Archive for the ‘Gene Therapy Research’ Category
How stem cell therapy can keep the immune system under control
A new study, appearing in Cell Stem Cell and led by researchers at the University of Southern California, outlines the specifics of how autoimmune disorders can be controlled by infusions of mesenchymal stem cells.
Mesenchymal stem cells (MSC) are highly versatile stem cells that originate from the mesoderm, or middle layer of tissue, in a developing embryo. MSC can be isolated from many different kinds of human tissue, including bone marrow and the umbilical cord.
Principal investigator Songtao Shi, professor at the Ostrow School of Dentistry of USC Center for Craniofacial Molecular Biology, said that recent studies have shown the benefits of administering MSC to patients with immune-related disorders such as graft versus host disease, systemic lupus erythematosus, rheumatoid arthritis, and more.
These studies showed that infusions of MSC appeared to quell the production and function of overactive immune cells, including T- and B-lymphocytes. However, the specific mechanism behind how MSC get the immune cells under control hasn't been fully understood.
"Mesenchymal-Stem-Cell-Induced Immunoregulation Involves FAS-Ligand-/FAS-Mediated T Cell Apoptosis" shines light on how infused MSCs target and defeat overactive immune cells.
Examining the effects of MSC infusion in mice with systemic sclerosis (SS)-like immune disorders, Shi and his colleagues discovered that a specific cellular mechanism known as the FAS/FAS-ligand pathway was the key to the remarkable immune system benefits.
Specifically, in mice with SS-like disorders, infusions of MSC caused T-lymphocyte death with FASL/FAS signaling and lessened symptoms of the immune disorder. However, MSC deficient in FAS-ligand failed to treat immune disorders in SS-afflicted mice.
With the hopeful results of the animal model study in mind, Shi's colleagues in China performed a pilot study with patients suffering from systemic sclerosis. Infusions of MSCs provided similar clinical benefits to patients, and experimental analysis revealed that the FASL/FAS pathway was also at work in humans with SS.
The identification of the cellular workings responsible for the stem cell treatments' success may eventually help doctors find optimal cell-based treatment for some immune diseases, Shi said.
Basic research portions of this study were supported by the National Institute of Dental and Craniofacial Research and the California Institute for Regenerative Medicine. Clinical studies were supported by a grant from the China Major International (Regional) Joint Research Project.
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How stem cell therapy can keep the immune system under control
World Government Election Fraud 2012 Part 4 – Video
28-04-2012 12:02 Playlist: I work a job and don't make a lot of money at work. Please be patient for the other parts. I hope to complete this documentary before June 2012. This educational documentary World Government Election Fraud 2012 reveals research about government, politics, energy, sovereignty, sustainability and many other topics including solutions. Diebold sells computer voting machines they had Republican conflict of interest during the 2 term voting Fraud of George Bush. Ron Paul Admits he is running the same platform George Bush used to win in 2000! Be your own leader. Do your own Research. Ron Paul wrote a book called The Case for Gold with Lewis Lehrman. Lew is a papal knight (Sovereign Military Order of Malta) Lew was on the board of directors of The Project for a New American Century. Lewis Lehrman is a member of the CFR and The Council for National Policy. The CNP consists of powerful individuals in the military industrial complex (Black Water aka Xe) banking cartels, multi-national corporations, previous presidents/presidential candidates, Prime Minister of Canada, Evangelical Christian Zionists, Scientology, Mormons and a lot of the guests Alex Jones has on his radio "program" on a regular basis. Meaning that, it is behind the phony patriot movement, tea party, libertarians and other types of activism. Butcher of Russia, Vladimir Lenin, was a Freemason. Aleister Crowley (1875-1947) became a Freemason of the 33rd degree in the Scottish rite and was a ...
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World Government Election Fraud 2012 Part 4 - Video
USP9x: Gene discovered can 'turn off' pancreatic cancer
Aggressive pancreatic tumors may be treatable with a new class of drugs because of the discovery of the USP9x gene, according to Cancer Research UK.
Less than one in five people with pancreatic cancer survive past the first year after being diagnosed, reported BBC News. The study, published in the journal Nature, showed that the gene in question was being switched off in cancerous cells. Drugs with the potential to turn USP9x back on and stop the spread of cancer are already being tested.
Research was first conducted on mice and then on human cell lines, according to AAP. In both cases, it showed that the gene is switched off by chemical "tags" on the surface of its DNA.
"We looked in human tumor specimens and we found that [USP9x] was missing in a fraction of patients the patients that did very poorly... the people who died the fastest," said researcher David Tuveson to News24. "Patients that had a low level of the gene expressed... they died very quickly after their operation and the patients who at the end of their life had lots of metastasis [spreading of the cancer], they had also a very low level of this protein."
More from GlobalPost:Cancer 'encyclopedia' to help personalize cancer care, scientists say
http://www.globalpost.com/dispatch/news/health/120430/usp9x-gene-discovered-pancreatic-cancer
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USP9x: Gene discovered can 'turn off' pancreatic cancer
Pancreatic cancer gene discovery
30 April 2012 Last updated at 02:45 ET
Aggressive pancreatic tumours may be treatable with a new class of drugs, according to Cancer Research UK
Less than one in five people with this form of cancer are still alive a year after being diagnosed.
A study, published in the journal Nature, showed that a gene was being switched off in the cancerous cells.
The reseachers said drugs were already being tested which had the potential to turn the gene back on, to stop the spread of the cancer.
Around 7,800 people in the UK are diagnosed with pancreatic cancer every year and it is the fifth most deadly cancer.
These results raise the possibility that a class of promising new cancer drugs may be effective at treating some pancreatic cancers
Studies in mice showed that a gene called USP9x, which normally stops a cell from dividing uncontrollably, is switched off in some pancreatic cancer cells.
The gene is not mutated, but other proteins and chemicals become stuck to it and turn the gene off.
Studies then showed that UPS9x was being turned off in human pancreatic cancer.
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Pancreatic cancer gene discovery
Improved Adult-Derived Human Stem Cells Have Fewer Genetic Changes Than Expected
--Study lends support to safe use for therapy
Newswise A team of researchers from Johns Hopkins University and the National Human Genome Research Institute has evaluated the whole genomic sequence of stem cells derived from human bone marrow cellsso-called induced pluripotent stem (iPS) cellsand found that relatively few genetic changes occur during stem cell conversion by an improved method. The findings, reported in the March issue of Cell Stem Cell, the official journal of the International Society for Stem Cell Research (ISSCR), will be presented at the annual ISSCR meeting in June.
Our results show that human iPS cells accrue genetic changes at about the same rate as any replicating cells, which we dont feel is a cause for concern, says Linzhao Cheng, Ph.D., a professor of medicine and oncology, and a member of the Johns Hopkins Institute for Cell Engineering.
Each time a cell divides, it has the chance to make errors and incorporate new genetic changes in its DNA, Cheng explains. Some genetic changes can be harmless, but others can lead to changes in cell behavior that may lead to disease and, in the worst case, to cancer.
In the new study, the researchers showed that iPS cells derived from adult bone marrow cells contain random genetic changes that do not specifically predispose the cells to form cancer.
