By RTT News, December 12, 2014, 07:18:00 AM EDT

(RTTNews.com) - Alnylam Pharmaceuticals, Inc. ( ALNY ) announced its pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas, or STArs: Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease. The company said its strategic therapeutic areas will remain focused on liver-expressed and genetically validated or pathogen-derived disease targets, with biomarkers for assessment of clinical activity early in Phase 1 trials.

In Genetic Medicine STAr, Alnylam said it is advancing a broad pipeline of RNAi therapeutics for rare diseases. Across the Genetic Medicine STAr, Alnylam plans on commercializing its products through direct marketing and sales in the U.S. and EU, while leveraging its landmark partnership with Genzyme, a Sanofi company, for commercialization in the rest-of-world.

In Cardio-metabolic Disease STAr, Alnylam is advancing pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in dyslipidemias, hypertension, non-alcoholic steatohepatitis, and type 2 diabetes. The company intends to seek strategic partnership opportunities for programs in Cardio-metabolic Disease STAr, while retaining significant product commercialization rights in the U.S. and EU.

In Hepatic Infectious Disease STAr, Alnylam is advancing a pipeline of RNAi therapeutics that address major global health challenges, including hepatitis B virus and hepatitis D virus infections, amongst other hepatic infectious disease opportunities. The company intends to seek strategic partnership opportunities for programs in its Hepatic Infectious Disease STAr, while retaining significant product commercialization rights in the U.S. and EU.

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Alnylam Pharma Announces Pipeline Growth Strategy For RNAi Therapeutics

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PUBLIC RELEASE DATE:

12-Dec-2014

Contact: Steve Yozwiak syozwiak@tgen.org 602-343-8704 The Translational Genomics Research Institute

PHOENIX, Ariz. -- Dec. 12, 2014 -- Using a basic genetic difference between men and women, the Translational Genomics Research Institute (TGen) has uncovered a way to track down the source of a neurological disorder in a young girl.

TGen's discovery relies on a simple genetic fact: Men have one X and one Y chromosome, while women have two X chromosomes. This women-only factor was leveraged by TGen investigators to develop a highly accurate method of tracking down a previously unrecognized disorder of the X-chromosome.

The study of a pre-teen girl, who went years with an undiagnosed neurobehavioral condition, was published today in the scientific journal PLOS ONE.

TGen's findings were made within its Dorrance Center for Rare Childhood Disorders, where investigators and clinicians apply the latest tools of genomic medicine to provide answers for parents seeking to identify the disease or disorder affecting their child.

The scientists sequenced, or spelled out in order, the complete genetic codes of DNA and RNA of the girl. Because girls inherit an X chromosome from each of their parents (boys inherit a Y chromosome from their father), they also sequenced her mother and father. On average, about half of all X chromosomes active in a female come from the mother and the other half from the father.

"We now have the tools to significantly accelerate the diagnostic process, reducing the need for children to undergo multiple tests that can be emotionally and physically taxing for the entire family," said Dr. David Craig, TGen's Deputy Director of Bioinformatics, Co-Director of the Dorrance Center and the paper's senior author.

Sequencing would reveal the proportion of X chromosomes, and if disproportionate, whether the more abundant of the two were damaged in some way, which often leads to X-linked genetic conditions.

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New TGen test uses the unique genetics of women to uncover neurologic disorders

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GENETICS: The REASON Why You Will NEVER HAVE LONG HAIR!
Free Giveaways Weekly Updates By Email! http://eepurl.com/Am3Kb Buy My Books, DVDs, and T-Shirts at http://www.howtoblackhairstore.com My Website: http://www.howtoblackhair.com Twitter:...

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Genetics - Cytogenetic maps part 6 of 6 Updated
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Media reports about behavioural genetics unintentionally induce unfounded beliefs, therefore going against the educational purpose of scientific reporting, writes the University of Montreals Alexandre Morin-Chass, following his study of 1,500 Americans. Among other things, we wanted to know if the public understood (or misunderstood) popular science articles about a new research field, genopolitics, and whether this popularization indeed helped people have an informed opinion on human genetics, Morin-Chass explained.

The study participants first had to read a news article about research on the influence of a gene on one of the following three traits: breast cancer, political ideology (liberal or conservative), or the tendency to go into debt. After reading the article assigned to them, they were then asked to estimate the influence of genetics on various biological (e.g., hair colour, height) or behavioural (e.g., violence, alcoholism) traits on a scale from 0% genetic to 100% genetic. They were told that there were no right or wrong answers. The purpose of the study was simply to examine the interpretation of facts.

