said it's important to find the answers to many questions, such as: Did the genes pass from soil bacteria to human pathogens or vice versa? Are the genes just the tip of a vast reservoir of shared resistance? Did some combination of luck and a new technique for studying genes across entire bacterial communities lead to the discovery of the shared resistance genes?

While humans only mix their genes when they have children, bacteria regularly exchange genes throughout their lifecycles. That means that when a strain of bacteria develops resistance to antibiotics, it can share this ability not only with its descendants but also with other bacteria, the researchers explained.

"I suspect the soil is not a teeming reservoir of resistance genes. But if factory farms or medical clinics continue to release antibiotics into the environment, it may enrich that reservoir, potentially making resistance genes more accessible to infectious bacteria," study senior author Gautam Dantas, an assistant professor of pathology and immunology, said in the news release.

-- Robert Preidt

Copyright 2012 HealthDay. All rights reserved.

SOURCE: Washington University School of Medicine in St. Louis, news release, Aug. 30, 2012

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Environmental 'Gene-Swapping' May Play Part in Antibiotic Resistance

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/b9kb5p/rnai_technologie) has announced the addition of Jain PharmaBiotech's new report "RNAi - Technologies, Markets and Companies" to their offering.

RNA interference (RNAi) or gene silencing involves the use of double stranded RNA (dsRNA). Once inside the cell, this material is processed into short 21-23 nucleotide RNAs termed siRNAs that are used in a sequence-specific manner to recognize and destroy complementary RNA. The report compares RNAi with other antisense approaches using oligonucleotides, aptamers, ribozymes, peptide nucleic acid and locked nucleic acid.

Various RNAi technologies are described, along with design and methods of manufacture of siRNA reagents. These include chemical synthesis by in vitro transcription and use of plasmid or viral vectors. Other approaches to RNAi include DNA-directed RNAi (ddRNAi) that is used to produce dsRNA inside the cell, which is cleaved into siRNA by the action of Dicer, a specific type of RNAse III. MicroRNAs are derived by processing of short hairpins that can inhibit the mRNAs. Expressed interfering RNA (eiRNA) is used to express dsRNA intracellularly from DNA plasmids.

Delivery of therapeutics to the target tissues is an important consideration. siRNAs can be delivered to cells in culture by electroporation or by transfection using plasmid or viral vectors. In vivo delivery of siRNAs can be carried out by injection into tissues or blood vessels or use of synthetic and viral vectors.

Because of its ability to silence any gene once the sequence is known, RNAi has been adopted as the research tool to discriminate gene function. After the genome of an organism is sequenced, RNAi can be designed to target every gene in the genome and target for specific phenotypes. Several methods of gene expression analysis are available and there is still need for sensitive methods of detection of gene expression as a baseline and measurement after gene silencing. RNAi microarray has been devised and can be tailored to meet the needs for high throughput screens for identifying appropriate RNAi probes. RNAi is an important method for analyzing gene function and identifying new drug targets that uses double-stranded RNA to knock down or silence specific genes. With the advent of vector-mediated siRNA delivery methods it is now possible to make transgenic animals that can silence gene expression stably. These technologies point to the usefulness of RNAi for drug discovery.

RNAi can be rationally designed to block the expression of any target gene, including genes for which traditional small molecule inhibitors cannot be found. Areas of therapeutic applications include virus infections, cancer, genetic disorders and neurological diseases. Research at academic centers that is relevant to RNAi-based therapeutics is mentioned.

Regulatory, safety and patent issues are discussed. Side effects can result from unintended interaction between an siRNA compound and an unrelated host gene. If RNAi compounds are designed poorly, there is an increased chance for non-specific interaction with host genes that may cause adverse effects in the host. However, there are no major safety concerns and regulations are in preliminary stages as the clinical trials are still ongoing and there are no marketed products. Many of the patents are still pending.