Little research was done previously to determine the number of DNA changes in stem cells, but because whole genome sequencing is getting faster and cheaper, we can now more easily assess the genetic stability of these cells derived by various methods and from different tissues, Cheng says. Last year, a study published in Nature suggested higher than expected cancer gene mutation rates in iPS cells created from skin samples, which, according to Cheng, raised great concerns to many in the field pertaining to usefulness and safety of the cells. This study analyzed both viral and the improved, nonviral methods to turn on stem cell genes making the iPS cells
To more thoroughly evaluate the number of genetic changes in iPS cells created by the improved, non-viral method, Chengs team first converted human blood-forming cells or their support cells, so-called marrow stromal cells (MSCs) in adult bone marrow into iPS cells by turning on specific genes and giving them special nutrients. The researchers isolated DNA from--and sequenced--the genome of each type of iPS cells, in comparison with the original cells from which the iPS cells were derived.
Cheng says they then counted the number of small DNA differences in each cell line compared to the original bone marrow cells. A range of 1,000 to 1,800 changes in the nucleic acid letters A, C, T and G occurred across each genome, but only a few changes were found in actual genes--DNA sequences that act as blueprints for our bodys proteins. Such genes make up two percent of the genome.
The blood-derived iPS cells contained six and the MSC-derived iPS cells contained 12 DNA letter changes in genes, which led the researchers to conclude that DNA changes in iPS cells are far more likely to occur in the spaces between genes, not in the genes themselves.
Next, the investigators examined the severity of the DNA changes--how likely each one would disrupt the function of each gene. They found that about half of the DNA changes were silent, meaning these altered blueprints wouldnt change the nucleic acid building code for its corresponding protein or change its function.
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Improved Adult-Derived Human Stem Cells Have Fewer Genetic Changes Than Expected
In saving teen, docs find "simple" genetic diseases not so simple
When he was 3 years old and falling behind in his childhood development, doctors diagnosed Jonathan Oliphint with a rare genetic disease.
There was a known treatment for the disease, in which the body produces an excess amount of ammonia, and doctors at Texas Children's Hospital soon began giving the boy a critical amino acid, arginine, his body was not making.
It was a classic case of the power of the emerging science of genetic medicine in which doctors could pinpoint the genetic causes of disease, and devise successful treatments.
That was the late 1990s, when genetics was a simpler science.
Problems return
The problem is, as scientists were discovering that the Human Genome Project and its elucidation of humanity's basic DNA would not immediately lead to medical breakthroughs, Oliphint started getting sick again.
He started having high blood pressure. By the time he was 15, half a dozen blood pressure medicines had failed, and Oliphint was in the ICU with an enlarged heart.
"They just couldn't get it under control," said the boy's mother, Jamie Oliphint. "It was scary. We didn't know what was going to happen."
As Oliphint's blood pressure problems increased, scientists were rewriting our understanding of genes and disease.
No so simple
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In saving teen, docs find "simple" genetic diseases not so simple
Molecular Biology and Genetics Unit opened at Nawaloka
The Molecular Biology and Genetics Diagnostics unit is a newly conceived unit of the Nawaloka Metropolis Laboratories Pvt. Ltd.
The Molecular Biology and Genetics Research unit was formed a year ago with the aim of diagnosing difficult bacterial and viral pathogens using technologies like PCR, FISH, and Flow Cytometry, Chairman of Metropolis Health Services, Mumbai, India Dr Sushil Shah said.
He was speaking at the inauguration ceremony of the Nawaloka Metropolis Molecular Biology and Genetics Research Laboratory recently.
Director General Manager, Nawaloka Hospitals PLC Prof Lal G Chandrasena said Nawaloka Metropolis Laboratory is the first Molecular and Genetics Research Diagnostic laboratory in Sri Lanka to offer state-of-the-art Smart Cycler ii which is quantitative/qualitative real time PCR (QPCR) tests to the diagnostic people and have established an internationally renowned reputation for providing excellent quality results.
Metropolis has been officially recognized from health professional organization CAP (College of American Pathologists).
Nawaloka Hospitals PLC Chairman Jayantha Dharmadasa said within the past six years Nawaloka Metropolis Laboratories have upgraded their laboratory facilities using modern and state-of-the-art equipment.
With the inauguration of Molecular Biology and Genetics Research facility Nawaloka Metropolis Laboratory will serve the people with many innovative tests and technologies, he said.
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Molecular Biology and Genetics Unit opened at Nawaloka
Gene Therapy Convention I-SMART on May 2nd 2012, 4 PM to 7 PM, at Bangalore Under the Aegis of DRDO, DST, Dr.Agarwal …
BANGALORE, India, April 30, 2012 /PRNewswire/ --
I-SMART (Indian Society For Molecular Advanced Regenerative Technologies) a conglomerate of Indian scientists from DRDO, DST, Dr.Agarwal Hospitals, Manacells, Gene Research Foundation has organized the first Gene Therapy Convention to be held at Bangalore on May 2nd2012.
People who are interestedare welcome to contact Dr.Agarwal Hospitals for registrations. Address: Dr.Agarwal Hospitals15, Eagle ST, Bangalore 560025, Phone 080-22240736, 22240200, 22221242, mobile +91-9845010510, 9845039271, 9844202364 email generesearchfoundation@gmail.comThe conference will shed new light into the frontiers of medicine where patients can be addressed with healthy DNA from their own blood.
I-SMART Millenium Awards will be presented to the national heroes, Dr.V.K.Saraswat (Chief of DRDO), Sri Oscar Fernandes (MP), Sreesanth (Cricketeer), Pujya Swamy Chidanand Saraswati (Parmarth Niketan, Rishikesh) Dr. Ramasami (Sec DST),On May 2nd4 pm 2012, Bangalore.
DNA-Gene therapyuses peripheral blood from the patientsfinger with Mana (DNA Activator) to generate the patients own healthy or fetal DNA.This is the molecule responsible for creating aparticular human body and thus it can also repair and regenerate the respective aging person.Thus customization of medical technologies has made it possible to treat various conditions associated with diseases of the eye, neurological disorders, diabetes, hypertension, cancer etc.
The work on this line of treatment was initiated over 20 years backby Dr.Agarwal Hospitals and Gene Research Foundation, with the help of The Department of Science & Technology, Govt. of India. For over 6 years Mana DNA gene therapy and the Defence Research Development Organization have added value to medical science.They have used high tech instrumentation and microscopes to show DNA formation within seconds of one drop of patients blood and Mana (DNA activator) coming together.
Through Dr.Agarwal Hospitals this medication has been used on eye patients (most of them are associated with a general disease) with great success. Over the last 8 years and 12000 patients using the patients own blood (autologous) shows no side effects or allergic reactions. This has taken a new step into medical technologies by giving it customization of each individual patient and seems to be one single major breakthrough in making the treatment a success.
After two days of debate and discussion the first Gene Therapy Convention I-SMART 2012 will be brought into mass productionto reach out to soldiers on the front (DRDO),and the masses (DST, through Manacells. An expansion plan to the tune of 300 cr is required and investors are welcome to come in at this stage. Over 100cr will go into a high end research facility which will monitor the mass production as well as be open to other sources.
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Gene Therapy Convention I-SMART on May 2nd 2012, 4 PM to 7 PM, at Bangalore Under the Aegis of DRDO, DST, Dr.Agarwal ...