The conclusions were troubling, to say the least. Morin-Chass observed that after reading an article published in the British Daily Telegraph in October 2010 about a "gene responsible for liberal ideas, the readers tended to generalize the influence of genetics to other behaviours or social orientations of which there was no mention in the news article (including sexual orientation and intelligence). The same phenomenon was observed among the readers of the other article, originally published in the Scientific American Mind magazine in June 2010, which associated a gene with susceptibility to debt.

However, public misunderstanding is not the only thing to blame for this misinterpretation. Generally, science reporters first goal is to inform the public about scientific developments. However, this practice is not disinterested; some news is purposely written in a manner intended to catch the publics attention with startling results in order to increase or to maintain market shares," Morin-Chass explained.

Genetic research into behaviour is certainly a minefield. It is often associated with other more controversial theoretical work, for example, in sociobiology, which attempts to explain social inequalities using the theory of evolution and the concept of natural selection. In contrast, current trends in research are based on empirical analysis of DNA data.

"Personally, I am in favour of this innovative approach to better understand our world, but I cant argue with the facts: the field is often misunderstood or even disregarded, Morin-Chass said. Some reduce it to its most deterministic form. The danger, which, in my mind, is present, is that scientific research findings could be manipulated for ideological purposes by certain social groups. Hence the importance of making sure the public understands the scope and limitations of such research.

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The above story is based on materials provided by Universit de Montral. Note: Materials may be edited for content and length.

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Current practices in reporting on behavioural genetics can mislead the public

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Rationale for Using Gene Therapy in B-Thalassemia Major and Sickle Cell Anemia
In this exclusive interview, Marina Cavazzana, MD, PhD, of Hpital Universitaire Necker -Enfants Malades, Paris, France, talks about the logic reasoning behi...

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Regulatory and market access hurdles in Europe for Molecular diagnostics biomarkers
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Unlocking the secrets of stem cell generation
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TORONTO A Canadian-led international team of researchers has begun solving the mystery of just how a specialized cell taken from a persons skin is reprogrammed into an embryonic-like stem cell, from which virtually any other cell type in the body can be generated.

The research is being touted as a breakthrough in regenerative medicine that will allow scientists to one day harness stem cells to treat or even cure a host of conditions, from blindness and Parkinsons disease to diabetes and spinal cord injuries.

Besides creating the reprogramming roadmap, the scientists also identified a new type of stem cell, called an F-class stem cell due to its fuzzy appearance. Their work is detailed in five papers published Wednesday in the prestigious journals Nature and Nature Communications.

Dr. Andras Nagy, a senior scientist at Mount Sinai Hospital in Toronto, led the team of 50 researchers from Canada, the Netherlands, South Korea and Australia, which spent four years analyzing and cataloguing the day-by-day process that occurs in stem cell reprogramming.

The work builds on the 2006-2007 papers by Shinya Yamanaka, who showed that adult skin cells could be turned into embryonic-like, or pluripotent, stem cells through genetic manipulation, a discovery that garnered the Japanese scientist the Nobel Prize in 2012.

Nagy likened the roughly 21-day process to complete that transformation to a black box, so called because scientists did not know what went on within the cells as they morphed from one cell type into the other.

It was just like a black box, Nagy said Wednesday, following a briefing at the hospital. You start with a skin cell, you arrive at a stem cell but we had no idea what was happening inside the cell.

Nagys team set about cataloguing the changes as they occurred by removing cells from culture dishes at set points during the three-week period, then analyzing such cellular material as DNA and proteins present at that moment.

The result is a database that will be available to scientists around the world, which the team hopes will spur new research to advance the field of stem cell-based regenerative medicine.

Co-author Ian Rogers, a scientist in Nagys lab, said the database will allow researchers to identify various properties of the developing stem cells, which could mean improving their ability to treat or cure disease.

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Canadian-led team of researchers shows how stem cells can be reprogrammed

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TORONTO A Canadian-led international team of researchers has begun solving the mystery of just how a specialized cell taken from a persons skin is reprogrammed into an embryonic-like stem cell, from which virtually any other cell type in the body can be generated.

The research is being touted as a breakthrough in regenerative medicine that will allow scientists to one day harness stem cells to treat or even cure a host of conditions, from blindness and Parkinsons disease to diabetes and spinal cord injuries.

Besides creating the reprogramming roadmap, the scientists also identified a new type of stem cell, called an F-class stem cell due to its fuzzy appearance. Their work is detailed in five papers published Wednesday in the prestigious journals Nature and Nature Communications.