The markets for RNAi are difficult to define as no RNAi-based product is approved yet but several are in clinical trials. The major use of RNAi reagents is in research but it partially overlaps that of drug discovery and therapeutic development. Various markets relevant to RNAi are analyzed from 2011 to 2021. Markets are also analyzed according to breakdown of technologies and use of siRNAs, miRNAs, etc.

Profiles of 161 companies involved in developing RNAi technologies are presented along with 226 collaborations. They are a mix of companies that supply reagents and technologies (nearly half of all) and companies that use the technologies for drug discovery. Out of these, 33 are developing RNAi-based therapeutics and 34 are involved in microRNAs. The bibliography contains selected 600 publications that are cited in the report. The text is supplemented with 34 tables and 10 figures.

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Research and Markets: RNAi - Technologies, Markets and Companies - Updated 2012 Report

ScienceDaily (Sep. 5, 2012) The first integrated understanding of how the human genome functions will be published this week -- the triumphant result of a collaborative five-year project involving more than 440 researchers working in 32 labs worldwide. The Encyclopedia of DNA Elements project, known as ENCODE, will publish simultaneously on Sept. 6 a massive number of scientific papers, including one main integrative paper and five others in Nature; 18 in Genome Research; six in Genome Biology; and other affiliated papers in Science, Cell, and other scientific journals.

During the ENCODE study, researchers found that more than 80 percent of the human genome sequence is linked to biological function. They also mapped more than 4 million regulatory regions where proteins interact with the DNA with exquisite specificity. These findings are a significant advance in understanding the precise and complex controls over the expression of genetic information within a cell.

"Penn State's contribution to the ENCODE project involves using the new ENCODE data to help explain how genetic variants that do not affect the structure of encoded proteins could affect a person's susceptibility to disease," said Ross Hardison, the T. Ming Chu Professor of Biochemistry and Molecular Biology at Penn State and a member of the ENCODE research team. The research, led by Hardison, is highlighted in the main integrative ENCODE paper to be published in the journal Nature.

"Genome-wide association studies can map with high resolution the places on our genomes where variation in the DNA sequence among individual persons affects their likelihood of having diabetes, cardiac disease, any of a large number of autoimmune diseases such as Crohn's disease, and other common diseases," Hardison said. Because most of these genetic variations are not in regions of the DNA that contain the codes for producing proteins, scientists suspected that some of these noncoding regions might have an important role in controlling the expression of genes.

Hardison's team at Penn State worked with others in the ENCODE Consortium to show, on a genome-wide scale, that many of the DNA regions that do not hold codes for proteins do, indeed, have an important role in controlling which genes are turned on and which are turned off. "Moreover, our research has made it possible to generate specific molecular hypotheses for how genetic variants in these DNA regions that control gene expression could affect the susceptibility to disease," Hardison said. "We demonstrate this process using, as an example, a locus associated with Crohn's and a few other autoimmune diseases. It is exciting to see our basic research revealing insights that help the progress of medical science, potentially facilitating a more personalized approach to medical practice."

In addition to Hardison, other Penn State scientists whose work on the ENCODE project is featured among the papers to be published on Sept. 6 include Programmer/Analyst Belinda Giardine, postdoctoral scholars Robert S. Harris and Weisheng Wu, and Professor of Biology and of Computer Science and Engineering Webb Miller.

The overall ENCODE findings bring into much sharper focus the continually active genome in which proteins routinely turn genes on and off using sites that are sometimes at great distances from the genes they regulate; where sites on a chromosome interact with each other, also sometimes at great distances; where chemical modifications of DNA influence gene expression; and where various functional forms of RNA, a form of nucleic acid related to DNA, help regulate the whole system. "The ENCODE catalog is like Google Maps for the human genome," said Elise Feingold, a program director at the National Institutes of Health National Human Genome Research Institute (NHGRI), who helped to start the ENCODE Project. "The ENCODE maps allow researchers to inspect the chromosomes, genes, functional elements and individual nucleotides in the human genome in much the same way."