Research and Markets: Recent Advances in Cancer Research and Therapy – Increased Research on one of the Major Causes …
DUBLIN--(BUSINESS WIRE)--
Research and Markets (http://www.researchandmarkets.com/research/msjp88/recent_advances_in) has announced the addition of Elsevier Science and Technology's new report "Recent Advances in Cancer Research and Therapy" to their offering.
Cancer continues to be one of the major causes of death throughout the developed world, which has led to increased research on effective treatments. Because of this, in the past decade, rapid progress in the field of cancer treatment has been seen. Recent Advances in Cancer Research and Therapy reviews in specific details some of the most effective and promising treatments developed in research centers worldwide. While referencing advances in traditional therapies and treatments such as chemotherapy, this book also highlights advances in biotherapy including research using Interferon and Super Interferon, HecI based and liposome based therapy, gene therapy, and p53 based cancer therapy. There is also a discussion of current cancer research in China including traditional Chinese medicine. Written by leading scientists in the field, this book provides an essential insight into the current state of cancer therapy and treatment.
Key Topics Covered:
1. Biotherapy of Cancer: Progress in China
2. Cancer Targeting Gene-Viro-Therapy (CTGVT) and Its Promising Future
3. Relationship Between Antiproliferative Activities and Class I MHC Surface Expression of Mouse Interferon Proteins on B16-F10 Melanoma Cells
4. Mitotic Regulator Hec1 as a Potential Target for Developing Breast Cancer Therapeutics
5. Advances in Liposome-based Targeted Gene Therapy of Cancer
6. Re-wiring the Intracellular Signaling Network in Cancer
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Research and Markets: Recent Advances in Cancer Research and Therapy - Increased Research on one of the Major Causes ...
Gene Ray, Founder of $2.6 Billion Titan Corp, Invests in Medistem's ERC Technology and Joins Advisory Board
SAN DIEGO, CA--(Marketwire -04/25/12)- Medistem Inc. (MEDS.PK - News) announced Dr. Gene Ray, founder of $2.6 Billion Titan Corp, has invested in and joined the Advisory Board for Medistem Inc. Dr. Ray is a Ph.D in Theoretical Physics, Inventor, and Entrepreneur who founded and sold Titan Corp for $2.6 Billion. Dr. Ray has a long track record of success in developing science and technology solutions for the Department of Defense and Homeland Security.
"When Dr. Ichim presented Medistem's story to me two years ago, I was intrigued by the possibility of security and military applications of the Universal Donor adult stem cell that Medistem has discovered," said Dr. Ray. "Since that time, Medistem has obtained FDA approval for clinical trials, numerous publications in peer-reviewed journals with top-notch Universities, and built an experienced management team. When Medistem asked for my help in assisting with strategic issues associated with value optimization and positioning in the arena of military/radioprotectant applications, I was eager to not only offer my expertise, but also to become an investor in the company."
A native of Kentucky, Dr. Ray's academic achievements include: B.S. in Mathematics, Physics & Chemistry, Murray State University, M.S. in Physics and Ph.D in Theoretical Physics, University of Tennessee. In addition to founding Titan Corporation, Dr. Ray has served as Executive Vice President at SAIC, Chief of the Strategic Division of the USAF and as a defense industry analyst. He is currently Chairman of the Board of Decision Sciences on the Board of Artel Corporation, and Chairman of the Cardiology Advisory Board at Scripps Clinic.
Medistem's lead product, the Endometrial Regenerative Cell (ERC), is a stem cell that is more economical to produce, due to its unique origin, and studies suggest it is more effective than other stem cell sources. In addition to the ongoing 60 patient double-blind RECOVER-ERC clinical trial for Heart Failure and its FDA approved Critical Limb Ischemia trial, the company has been investigating possibility of using its new stem cell in protection from radiation injury. Medistem filed patent number 61/625657 covering use of ERC for radiation protection. Dr. Ray's experience working with The Department of Defense and Homeland Security will significantly help Medistem position itself for potential military application of ERCs for radiation exposure.
"I have followed the work of Dr. Ray for some time now with great admiration," said Thomas Ichim, CEO of Medistem. "There are few people in the world that have the unique combination of being a genuine innovator while at the same time having the talent to transform ideas into return on investment to shareholders. Dr. Ray has successfully optimized value in businesses he founded ranging from novel computer systems, to translation services, to food pasteurization, and more recently to detection of nuclear material using muon-based technology. This ability to seamlessly transverse across disciplines, while finding means of successful commercialization, suggested to us that Dr. Ray will add great value to the breadth of applications that our stem cell product may be used for."
Commercialization of therapies for radioprotection fall under the "Animal Efficacy" rule developed by the U.S. Food and Drug Administration (FDA) in 2002 which eliminates the requirement for Phase II and Phase III clinical trials. Under this rule marketing approval is based upon efficacy studies in representative animal species and only Phase I safety data is needed. Medistem believes safety data from its current clinical trials will be sufficient in combination with animal efficacy data, thereby making the commercialization of these therapies ready for market quickly.
"We believe that Dr. Ray will be of great assistance in positioning us in the expanding market for stem cell-based therapeutics," said Dr. Vladimir Bogin, Chairman of Medistem. "Development of novel radioprotectants in the area of cell therapy has attracted significant defense interest. The company Osiris received a $4.2 million upfront grant for large animal studies along with a procurement order of $224.7 million (http://investor.osiris.com/releasedetail.cfm?releaseid=284617) while Cellarant last year received a $153 million award for development and stockpiling of their hematopoietic progenitor cells from the Biomedical Advanced Research and Development Authority (BARDA) for use in radiation sickness (http://www.cellerant.com/pr_090110.html). We plan to seek similar contracts/grants for our ongoing work in this space."
"It is our honor to have Dr. Ray personally invest in and join the Medistem Family. Through leveraging his experience, wisdom, and ingenuity, we are confident in accelerating the process of value optimization for our shareholders," said Vladimir Zaharchook, Vice Chairman and Vice President of Medistem.
About Medistem Inc. Medistem Inc. is a biotechnology company developing technologies related to adult stem cell extraction, manipulation, and use for treating inflammatory and degenerative diseases. The company's lead product, the endometrial regenerative cell (ERC), is a "universal donor" stem cell being developed for critical limb ischemia and heart failure. A publication describing the support for use of ERC for this condition may be found at http://www.translational-medicine.com/content/pdf/1479-5876-6-45.pdf.
Cautionary Statement This press release does not constitute an offer to sell or a solicitation of an offer to buy any of our securities. This press release may contain certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking information. Factors which may cause actual results to differ from our forward-looking statements are discussed in our Form 10-K for the year ended December 31, 2007 as filed with the Securities and Exchange Commission.
Mayo Clinic identifies gene critical to development and spread of lung cancer
Public release date: 24-Apr-2012 [ | E-mail | Share ]
Contact: Paul Scotti scotti.paul@mayo.edu 904-953-2299 Mayo Clinic
JACKSONVILLE, Fla. A single gene that promotes initial development of the most common form of lung cancer and its lethal metastases has been identified by researchers at Mayo Clinic in Florida. Their study suggests other forms of cancer may also be driven by this gene, matrix metalloproteinase-10 (MMP-10).