Dr. Andras Nagy, a senior scientist at Mount Sinai Hospital in Toronto, led the team of 50 researchers from Canada, the Netherlands, South Korea and Australia, which spent four years analyzing and cataloguing the day-by-day process that occurs in stem cell reprogramming.

The work builds on the 2006-2007 papers by Shinya Yamanaka, who showed that adult skin cells could be turned into embryonic-like, or pluripotent, stem cells through genetic manipulation, a discovery that garnered the Japanese scientist the Nobel Prize in 2012.

Nagy likened the roughly 21-day process to complete that transformation to a black box, so called because scientists did not know what went on within the cells as they morphed from one cell type into the other.

It was just like a black box, Nagy said Wednesday, following a briefing at the hospital. You start with a skin cell, you arrive at a stem cell but we had no idea what was happening inside the cell.

Nagys team set about cataloguing the changes as they occurred by removing cells from culture dishes at set points during the three-week period, then analyzing such cellular material as DNA and proteins present at that moment.

The result is a database that will be available to scientists around the world, which the team hopes will spur new research to advance the field of stem cell-based regenerative medicine.

Co-author Ian Rogers, a scientist in Nagys lab, said the database will allow researchers to identify various properties of the developing stem cells, which could mean improving their ability to treat or cure disease.

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Researchers show how stem cells can be reprogrammed

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Scientists at UB and other institutions have turned cells normally used as model cells, known as immortalized cells, into stem or, as they call it, stem-like cells, using nothing more than mechanical stress. They have done it without employing the potentially hazardous techniques previously used to obtain similar results.

The researchers use the term stem-like cells to describe cells in tissue culture that have many of the biochemical markers of stem cells. Determining whether or not they can differentiate will be the focus of future research.

The finding is described in a paper published recently online before print in the Proceedings of the National Academy of Sciences. The researchers discovered that changing the mechanical stresses on neuronal and other cell types in tissue culture allowed them to be reprogrammed into stem-like cells.

Normal cell types in tissue culture are spread out and have differentiated internal structures, but changing cell mechanics caused the cells to turn into clusters of spherical cells that had many of the biochemical markers of cells, says Frederick Sachs, SUNY Distinguished Professor in the Department of Physiology and Biophysics and senior author.

The stem cell advance was made possible by the development of a genetically encoded optical probe by Fanje Meng, research assistant professor in the Department of Physiology and Biophysics and lead UB author. The probe measures the mechanical stress in actin, a major structural protein present in all cells. Actin is involved in muscle contraction and numerous cellular processes, including cell signaling, how cells are shaped and how they move.

The actin probes will provide researchers with a method of studying how mechanical forces influence living cells, tissues, organs and animals in real time.

This probe allows us, for the first time, to measure the stress in actin within living cells, explains Sachs. We saw gradients of stress in actin filaments even in single living cells.

Much of existing biomechanics will have to be rethought, since many studies have assumed that the stresses are uniform, Sachs continues. The actin stress probe showed that the tension in actin fibers in stem cells is higher than in normal cells. That was very surprising to us.

He adds that while mechanics are well known to have a role in cellular processes, the details are poorly understood because there have been few ways to measure the stress in specific proteins. A clinically relevant example is that metastatic cancer cells, the fatal variety, have different mechanics than cells of the parent tumor.

This probe will allow cancer researchers to better understand what allows cells to become metastatic, says Sachs.

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Mechanical cues reprogram normal cell lines into stem-like cells

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Beverly Hills, CA (PRWEB) December 11, 2014

R3 Stem Cell is proud to welcome Dr. George Graf as a Featured Regenerative Medicine Doctor in the Los Angeles and Beverly Hills area. Dr. Graf is a first rate pain management doctor, who offers several types of stem cell procedures and platelet rich plasma therapy for all types of spinal conditions such as neck and back pain, arthritis, disc degeneration and more. Those interested should call (844) GET-STEM for more information and scheduling.

R3 Stem Cell is a nationwide provider of regenerative medicine products and education for both doctors and patients. The company only works with the top doctors and practices in the field of stem cell therapy. Dr. Graf is Double Board Certified and is very highly regarded by his peers and patients.

The conditions Dr. Graf treats include degenerative disc disease, spinal arthritis, scoliosis, neuropathy, failed back surgery syndrome and more. Regenerative medicine offers the potential to not only bring pain relief, but also help repair and regenerate damaged tissue.

Along with Dr. Graf being a regenerative medicine expert in the LA and Beverly Hills area, R3 also works with Dr. Raj. Dr. Raj is a Double Board Certified orthopedic specialist, who offers regenerative medicine procedures for rotator cuffs, hip and knee arthritis, sports injuries and much more. Between Dr. Graf and Dr. Raj, the whole body is covered for treatments.