"During the early debates about the Human Genome Project, researchers had calculated that only a few percent of the sequence encoded proteins, the workhorses of the cell," said Eric D. Green, director of NHGRI. "Early on, some scientists even argued that most of the genome was 'junk.' ENCODE now gives us much more appreciation of the complex molecular ballet that converts genetic information into living cells and organisms, and we can now say that there is very little, if any, junk DNA."

Hundreds of researchers in the United States, United Kingdom, Spain, Singapore and Japan performed more than 1,600 sets of experiments on 147 types of tissue with technologies standardized across the consortium. The experiments relied on innovative uses of new next-generation sequencing technologies enabled, in part, by NHGRI's technology initiative for DNA sequencing. In total, ENCODE generated more than 15-trillion bytes of raw data and its analysis consumed the equivalent of more than 300 years of computer time.

The ENCODE project received principal funding from the National Human Genome Research Institute at the National Institutes of Health. Computation was enabled, in part, through the Penn State Cyberstar Computer, funded by the National Science Foundation (grant OCI-0821527).

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Huge human gene study includes Penn State University research

September 5, 2012

Connie K. Ho for redOrbit.com Your Universe Online

A group of scientists from Singapore recently discovered three new genes related to Primary Angle Closure Glaucoma (PACG), which is one of the main causes of blindness for Chinese people.

The new findings, recently published in the scientific journal Nature Genetics, will assist 15 million people throughout the globe who suffer from the disease.

This provides further evidence that genetic factors play a role in development of PACG, explained lead principal investigator Aung Tin, a professor of ophthalmology at NUH and deputy executive director at SERI who has studied PACG for over 10 years, in a prepared statement. It is a major achievement for our Singapore team leading the largest international consortium of doctors and scientists involved in glaucoma research. The results may lead to new insights into disease understanding and open the possibility of novel treatments in the future as well as the potential of early identification of people at risk of the disease.

80 percent of these 15 million who have PACG reside in Asia and the results can be very beneficial to medical experts who work with this particular population.

This is a landmark study identifying three genes that contribute to angle-closure glaucoma, a form of glaucoma that is particularly common in Asians. These data are the first critical steps toward a better understanding of the underlying molecular events responsible for this blinding disease, mentioned Janey Wiggs, the Paul Austin Chandler Associate Professor of Ophthalmology at Harvard Medical School, in the statement.

The study was a collaborative effort by scientists hailing from the Singapore Eye Research Institute (SERI), Singapore National Eye Centre (SNEC), the Genome Institute of Singapore (GIS), the National University of Singapore (NUS), National University Hospitals (NUH) Department of Ophthalmology and the Tan Tock Seng Hospital (TTSH).

This is a landmark finding, and may potentially change how we view PACG as a disease with genetic links. It highlights how a collective effort from scientists and clinicians and clinician-scientists can unravel diseases of major importance to Singapore. Because this disease is more common in Asians than in the Western populations, such studies will not be done in the US/Europe. This study has to be done in Asia as it is a disease with more implication for Asians. As such, Singapore has led the way forward, noted Wong Tien Yin, executive director of SERI as well as head and chair professor of the Department of Ophthalmology at NUHS, in the statement.

The group of investigators completed a genome-wide association study (GWAS) of 1,854 PACG cases and 9,608 controls of more than five sample collections throughout Asia. Another 1,917 PACG cases received validation experiments and 8,943 controls were taken from another six sample collections on a global scale. 1,293 Singapore residents with PACG and 8,025 Singaporean controls were part of the study.

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Gene Found In Glaucoma Largely Affects Asian Populations

Researchers have discovered a gene mutation that protects people in Southeast Asia against malaria in much the same fashion that a sickle cell trait protects Africans from the disease.

But while the sickle cell protects against the frequently lethal form of the disease caused by the parasite Plasmodium falciparum, the newly discovered gene mutation protects against Plasmodium vivax, which is generally thought to be more benign.

Malaria causes an estimated 1 million deaths per year worldwide, and at least half the world's population lives in areas at risk for the disease.

The sickle cell trait developed in Africa, where malaria is epidemic. People with one mutated gene have red blood cells that may occasionally assume a characteristic sickled shape under stress, generally with few or no severe effects.