The study, published in the journal PLoS ONE on April 24, shows that MMP-10 is a growth factor secreted and then used by cancer stem-like cells to keep themselves vital. These cells then drive lung cancer and its spread, and are notoriously immune to conventional treatment.
The findings raise hope for a possible treatment for non-small cell lung cancer, the leading cause of U.S. cancer deaths. Researchers discovered that by shutting down MMP-10, lung cancer stem cells lose their ability to develop tumors. When the gene is given back to the cells, they can form tumors again.
The power of this gene is extraordinary, says senior investigator Alan Fields, Ph.D., the Monica Flynn Jacoby Professor of Cancer Research within the Department of Cancer Biology at Mayo Clinic in Florida.
"Our data provides evidence that MMP-10 plays a dual role in cancer. It stimulates the growth of cancer stem cells and stimulates their metastatic potential,'' he says. "This helps explain an observation that has been seen in cancer stem cells from many tumor types, namely that cancer stem cells appear to be not only the cells that initiate tumors, but also the cells that give rise to metastases."
Dr. Fields says the findings were unexpected, for several reasons.
The first is that the cancer stem cells express MMP-10 themselves, and use it for their own growth. Most of the known members of the matrix metalloproteinase genes are expressed in the tumor's microenvironment, the cells and tissue that surround a tumor, he says. The enzymes produced by these genes are involved in breaking down the microenvironment that keeps a tumor in place, allowing cancer cells to spread, which is why other genes in this family have been linked to cancer metastasis.
"The fact that a gene like MMP-10, which codes for a matrix metalloproteinase that has been linked to metastasis, is actually required for the growth and maintenance of cancer stem cells is very surprising. One would not have predicted that such a gene would be involved in this process," Dr. Fields says.
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Mayo Clinic identifies gene critical to development and spread of lung cancer
Scientists identify 'critical' gene for development and spread of lung cancer
Scientists from the Mayo Clinic have identified a single gene that appears to be a major driving force in the development and spread of the most common form of lung cancer. The study also suggested the gene may play a role in a number of other different types of cancer.
Matrix metalloproteinase-10 (MMP-10) is a growth factor gene used by cancer stem-like cells to keep themselves healthy, as well as migrate into the bloodstream or lymph nodes. Thanks in part to MMP-10, these stem cells are highly resistant to cancer treatments.
This family of [MMP] genes have been implicated for a long time in the process of metastasis the ability of tumors to migrate out of the primary site and survive and move to a distal site, Dr. Alan P. Fields, the Monica Flynn Jacoby Professor of Cancer Research at Mayo Clinic in Florida, told FoxNews.com.
According to Fields, metastasis depends on the ability of MMP genes to degrade the stroma or the environment surrounding the tumor. The stroma normally provides structure for tissues and acts as a barrier against cancer cells.
However, it was a surprise to researchers when they found that MMP was not only involved in metastasis, but also in the earliest stages of tumor growth.
In a mouse model of lung cancer, when we inhibited MMP, we found these animals were deficient in their ability to initiate tumors when we attempted to activate tumor formations, Fields said. We expected the tumors would form, but not progress to the point of metastasizing. But the tumors never started growing.
The study suggested it was the overexpression of MMP-10 specifically that drives the cancer stem cells. In normal tissues, the amount of MMP-10 is very low, but in cancerous tissues it is expressed much more highly. Besides lung cancer, MMP-10 is also suspected to play a role in colorectal, breast, prostate, ovarian and kidney cancers, as well as melanoma and renal cell carcinoma.
The finding suggests drugs or compounds that inhibit MMP-10 activity could be effective as anti-tumor agents with the potential to prevent the spread of tumors or even cause them to regress.
Current cancer treatments, such as chemotherapy, target the cells that make up the bulk of a tumor. But because the cancer-driving stem cells are left intact, the cancer can and often does return.
Tumor stem cells are very resistant to these therapeutic agents, so they remain at the site of the tumor even as the tumor regresses and the patient goes it to remission, Fields explained. Then what happens is you see a relapse or recurrence of the tumor due to these stem cells.
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Scientists identify 'critical' gene for development and spread of lung cancer
A striking link is found between the Fragile-X gene and mutations that cause autism
Public release date: 25-Apr-2012 [ | E-mail | Share ]
Contact: Peter Tarr tarr@cshl.edu 516-367-8455 Cold Spring Harbor Laboratory
Cold Spring Harbor, NY A team led by scientists at Cold Spring Harbor Laboratory (CSHL) publishes research today indicating a striking association between genes found disrupted in children with autism and genes that are targets of FMRP, the protein generated by the gene FMR1, whose dysfunction causes Fragile-X syndrome. The new study appears online April 25 in the journal Neuron.
Fragile-X syndrome is the most common cause of inherited intellectual disability. It is also counted among the autism spectrum disorders (ASDs) owing to the co-occurrence of autism-like symptoms in patients. A usually devastating disorder, Fragile X occurs when the FRM1 gene fails to direct nerve cells to manufacture FMRP, the FMR1-encoded protein, which plays a vital role in neural development and synaptic plasticity.
"A surprising proportion -- up to 20% -- of the candidate genes we see in our sample drawn from 343 autism families appear to be regulated by FMRP," says CSHL Research Investigator Dr. Michael Ronemus, co-first author of the new study. "Because of research connecting FMRP to the phenomenon of neuroplasticity, our work indicates a possible convergence of mechanisms causing autism," adds CSHL Professor Michael Wigler, the senior author of the study. Neuroplasticity is the process by which our brains become sensitized and desensitized to repetitive inputs.
Besides team leader Wigler, a geneticist, others who worked on the study included CSHL sequencing authority Dr. W. Richard McCombie, a sequencing team at Washington University, St. Louis, and Drs. Michael Schatz, Ivan Iossifov and Dan Levy of CSHL, all computational biologists.
Families studied by the team were part of the Simons Simplex Collection. This collection is comprised of "simplex" autism families: those with at least two children, only one of whom has autism spectrum disorder or ASD. In such cases, disease causation has been previously linked to de novo, or spontaneously occurring, gene mutations.
The new research reached its conclusions based on full exome sequencing of each family member's genome. The exome is the portion of the genome less than 2% of the total -- in which DNA encodes proteins. By studying simplex families, researchers seek to discover, among other things, the fraction of autism caused by gene mutations that are not inherited.
This study focused on small-scale de novo mutations changes in DNA as small as a single DNA "letter" relative to the normal sequence, and small insertions or deletions as large as 10 or 15 letters. In the majority of children with small de novo mutations those mutations traced to the father's germ cells (sperm), in an age-dependent manner. Thus, the children of older parents were more likely to have the sorts of mutations that can cause autism.
There are many types of gene mutations, some more likely to result in biological dysfunction than others. One of the most important findings of this study concerned the type of small-scale de novo mutations most likely to play a causative role in autism. "We found that those small-scale de novo changes that disrupt genes in other words, those that disrupt the production of a normal length protein -- are twice as frequent in affected vs. unaffected children," says CSHL Assistant Professor Ivan Iossifov, a quantitative biologist who is the study's other co-first author. (In technical terms, these "disrupting" mutations include frame-shift, splice-site, and nonsense mutations, but not missense mutations.)
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A striking link is found between the Fragile-X gene and mutations that cause autism
Gene critical to development and spread of lung cancer identified
ScienceDaily (Apr. 24, 2012) A single gene that promotes initial development of the most common form of lung cancer and its lethal metastases has been identified by researchers at Mayo Clinic in Florida. Their study suggests other forms of cancer may also be driven by this gene, matrix metalloproteinase-10 (MMP-10).
The study, published in the journal PLoS ONE on April 24, shows that MMP-10 is a growth factor secreted and then used by cancer stem-like cells to keep themselves vital. These cells then drive lung cancer and its spread, and are notoriously immune to conventional treatment.
The findings raise hope for a possible treatment for non-small cell lung cancer, the leading cause of U.S. cancer deaths. Researchers discovered that by shutting down MMP-10, lung cancer stem cells lose their ability to develop tumors. When the gene is given back to the cells, they can form tumors again.
The power of this gene is extraordinary, says senior investigator Alan Fields, Ph.D., the Monica Flynn Jacoby Professor of Cancer Research within the Department of Cancer Biology at Mayo Clinic in Florida.
"Our data provides evidence that MMP-10 plays a dual role in cancer. It stimulates the growth of cancer stem cells and stimulates their metastatic potential,'' he says. "This helps explain an observation that has been seen in cancer stem cells from many tumor types, namely that cancer stem cells appear to be not only the cells that initiate tumors, but also the cells that give rise to metastases."
Dr. Fields says the findings were unexpected, for several reasons.
The first is that the cancer stem cells express MMP-10 themselves, and use it for their own growth. Most of the known members of the matrix metalloproteinase genes are expressed in the tumor's microenvironment, the cells and tissue that surround a tumor, he says. The enzymes produced by these genes are involved in breaking down the microenvironment that keeps a tumor in place, allowing cancer cells to spread, which is why other genes in this family have been linked to cancer metastasis.
"The fact that a gene like MMP-10, which codes for a matrix metalloproteinase that has been linked to metastasis, is actually required for the growth and maintenance of cancer stem cells is very surprising. One would not have predicted that such a gene would be involved in this process," Dr. Fields says.
The researchers also did not expect to find that cancer stem cells produce much more MMP-10 than do the rest of the cells that make up the bulk of the tumor.
"MMP-10 acts to keep these cancer stem cells healthy and self renewing, which also helps explain why these cells escape conventional chemotherapy that might destroy the rest of the tumor," Dr. Fields says. "That is why lung cancer often recurs after treatment, and why its spread to other parts of the lung, as well as nearby lymph nodes, the brain, liver and spinal cord can't be stopped."
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Gene critical to development and spread of lung cancer identified
Nano nod for lab-on-a-chip
The Domino technology uses a plastic chip that can perform 20 genetic tests from a single drop of blood.
You wouldn't know it from appearances, but a metal cube the size of a toaster, created at the University of Alberta, is capable of performing the same genetic tests as most fully equipped modern laboratoriesand in a fraction of the time.
At its core is a small plastic chip developed with nanotechnology that holds the key to determining whether a patient is resistant to cancer drugs or has viruses like malaria. The chip can also pinpoint infectious diseases in a herd of cattle.
Talk about thinking outside the box.
Dubbed the Domino, the technologydeveloped by a U of A research teamhas the potential to revolutionize point-of-care medicine. The innovation has also earned Aquila Diagnostic Systems, the Edmonton-based nano startup that licensed the technology, a shot at $175,000 as a finalist for the TEC NanoVenturePrize award.
Were basically replacing millions of dollars of equipment that would be in a conventional, consolidated lab with something that costs pennies to produce and is field portable so you can take it where needed. Thats where this technology shines, said Jason Acker, an associate professor of laboratory medicine and pathology at the U of A and chief technology officer with Aquila.
The Domino employs polymerase chain reaction technology used to amplify and detect targeted sequences of DNA, but in a miniaturized form that fits on a plastic chip the size of two postage stamps. The chip contains 20 gel postseach the size of a pinheadcapable of identifying sequences of DNA with a single drop of blood.
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Thats the real value propositionbeing able to do multiple tests at the same time, Acker said, adding that the Domino has been used in several recently published studies, showing similar accuracy to centralized labs.
The Domino effect: Personalized medicine
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Nano nod for lab-on-a-chip
Pathway Genomics Adds Prominent Bioinformatics Experts to Scientific Advisory Board
SAN DIEGO--(BUSINESS WIRE)--
Pathway Genomics Corporation, a genetic testing laboratory specializing in nutrition and exercise response, inherited disease, prescription drug response and health condition risks, has established a world-leading scientific advisory board. Among the board members are James Fowler, Ph.D., professor of medical genetics at UCSD School of Medicine, Christoph Lange, Ph.D., associate professor of biostatistics at Harvard University School of Public Health, and Nicholas Schork, Ph.D., director of bioinformatics and biostatistics at the Scripps Translational Science Institute.
Additionally, the companys internal computational and bioinformatics team is led by Lixin Zhou, Ph.D., former senior scientist at Illumina and former collaborative bioinformatics investigator at The Institute for Genomic Research, an organization of The J. Craig Venter Institute.
Working with innovators in specific and technical fields helps Pathway bring highly accurate, useful and actionable information to physicians and their patients, said Michael Nova, M.D., Pathway Genomics chief medical officer. Were committed to seeking out this actionable genetic information through computational biology methods, and cloud-based bioinformatics.
An acclaimed behavioral geneticist, James Fowler, Ph.D., is currently a professor of medical genetics at UCSD School of Medicine, and is world-renowned for his breakthrough discoveries in genetics and social networking, behavioral economics, cooperation, and political behavior.
Christoph Lange, Ph.D., is an assistant professor of medicine at Harvard Medical School and an associate professor of biostatistics at Harvard School of Public Health. Langes current research interests fall into the broad areas of statistical genetics and generalized linear models.
Nicholas J. Schork, Ph.D., is a professor at The Scripps Research Institute in the department of molecular and experimental medicine and director of bioinformatics and biostatistics at the Scripps Translational Science Institute. Schorks research focuses are in quantitative human genetics and integrated approaches to complex biological and medical problems. He has published over 350 scientific articles and book chapters analyzing complex, multifactorial traits and diseases.
Pathways scientific advisory board consists of 10 leaders in various fields including behavioral genetics, bioinformatics, biostatistics, endocrinology, human epigenetics, metabolism, nutrigenomics, nutrition, obesity and exercise genetics, oncology, and weight management. To view the companys full scientific advisory board, visit http://www.pathway.com/sab.
About Pathway Genomics
Pathway Genomics owns and operates an on-site genetic testing laboratory that is accredited by the College of American Pathologists (CAP), accredited in accordance with the U.S. Health and Human Services Clinical Laboratory Improvement Amendments (CLIA) of 1988, and licensed by the state of California. Using only a saliva sample, the company incorporates customized and scientifically validated technologies to generate personalized reports, which address a variety of medical issues, including an individuals carrier status for recessive genetic conditions, food metabolism and exercise response, prescription drug response, and propensity to develop certain diseases such as heart disease, type 2 diabetes and cancer. For more information about Pathway Genomics, visit http://www.pathway.com.