People carrying two copies of the mutated gene, however, show much more severe sickling under similar conditions, and this can impede blood flow to organs, causing severe pain and organ deterioration.

The disease can be fatal. The trait has survived in the population, however, because carriers have a decreased vulnerability to P. falciparum malaria. They contract the disease, but symptoms are less severe.

The mechanism by which the trait provides protection is not yet clear, but it may involve changes in the membranes of red blood cells, which are attacked by the parasite.

A team of researchers led by geneticist Ivo Mueller of the Walter and Eliza Hall Institute in Parkville, Australia, and the Barcelona Center for International Health Research in Spain studied a genetic disorder called Southeast Asian Ovalocytosis or SAO, which causes red blood cells to become elliptical in shape or rod-shaped.

One copy of the gene produces mild disease, while two copies are generally lethal. Previous studies have shown that the carrier or trait form of the disorder, with one mutated gene, provides some protection against P. falciparum. Now Mueller and his colleagues have demonstrated that the mutation provides significant protection against P. vivax.

The team reported in the journal PLoS Medicine that they studied 1,975 children under the age of 15 in three independent epidemiological studies in Papua, New Guinea. About 10% to 15% of the population in that area carries the mutation.

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Newly discovered gene mutation offers malaria defense like sickle cell

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Linda Aagard 801-587-7639 University of Utah Health Sciences

SALT LAKE CITY For the first time, a mutation in HIF2, a specific group of genes known as transcription factors that is involved in red blood cell production and cell metabolism, has been identified in cancer tumor cells.

Researchers from Huntsman Cancer Institute (HCI) at the University of Utah and the National Institutes of Health found the mutation in tumor cells of two patients with the rare cancers paraganglioma/pheochromocytoma and somatostatinoma. The mutation was previously identified in connection with a non-cancerous hereditary condition, but never before in spontaneously arising cancers. The research results appear in the September 6, 2012 issue of the New England Journal of Medicine.

Transcription factors are proteins that bind to specific sequences of DNA to regulate cell functions. Hypoxia-inducible factors (HIFs) are transcription factors that control a wide range of functions connected to the way cells respond to oxygen, including metabolism (the way energy is produced) and the creation of red blood cells. Increased amounts of HIF in cancer cells was found to be responsible for the unique way they generate energy, referred to as the Warburg effect, named for Otto Warburg, a German physiologist who received the 1931 Nobel Prize in Physiology for this research.

"These HIF pathway mutations were first discovered while studying people with a condition called polycythemia that makes the body overproduce red blood cells," said one of the senior authors, Josef Prchal, M.D., professor in the Division of Hematology and Hematologic Malignancies at the University of Utah School of Medicine, an HCI investigator, and an expert on polycythemic disorders who has previously discovered other mutations in other HIF pathway genes.

The findings raise some important questions for future research. "The patients in which we found these mutations have a rare combination of diseases, paraganglioma/pheochromocytoma and polycythemia, yet they shared similar mutations," said Prchal. "Learning whether HIF pathway mutations are present in other cancers could increase understanding of the mechanisms of cancer cell metabolism and offer a possible new target for cancer treatment development."

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Felipe Lorenzo, M.D., Ph.D., postdoctoral fellow in the University of Utah Division of Hematology and Hematologic Malignancies, collaborated on this article. Other co-authors are associated with various entities of the National Institutes of Health. The research was supported, in part, by the Intramural Research Program of the National Institutes of Health, the National Institute of Child Health and Human Development, and the National Institute of Neurological Disorders and Stroke and VAH Merit Award to Dr. Prchal. The National Institutes of Health support Huntsman Cancer Institute through P30 CA042014.

The mission of Huntsman Cancer Institute (HCI) at the University of Utah is to understand cancer from its beginnings, to use that knowledge in the creation and improvement of cancer treatments, to relieve the suffering of cancer patients, and to provide education about cancer risk, prevention, and care. HCI is a National Cancer Institute-Designated Cancer Center, which means that it meets the highest national standards for cancer care and research and receives support for its scientific endeavors. HCI is also a member of the National Comprehensive Cancer Network (NCCN), a not-for-profit alliance of the world's leading cancer centers that is dedicated to improving the quality and effectiveness of care provided to patients with cancer. For more information about HCI, please visit http://www.huntsmancancer.org.