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Pathway Genomics Adds Prominent Bioinformatics Experts to Scientific Advisory Board
Genetic variants, tobacco exposure and lung cancer risk
Public release date: 25-Apr-2012 [ | E-mail | Share ]
Contact: Zachary Rathner Zachary.Rathner@oup.com 301-841-1286 Journal of the National Cancer Institute
There is an association between the rs1051730-rs16969968 genotype and objective measures of tobacco exposure, which indicates that lung cancer risk is largely, if not entirely, mediated by level of tobacco exposure, according to a study published April 25 in the Journal of the National Cancer Institute.
The rs1051730-rs16969968 genotype is known to be associated with heaviness of smoking, lung cancer risk, and other smoking-related outcomes. Prior studies have generally depended on self-reported smoking behavior, which may have underestimated associations and masked the contribution of heaviness of smoking to the associations of these polymorphisms with lung cancer and other health outcomes.
In order to determine the association between the rs1051730-rs16969968 genotype and self-reported cigarette consumption and plasma or serum cotinine levels, Marcus R. Munaf, Ph.D., of the School of Experimental Psychology at the University of Bristol and colleagues, examined data from six independent studies that looked at self-reported daily cigarette consumption and plasma or serum cotinine levels among cigarette smokers and conducted a meta-analysis of pooled per-allele effects. In addition, the researchers looked at the link between the genotypes and lung cancer risk using published data on the association between cotinine levels and lung cancer risk.
The researchers found that the rs1051730-rs16969968 genotype is strongly associated with tobacco exposure measured through cotinine levels, and that the association is strong even after adjustment for self-reported cigarette consumption. "These data therefore support the conclusion that association of rs1051730-rs16969968 genotype with lung cancer risk is mediated largely, if not wholly, via tobacco exposure," the researchers write.
The researchers point out certain limitations of the study, however, namely that the data were drawn from disparate studies from various populations. The data also relies on current smoking measures, rather than lifetime exposure, which is more strongly associated with lung cancer risk.
However, they have confidence in their results, which show that phenotype precision is important to uphold in GWAS studies, rather than ever-larger sample sizes, they say. "The use of objective measures of smoking behavior in genome-wide studies may reveal novel variants associated with these outcomes, which would be undetectable using conventional self-report measures."
In an accompanying editorial, Margaret R. Spitz, M.D., MPH, of the Department of Molecular and Cellular Biology at the Dan L. Duncan Cancer Center at Baylor College of Medicine, writes that these findings "confirm that cigarettes per day is an imprecise measure of nicotine consumption, and favor the interpretation that the association between these variants and lung cancer is mediated by smoking. But the degree to which the association is mediated by smoking is yet to be determined." They add that more studies, including mouse and cellular models, along with emerging metabolic markers, "may help tease apart the direct and indirect associations of these variants with lung cancer risk."
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Genetic variants, tobacco exposure and lung cancer risk
Genetic Associations with Concussions Discussed by AMSSM Researcher
Newswise ATLANTA, Ga. Thomas R. Terrell, MD presented Prospective Cohort Study of the Association of Genetic Polymorphisms and Concussion Risk and Postconcussion Neurocognitive Deficits in College Athletes at the 21st American Medical Society for Sports Medicine Annual Meeting in Atlanta, Ga. on April 23, 2012.
A multi-center prospective cohort study of over 3,200 college and high school athletes was designed to look at the association of genetic polymorphisms with risk of acute concussion and for an associative link with longer duration of symptoms. Following analysis trying to link certain genetic polymorpisms, those evaluated did not show an association with prospective concussions, although some association was found in a pooled analysis of self-reported and prospective concussions.
Dr. Terrell, a two-time AMSSM Foundation Research Award winner, commented, Although we did not find an association of these genetic factors in association prospectively with concussions, the next segment of our research is to evaluate other genetic factors, particularly for associations with severe or recurrent concussions. He was optimistic about possible associations and said, As we look at further data and expand our numbers of concussions included in the study, part of the Tau gene and other genetic polymorphisms have a link in explaining neurocognitive recovery
The AMSSM annual conference features lectures and research addressing the most challenging topics in sports medicine today including prevention of sudden death, cardiovascular issues in athletes, concussion, biologic therapies, and other controversies facing the field of sports medicine.
More than 1,200 sports medicine physicians from across the United States and 12 countries around the world are attending the meeting.
Dr. Terrell is an Associate Professor at the University of Tennessee Graduate School of Medicine and holds a Certificate of Added Qualification in Sports Medicine.
The AMSSM is a multi-disciplinary organization of sports medicine physicians whose members are dedicated to education, research, advocacy, and the care of athletes of all ages. Founded in 1991, the AMSSM is now comprised of more than 2,000 sports medicine physicians whose goal is to provide a link between the rapidly expanding core of knowledge related to sports medicine and its application to patients in a clinical setting.
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Genetic Associations with Concussions Discussed by AMSSM Researcher
NewLink Genetics Receives Notice of Allowance From USPTO for New Patent Broadly Covering Its D-1MT IDO Pathway Inhibitor
AMES, Iowa, April 24, 2012 (GLOBE NEWSWIRE) -- NewLink Genetics Corporation (Nasdaq:NLNK - News) today announced that the United States Patent & Trademark Office (USPTO) has allowed broad claims to oral pharmaceutical compositions comprising 1-methyl-D-tryptophan (D-1MT) (US Serial No. 12/175,538) and also to oral pharmaceutical compositions comprising 1-methyl-DL-tryptophan (US Serial No. 11/603,291). The company holds exclusive rights to the allowed applications.
"These patents will strengthen our D-1MT franchise in the United States," commented Dr. Charles Link, NewLink's Chairman and CEO, "and should facilitate our ongoing discussions with potential development and marketing partners for this product candidate.
"We have studied D-1MT in multiple single-agent phase 1 studies as well as in separate phase 1 studies evaluating D-1MT in combination with either docetaxel or an autologous dendritic cell vaccine," Noted Dr. Nicholas Vahanian, NewLink's President and Chief Medical Officer. "Data from several of these studies will be presented at the ASCO annual meeting in June."
About D-1MT and inhibition of the IDO pathway
IDO pathway inhibitors, including D-1MT, represent a potential breakthrough approach to cancer therapy using small-molecule, anti-toleragenic product candidates intended to counteract a key mechanism by which tumors evade immune-mediated destruction. IDO is an enzyme that regulates immune response by suppressing T-cell function and enabling local tumor immune escape. Recent studies have demonstrated that IDO is overexpressed in many cancers, within both tumor cells as a direct defense against T-cell attack, and also within antigen presenting cells in tumor draining lymph nodes whereby IDO promotes peripheral tolerance to tumor associated antigens (TAAs). When hijacked by developing cancers in this manner, IDO may facilitate the survival, growth, invasion, and metastasis of malignant cells expressing TAAs that might otherwise be recognized and attacked by the immune system as foreign. D-1MT is currently in multiple Phase 1B/2 studies evaluating the addition of D-1MT to Taxotere in the treatment of breast cancer and the addition of D-1MT to an autologous P-53 dendritic cell vaccine also in the treatment of breast cancer patients. In addition to its clinical D-1MT product candidate, NewLink has an active program directed at discovering and developing other IDO pathway inhibitors.