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HIF gene mutation found in tumor cells offers new clues about cancer metabolism

Editor's Choice Main Category: Cystic Fibrosis Article Date: 05 Sep 2012 - 10:00 PDT

Current ratings for: Ivacaftor Improves Lung Function And Symptoms In Cystic Fibrosis Patients With Specific Genetic Mutation

The STRIVE study randomised 161 patients aged 12 and over with cystic fibrosis and at least one copy of the G551D mutation in the CFTR gene to ivacaftor (150mg every 12 hours) or placebo. Results showed a mean absolute improvement of 10.6% in predicted FEV1 after 24 weeks' treatment with ivacaftor compared to placebo (p<0.0001), sustained at 10.5% at 48 weeks.

The ENVISION study, which included 52 children aged 6-11 years, showed similar absolute improvement of 12.5% in predicted FEV1 with ivacaftor at 24 weeks compared to placebo (p<0.0001), with a 10.0% improvement maintained at 48 weeks.

STRIVE showed a significant reduction in pulmonary exacerbations and clinically significant improvement in the respiratory domain of patients' quality of life with ivacaftor. Both studies showed an increase in body weight (mean increases of 2.7kg at 48 weeks in STRIVE and 2.8kg in ENVISION).

"Ivacaftor is the first medicine to treat the underlying cause of cystic fibrosis in people with the G551D mutation, a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene," said Stuart Elborn, Professor of Respiratory Medicine at Queen's University, Belfast, Northern Ireland and a principal investigator. "These data showing the consistent and sustained benefit of this medicine confirm that ivacaftor has the potential to make a significant difference to the lives of children, adolescents and adults with this form of CF."

He added: "The data don't capture the full benefit for patients. It's been very noticeable in the patients I look after that they are able to do things they previously couldn't after starting treatment with ivacaftor. They feel better and more able to plan for the future."

Fewer patients in the ivacaftor treatment groups discontinued treatment due to adverse events compared to placebo in both trials. Most adverse events associated with ivacaftor were mild to moderate, with some of the commonest including headache, upper respiratory tract infection, rash, diarrhea and abdominal pain.

Data reported from the follow-up PERSIST study showed that improvements in lung function with ivacaftor were sustained for patients continuing treatment from the STRIVE and ENVISION trials for up to 96 weeks. Those switched from placebo to open-label ivacaftor showed similar improvements in FEV1 to those seen in the patients starting on the drug during the placebo-controlled trials.

Reporting the findings, Dr Edward McKone, Consultant Respiratory Physician at St Vincent's University Hospital, Dublin, Ireland, said:

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Ivacaftor Improves Lung Function And Symptoms In Cystic Fibrosis Patients With Specific Genetic Mutation

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Haley Bridger hbridger@broadinstitute.org 617-714-7968 Broad Institute of MIT and Harvard

Cambridge, MA. Wed. September 5, 2012 Most of the DNA alterations that are tied to disease do not alter protein-coding genes, but rather the "switches" that control them. Characterizing these switches is one of many goals of the ENCODE project a sweeping, international effort to create a compendium of all of the working parts of the human genome that have not been well studied or well understood.

The function of the vast majority of the human genome has remained largely unknown, but the Encyclopedia of DNA Elements (ENCODE) project, launched in 2003, set out to change that. Comprised of more than 30 participating institutions, including the Broad Institute, the ENCODE Project Consortium has helped to ascribe potential biochemical function to a large fraction of the non-coding genome. This work has revealed elements that act like dimmer switches, subtly turning up or down a gene's activity and influencing what parts of the genome are utilized in different kinds of cells. The team characterized and mapped out the locations of thousands of these switches and signals. More than 30 papers detailing these results appear online in Nature, Science, Genome Research, and Genome Biology this week.

"With these maps in hand, we can begin to understand why genetic variants that land in the annotated regions may predispose people to disease," said Brad Bernstein, a senior associate member at the Broad Institute and an associate professor of pathology at Massachusetts General Hospital (MGH) and Harvard Medical School. Bernstein is also a principal investigator in the ENCODE Consortium. "It turns out that many of the variants that genetic researchers have tied to various diseases lupus, Crohn's disease, metabolic diseases, high cholesterol, and much more sit in these regions that alter how genes are expressed in specific kinds of cells."

Researchers from the Broad, MIT, and MGH found that variants associated with autoimmune diseases such as lupus and rheumatoid arthritis sit in regions that are active only in immune cells, whereas variants tied to cholesterol and metabolic diseases sit in regions active in liver cells.

In order to generate detailed maps of the switches that lie between and even nestled within genes, data collection centers in the consortium amassed high-quality and comprehensive datasets detailing the function of elements in the genome. By looking across more than 140 cell types, they generated more than 1,500 datasets.

Several groups at the Broad Institute contributed to this work, including the Genome Sequencing and Analysis Program and the Epigenomics Program, which helped generate many of the datasets for the project. The term "epigenome" refers to a layer of chemical information on top of the genetic code, which helps determine when and where (and in what types of cells) genes will be active. This layer of information includes a suite of chemical changes that appear across the genetic landscape of every cell, and can differ dramatically between cell types. Researchers at the Broad and at the other ENCODE data collection centers developed ways to characterize these epigenetic "marks" across cell types.

"By bringing together computational groups from across the world and gathering all of the data generated, we can get at much more complex questions," said Manolis Kellis, an associate member of the Broad Institute, principal investigator at the MIT Computer Science and Artificial Intelligence Lab (CSAIL), and an associate professor of Computer Science at MIT. Kellis is also head of the MIT Computational Biology Group and a principal investigator in the ENCODE Consortium.

Maps and data generated through the ENCODE project have been publicly released as they have become available. With these maps, Bernstein, Kellis, and their Broad colleagues:

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Mapping a genetic world beyond genes

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/96czlk/gene_therapy_tec) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2011, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2011-2021. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

The voluminous literature on gene therapy was reviewed and selected 700 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 72 tables and 14 figures.

Profiles of 187 companies involved in developing gene therapy are presented along with 208 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

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Research and Markets: Gene Therapy - Technologies, Markets and Companies - Updated 2012 Report

NEW YORK, Sept. 5, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NBS) ("NeoStem" or the "Company"), a cell therapy company, today announced that Company management of a NeoStem company, Progenitor Cell Therapy ("PCT"), an internationally recognized contract development and manufacturing organization (CDMO), has been invited to present on its core expertise in development of commercial manufacturing processes for cell therapy at two cell therapy conferences in September. At each, PCT will offer its unique perspective as an industry leader in contract development and manufacturing of cell therapy products, with over 12 years of exclusive cell-therapy focused experience.

Timothy Fong, Ph.D, M.B.A, PCT's Vice President, Technology and Product Development, will be sharing PCT's expertise in cell therapy manufacturing with a focus on commercialization. At IBC Life Sciences' Cell Therapy Bioprocessing Conference, he will chair a panel on quality assurance and controls and will give a presentation entitled "From Concept to Product: Considerations for Developing a Robust Commercial Manufacturing Process", which will include considerations for developing a robust commercial manufacturing process. He will also speak at the Stem Cells USA and Regenerative Medicine Congress on "Cell manufacturing considerations for first-in-world stem cell therapeutics".

Dr. Fong stated, "As a cell therapeutic progresses from concept to product, the development of a commercial manufacturing process may contain unexpected technical and quality issues. The development path should follow several defined steps. My presentations will discuss the key steps in the process and highlight critical areas that need to be addressed to develop a successful commercial manufacturing process. PCT helps clients bridge the gap between discovery and patient care through efficient transfer of cell-based therapies from laboratory into clinical practice."