About NewLink Genetics Corporation
NewLink Genetics Corporation is a biopharmaceutical company focused on discovering, developing and commercializing novel immunotherapeutic products to improve cancer treatment options for patients and physicians. NewLink's portfolio includes biologic and small molecule immunotherapy product candidates intended to treat a wide range of oncology indications. NewLink's product candidates are designed with an objective to harness multiple components of the innate immune system to combat cancer, either as a monotherapy or in combination with current treatment regimens, without incremental toxicity. NewLink's lead product candidate, HyperAcute Pancreas cancer immunotherapy is being studied in a Phase 3 clinical trial in surgically resected pancreatic cancer patients (patient information is available at http://www.pancreaticcancer-clinicaltrials.com). This clinical trial is being performed under a Special Protocol Assessment with the U.S. Food and Drug Administration. NewLink and its collaborators have completed patient enrollment for a Phase 1/2 clinical trial evaluating its HyperAcute Lung cancer immunotherapy product candidate for non-small cell lung cancer and a Phase 2 clinical trial for its HyperAcute Melanoma cancer immunotherapy product candidate. NewLink also is developing d-1-methyltryptophan, or D-1MT, a small molecule, orally bioavailable product candidate from NewLink's proprietary indoleamine (2, 3) dioxygenase, or IDO, pathway inhibitor technology. Through NewLink's collaboration with the National Cancer Institute, NewLink is studying D-1MT in various chemotherapy and immunotherapy combinations in two Phase 1B/2 safety and efficacy clinical trials. For more information please visit http://www.linkp.com.
Safe Harbor Statement
This press release contains "forward-looking statements" for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the issuance of, and protection provided by, the above described patents (U.S. Serial Nos. 12/175,538 and 11/603,291) and statements regarding the potential for the patents to facilitate development, marketing and partnering efforts. Such statements are based on management's current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: the protection and market exclusivity provided by the Company's intellectual property; risks related to the drug discovery and the regulatory approval process; and, the impact of competitive products and technological changes. These and other factors are identified and described in more detail in the Company's filings with the Securities and Exchange Commission, including without limitation the Company's annual report on Form 10-K for the year ended December 31, 2011, as amended, and subsequent filings. The Company disclaims any intent or obligation to update these forward-looking statements.
Seattle Genetics to Host Conference Call and Webcast Discussion of First Quarter 2012 Financial Results on May 8, 2012
BOTHELL, Wash.--(BUSINESS WIRE)--
Seattle Genetics, Inc. (SGEN - News) announced today that it will report its first quarter 2012 financial results on Tuesday, May 8, 2012, after the close of financial markets. Following the announcement, company management will host a conference call and webcast discussion of the results and provide a general corporate update. Access to the event can be obtained as follows:
LIVE access on Tuesday, May 8, 2012
1:30 p.m. Pacific Time (PT) / 4:30 p.m. Eastern Time (ET)
REPLAY access
About Seattle Genetics
Seattle Genetics is a biotechnology company focused on the development and commercialization of monoclonal antibody-based therapies for the treatment of cancer. The U.S. Food and Drug Administration granted accelerated approval of ADCETRIS in August 2011 for two indications. ADCETRIS is being developed in collaboration with Millennium: The Takeda Oncology Company. In addition, Seattle Genetics has three other clinical-stage ADC programs: SGN-75, ASG-5ME and ASG-22ME. Seattle Genetics has collaborations for its ADC technology with a number of leading biotechnology and pharmaceutical companies, including Abbott, Bayer, Celldex Therapeutics, Daiichi Sankyo, Genentech, GlaxoSmithKline, Millennium, Pfizer and Progenics, as well as ADC co-development agreements with Agensys, an affiliate of Astellas, and Genmab. More information can be found at http://www.seattlegenetics.com.
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Seattle Genetics to Host Conference Call and Webcast Discussion of First Quarter 2012 Financial Results on May 8, 2012
Will we ever correct diseases before birth? | Not Exactly Rocket Science
Heres the sixth piece from my new BBC column
Every year, millions of people are born with debilitating genetic disorders, a result of inheriting just one faulty gene from their parents. They may have been dealt a dud genetic hand, but they do not have to stick with it. With the power of modern genetics, scientists are developing ways of editing these genetic errors and reversing the course of many hard-to-treat diseases.
These gene therapies exploit the abilities of viruses biological machines that are already superb at penetrating cells and importing genes. By removing their ability to reproduce, and loading them with the genes of our choice, we can transform viruses from causes of disease into vectors for cures.
After a few shaky starts, some of these approaches are beginning to hit their stride. Thirteen children with SCID, an immune disorder that leaves people fatally vulnerable to infections, now have working immune systems. Several British patients with haemophilia, which prevents their blood from clotting properly, can now produce a clotting protein called factor IX, which they once had to inject. A British man and three Americans with inherited forms of progressive blindness can see again.
It is still early days as far as trumpeting gene therapy cures are concerned, but even if they do succeed there is still one significant limitation that cannot be overlooked. Treating adults and children in this way will do for some disorders, but genetic disorders cause irreparable organ damage, or even death, very early. With some of the diseases that we look at, five years old is too late. Sometimes, you dont get to the age of five, says Simon Waddington from University College London. Every single one is a little bit niche but when you list them all out, theres quite a lot of them.
To treat such conditions, we need to intervene as early as possible, and this means correcting genetic disorders in the womb. There are advantages to such prenatal gene therapy. Organs that are hard to target after birth, such as airways blocked with mucus in cystic fibrosis patients, may be easier to reach in the womb. Being smaller, foetuses need a relatively smaller amount of delivery vector. And their immune systems are naive, so they are unlikely to mount an immune response to these vectors.
Risk assessment
So far, several teams have tested prenatal gene therapy in animals, including mice, monkeys and sheep. The results have been promising. In several cases, the animals produce decent levels of foreign proteins for many months, and their immune systems tolerate the added genes. Some have even been cured of their diseases.
Despite these successes, the research has reached an impasse. No one has tried prenatal gene therapy in humans, and no clinical trials are in the works. This is understandable. Altering a foetus genes is a sombre prospect, especially as gene therapy is still a relatively immature technology. It hasnt been embedded enough yet, says Waddington. If these treatments can prove their safety and effectiveness in adults, the field will move towards trials in newborn babies, and from there to prenatal tests.
For now, there are still many potential risks to address. Were still very much looking at which is the right vector to use, says Anna David, from University College London. Lentiviruses and retroviruses (such as HIV) shunt their genes into those of their host. They would seem to provide an ideal way of correcting a faulty gene, either by overwriting it, or providing a cell with working copies.
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Will we ever correct diseases before birth? | Not Exactly Rocket Science
Research and Markets: Breaking the Gene Therapy Barrier for Life-Threatening Red Blood Cell Disorders
DUBLIN--(BUSINESS WIRE)--
Research and Markets (http://www.researchandmarkets.com/research/xvg9rk/breaking_the_gene) has announced the addition of GlobalData's new report "Breaking the Gene Therapy Barrier for Life-Threatening Red Blood Cell Disorders" to their offering.
Breaking the Gene Therapy Barrier for Life-Threatening Red Blood Cell Disorders takes a look at gene therapy for the treatment of blood disorders such as beta-thalassemia and sickle cell anemia. An international team of researchers led by scientists at Weill-Cornell Medical College in New York City have designed a novel gene therapy strategy with potentially curative implications for patients with beta-thalassemia and sickle cell anemia. This development represents an important advance in addressing significant unmet market needs regarding the future development of therapeutic products for these two serious red blood cell disorders.