NeoStem and PCT invite you to attend the conference(s), see Dr. Fong's talks, and connect with the PCT team at PCT's booths. If you are a colleague of PCT or NeoStem, PCT can offer you a registration discount. Please contact PCT at bdm@pctcelltherapy.com for more details.

IBC Life Sciences' 2nd Annual Cell Therapy Bioprocessing Conference

Terrapinn 4th Annual Stem Cells USA and Regenerative Medicine Congress

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a large role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT") with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cells product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a patent and patent pending (IP) portfolio, we believe we are well positioned to succeed.

For more information on NeoStem, please visit http://www.neostem.com. For more information on PCT, please visit http://www.pctcelltherapy.com.

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Progenitor Cell Therapy, a NeoStem Company, Invited to Present at Two Conferences in September

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/9f72rt/drug_delivery_in_c) has announced the addition of Jain PharmaBiotech's new report "Drug Delivery in Cancer - Technologies, Markets and Companies" to their offering.

Drug delivery remains a challenge in management of cancer. Approximately 12.5 million new cases of cancer are being diagnosed worldwide each year and considerable research is in progress for drug discovery for cancer. Cancer drug delivery is no longer simply wrapping up cancer drugs in a new formulations for different routes of delivery. The focus is on targeted cancer therapy. The newer approaches to cancer treatment not only supplement the conventional chemotherapy and radiotherapy but also prevent damage to normal tissues and prevent drug resistance.

Innovative cancer therapies are based on current concepts of molecular biology of cancer. These include antiangiogenic agents, immunotherapy, bacterial agents, viral oncolysis, targeting of cyclic-dependent kinases and tyrosine kinase receptors, antisense approaches, gene therapy and combination of various methods. Important methods of immunotherapy in cancer involve use of cytokines, monoclonal antibodies, cancer vaccines and immunogene therapy.

Several innovative methods of drug delivery are used in cancer. These include use of microparticles as carriers of anticancer agents. These may be injected into the arterial circulation and guided to the tumor by magnetic field for targeted drug delivery. Polyethylene glycol (PEG) technology has been used to overcome some of the barriers to anticancer drug delivery. Encapsulating anticancer drugs in liposomes enables targeted drug delivery to tumor tissues and prevents damage to the normal surrounding tissues. Monoclonal antibodies can be used for the delivery of anticancer payloads such as radionucleotides, toxins and chemotherapeutic agents to the tumors.

Antisense oligonucleotides have been in clinical trials for cancer for some time now. RNAi has also been applied in oncology. Small interfering RNAs (siRNAs) can be targeted to tumors and one example is suppression of H-ras gene expression indicating the potential for application in therapy of ovarian cancer. Cancer gene therapy is a sophisticated form of drug delivery for cancer. Various technologies and companies developing them are described. Nucleic acid-based cancer vaccines are also described.

Drug delivery strategies vary according to the type and location of cancer. Role of drug delivery in the management of cancers of the brain, the bladder, the breast, the ovaries and the prostate are used as examples to illustrate different approaches both experimental and clinical. Biodegradable implants of carmustine are already used in the treatment of malignant brain tumors.

The market value of drug delivery technologies and the anticancer drugs are difficult to separate. Cancer market estimates from 2011-2021 are given according to organs involved and the types of cancer as well as according to technologies. Distribution of the into major regions is also described.

Profiles of 213 companies involved in developing innovative cancer therapies and methods of delivery are presented along with their 242 collaborations. The bibliography contains over 650 publications that are cited in the report.The report is supplemented with 58 tables and 9 figures.

Key Topics Covered:

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Research and Markets: Drug Delivery in Cancer - Technologies, Markets and Companies - Updated 2012 Report

Public release date: 5-Sep-2012 [ | E-mail | Share ]

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

New Rochelle, NY, September 5, 2012According to the Centers for Disease Control and Prevention (CDC), 1 in 88 children in the U.S. has autism spectrum disorder (ASD), a broad group of neurodevelopmental disorders. Children and adolescents with ASD are typically fascinated by screen-based technology such as videogames and these can be used for educational and treatment purposes as described in an insightful Roundtable Discussion published in Games for Health Journal: Research Development, and Clinical Applications, a peer-reviewed publication from Mary Ann Liebert, Inc.. The article is available free on the Games for Health Journal website.