Scope
- Introduction and detail on two red blood cell disorders - sickle cell anemia and beta-thalassemia
- Current gene therapy initiatives for beta-thalassemia and sickle cell anemia
- Challenges to Gene Therapy
- Market Implications
Reasons to buy
- Understand the effectiveness of gene therapy in the treatment of red blood cell disorders
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Research and Markets: Breaking the Gene Therapy Barrier for Life-Threatening Red Blood Cell Disorders
Raiders' Rolando McClain: Stem-cell therapy worked well
Oakland Raiders linebacker Rolando McClain turned to stem-cell therapy to deal with the aches and pains that come with being a professional football player, the Mobile Press-Register reported Sunday.
Reuter: Impact LB prospects
According to the newspaper, McClain, who missed only one game last season but was hampered by knee pain and an ankle injury, had stem cells taken from his own fat and injected into his knee and leg. McClain credits the procedure for helping him do what he needs to do to prepare for the 2012 season.
"It feels a lot better," McClain told the newspaper, adding he has been able to work out "with hardly any pain at all."
Jason R. Williams, the radiologist who performed the procedure in Alabama, called the experimental therapy "the future of medicine." However, the newspaper also reported that the FDA has warned consumers about the possible pitfalls of stem-cell treatments.
"There is a potential safety risk when you put cells in an area where they are not performing the same biological function as they were when in their original location in the body," said Stephanie Simek, deputy director of the FDA's Office of Cellular, Tissue and Gene Therapies, in a statement.
The newspaper reported that McClain and fellow University of Alabama product Marquis Maze have sought the treatment. Peyton Manning, who recently joined the Denver Broncos after missing the 2011 season with neck issues, reportedly also had a similar procedure outside of the country.
While McClain might be feeling healthier, he has other issues to contend with this offseason. The Birmingham News reported earlier this month that the two-year NFL veteran settled with an Alabama student who claimed McClain struck him with a vehicle and physically assaulted him.
According to the News, McClain also is facing a May 17 trial after allegedly firing a gun in an altercation with a man in November.
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Raiders' Rolando McClain: Stem-cell therapy worked well
NIH scientists link quickly spreading gene to Asian MRSA epidemic
Public release date: 22-Apr-2012 [ | E-mail | Share ]
Contact: Ken Pekoc niaidnews@niaid.nih.gov 301-402-1663 NIH/National Institute of Allergy and Infectious Diseases
National Institutes of Health (NIH) scientists and their colleagues in China have described a rapidly emerging Staphylococcus aureus gene, called sasX, which plays a pivotal role in establishing methicillin-resistant S. aureus (MRSA) epidemics in most of Asia. Senior author Michael Otto, Ph.D., of NIH's National Institute of Allergy and Infectious Diseases, says these findings illustrate at the molecular level how MRSA epidemics may emerge and spread. Moreover, their study identifies a potential target for novel therapeutics.
MRSA is a leading cause of severe infections that occur predominantly in hospitals. MRSA epidemics happen in waves, with old clones of MRSA bacteria disappearing and new clones emerging, a process whose molecular underpinnings are not fully understood.
Previous data indicated that the sasX gene is extremely rare. Therefore, the researchers were surprised when they analyzed 807 patient samples of invasive S. aureus taken over the past decade from three Chinese hospitals. Their data showed that sasX is more prevalent in MRSA strains from China than previously thought, and the gene's frequency is increasing significantly: From 2003 to 2011, the percentage of MRSA samples containing sasX almost doubled, from 21 to 39 percent.
This finding suggests that the sasX gene is involved in molecular processes that help MRSA spread and cause disease. The researchers determined in laboratory and mouse studies that sasX helps bacteria to colonize in the nose, cause skin abscesses and lung disease, and evade human immune defenses. Further, the scientists say their work provides additional evidence for a long-held theory that the emergence of new clones of highly virulent MRSA bacteria occurs through horizontal gene transfer, the exchange of DNA between different strains. Notably, the sasX gene is embedded in a so-called mobile genetic element, a DNA segment that can transfer easily between strains.
Most sasX-positive samples found in the study were from the ST239 group, the predominant MRSA lineage in China and large parts of Asia. However, because the scientists have already observed the transfer of sasX to MRSA clones other than those belonging to the ST239 group, Dr. Otto and his team predict that the frequency of sasX will increase internationally. They plan to both monitor its spread and work to develop therapeutics to prevent MRSA strains expressing sasX from colonizing and infecting people.
Min Li, Ph.D., associate professor at Fudan University in Shanghai, a former postdoctoral fellow in Dr. Otto's laboratory, designed the study. Other collaborators are from the University of California, San Francisco.
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ARTICLE: M Li et al. MRSA epidemic linked to a quickly spreading colonization and virulence determinant. Nature Medicine DOI: 10.1038/nm.2692 (2012).
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NIH scientists link quickly spreading gene to Asian MRSA epidemic
WSU Prof Completes Genome Sequence of Great Ape Living With Humans
Bonobos at the Cincinnati Zoo. Photo: Greg Hume via Wikimedia Commons.
DETROIT A Wayne State University School of Medicine researcher is one step closer to understanding the genetic basis that enable bonobos, one of humankinds sibling species, to learn language, play music and use rudimentary tools.
Derek Wildman, Ph.D., led a team that isolated the DNA and sequenced the genome, or whole inherited genetic make-up, of Kanzi, a bonobo based at the Bonobo Hope Great Ape Trust Sanctuary in Des Moines, Iowa. The sequencing, only the second of its kind, was performed at both WSU and an off-site private company.
Wildmanis associate professor of Molecular Medicine and Genetics, and of Obstetrics and Gynecology. He is the director of the Molecular Evolution Group at WSUs Center for Molecular Medicine and Genetics. To learn more about the center, visit http://www.homopan.wayne.edu
Kanzi, 32, was raised from birth in a family of five humans and eight bonobos, and was trained to use and understand simple spoken English to communicate.
He also plays music, makes fire, cooks simple meals, and makes and uses flint knives. The goal of sequencing Kanzisgenome is to understand the unique abilities of Kanzi and the other bonobos living at the sanctuary.
We can compare Kanzisgenome to the genomes of humans, and other primates in order to see what is unique about Kanzifrom a genetic perspective, Dr. Wildmansaid. We also can see what Kanzishares with other primates. Because we have also sequenced his transcriptomewe can build gene models that are more accurate, and we can see which genes are expressed in his blood, and in the placenta of Kanzis son, Teco. This is a very important first step in untangling nature from nurture in the cognitive development of bonobos.
Next, the team will compare the genomes and epigenomes of bonobos raised with human language to those that have not had such experiences.
The bonobo is an endangered African great ape found only in the Democratic Republic of Congo. According to the Bonobo Conservation Initiative, bonobos and people share 98 percent of the same genetic make-up. Physically, their anatomy closely resembles Australopithecus, an early human ancestor. Bonobos walk on two feet more easily and for longer periods than other great apes.
The science world acknowledges that bonobos are extraordinarily smart primates.
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WSU Prof Completes Genome Sequence of Great Ape Living With Humans