Individuals with ASD have difficulty with communication and social interaction, but they often have particularly good visual perceptual skills and respond well to visual stimuli. Videogames offer opportunities for successful learning, motivation to improve skills such as planning, organization, and self-monitoring, and reinforcement of desired behaviors without the need for direct human-to-human interaction.

Autism is a growing area of interest for the gamification community, and Games for Health Journal continues to explore various aspects of how videogame technology can be beneficial in treating this complex spectrum of disorders. In a previous issue of the Journal, the article "Comparing Energy Expenditure in Adolescents with and without Autism while Playing Nintendo Wii Games" described how gaming might help individuals with ASD increase their daily physical activity to prevent obesity.

"Children and young adults with ASD have unique opportunities to capitalize on their interest and aptitude in videogames as a resource to develop desired social behaviors and life skills and to increase their physical activity," says Games for Health Journal Editor-in-Chief Bill Ferguson, PhD, who moderated the Roundtable.

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About the Journal

Games for Health Journal (http://www.liebertpub.com/g4h) breaks new ground as the first journal to address this emerging and increasingly important area of health care. The Journal provides a bimonthly forum in print and online for academic and clinical researchers, game designers and developers, health care providers, insurers, and information technology leaders. Articles explore the use of game technology in a variety of clinical applications. These include disease prevention and monitoring, nutrition, weight management, and medication adherence. Gaming can play an important role in the care of patients with diabetes, post-traumatic stress disorder, Alzheimer's disease, and cognitive, mental, emotional, and behavioral health disorders.

About the Publisher

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Can videogaming benefit young people with autism spectrum disorder?

Public release date: 5-Sep-2012 [ | E-mail | Share ]

Contact: Cathia Falvey cfalvey@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 5, 2012A group of experts has developed consensus recommendations for future research directions to determine why nearly two-thirds of Americans with Alzheimer's disease (AD) are women. The recommendations are published in a Roundtable discussion in Journal of Women's Health, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Women's Health website at http://www.liebertpub.com/jwh.

An estimated 5.4 million Americans are affected by AD and related dementias, and that number will likely rise to 11-16 million people by the year 2050 if no effective cures or preventive measures are developed. The main risk factors for AD are age and sex, with affected women outnumbering men 2 to 1. This may be due at least in part to the fact that women tend to live longer.

An interdisciplinary roundtable of experts convened by the Society for Women's Health Research (Washington, DC) led to a set of recommendations to help guide future AD research and make the evaluation of sex and gender differences a component of future studies. The consensus recommendations encompass seven themes, including the need to assess the link between sex and AD incidence, raise awareness of sex differences among the research community, and to take into account sex-based differences in the experimental design and data analysis of studies on disease risk, early diagnosis, and drug discovery.

"There are still major gaps in our knowledge of the role of sex and gender in the onset and progression of Alzheimer's disease, and these recommendations will provide a useful guide for future research in this area," says Susan G. Kornstein, MD, Editor-in-Chief of Journal of Women's Health, Executive Director of the Virginia Commonwealth University Institute for Women's Health, Richmond, VA, and President of the Academy of Women's Health.

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About the Journal

Journal of Women's Health, published monthly, is a core multidisciplinary journal dedicated to the diseases and conditions that hold greater risk for or are more prevalent among women, as well as diseases that present differently in women. The Journal covers the latest advances and clinical applications of new diagnostic procedures and therapeutic protocols for the prevention and management of women's healthcare issues. Tables of content and a sample issue may be viewed on the Journal of Women's Health website at http://www.liebertpub.com/jwh. Journal of Women's Health is the Official Journal of the Academy of Women's Health.

About the Academy

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Why does Alzheimer's disease affect twice as many women as men?