All of the treatment options are outpatient and very low risk. Platelet Rich Plasma Therapy involves a person's own blood, which is immediately processed and injected into the problem area. Bone marrow derived stem cell therapy involves an aspiration from one's iliac crest, with the material being processed to concentrate stem cells and then inject into the problem area. Additionally, amniotic stem cells are offered, with the fluid being obtained from a consenting donor undergoing a scheduled C-section. The fluid is processed at an FDA regulated lab and no fetal tissue is involved whatsoever.

To date, several small studies have shown excellent benefit with regenerative medicine procedures. This has been extremely encouraging, and allowed stem cell therapy to exponentially increase in popularity nationwide. R3 Stem Cell is at the forefront in regenerative medicine, teaming with the top doctors such as Drs. Raj and Graf to help patients achieve pain relief and avoid surgery.

Call (844) GET-STEM today for more information and scheduling with a top stem cell doctor today.

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R3 Stem Cell Welcomes Beverly Hills Pain Specialists Dr. George Graf as a Featured Regenerative Medicine Doctor

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Spinal cord injury support: help at the outset
Personal injury cases can take a long time to conclude but there are also lots of ways that Aspire Law can help you in the short and medium term. One of our initial priorities will be to talk...

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Global Regenerative Medicine Market 2015-2019
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Toddlers who undergo total body irradiation in preparation for bone marrow transplantation are at higher risk for a decline in IQ and may be candidates for stepped up interventions to preserve intellectual functioning, St. Jude Children's Research Hospital investigators reported. The findings appear in the current issue of the Journal of Clinical Oncology.

The results clarify the risk of intellectual decline faced by children, teenagers and young adults following bone marrow transplantation. The procedure is used for treatment of cancer and other diseases. It involves replacing the patient's own blood-producing stem cells with those from a healthy donor.

Researchers tracked IQ scores of 170 St. Jude patients before and for five years after transplantation, making this the most comprehensive effort yet to determine how the procedure affects intelligence. The patients ranged in age from 4 months to 23 years when their transplants occurred. The procedure had little lasting impact on the IQ scores of most patients.

"For the great majority of patients, these findings provide reassurance that transplantation will not have a significant negative impact on cognitive development," said corresponding author Sean Phipps, Ph.D., chair of the St. Jude Department of Psychology. "We have also identified a high-risk group of younger patients who may benefit from more intensive interventions, including developmental stimulation and other rehabilitative therapies designed to prevent a decline in intellectual functioning and aid in recovery."

The high-risk group includes patients whose transplants occurred when they were aged 3 years or younger and involved total body irradiation (TBI). TBI is used to prepare patients for transplantation by killing remaining cancer cells and protecting the transplanted cells from their immune systems. TBI is associated with a range of short-term and long-term side effects. At St. Jude, therapeutic advances have significantly reduced the use of TBI in bone marrow transplantations.

Previous studies of bone marrow transplantation survivors reported conflicting results about the long-term impact of age and TBI on cognitive abilities.

Before transplantation, the average IQ scores of all patients in this study were in the normal range. One year after transplantation, average IQ scores of patients aged 5 and younger had declined sharply. But scores of most patients rebounded in subsequent years. Five years after the procedure, IQ scores for most patients, even the youngest survivors, had largely recovered and fell within the range of normal intelligence.

Patients in the high-risk group were the lone exception. IQ scores of patients who were both aged 3 or younger when their transplants occurred and who received TBI failed to recover from the first-year decline. Five years after transplantation, these survivors had average IQ scores in the low-normal range of intelligence. Their scores were more than 16 points lower than the scores of patients who were just as young when their transplants occurred but did not receive TBI.

Of the 72 patients in this study whose transplants included TBI, researchers found there was a long-term impact on intellectual functioning only of patients who were aged 3 or younger at transplantation.

"The significant first-year decline reflects the intensity of transplantation, which our results suggest leads to greater disruption in development in the youngest children than was previously recognized," said the study's first author Victoria Willard, Ph.D., a St. Jude psychology department research associate.

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COPD Treatment Therapy Chronic Disease Herbal Rehab
Click http://www.healthproductsbusiness.com/respiratory/copd/ New COPD treatment helps adult patient chronic obstructive pulmonary disorder disease, breathe more easily, a natural ayurvedic...

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PUBLIC RELEASE DATE:

11-Dec-2014

Contact: John Wallace wallacej@vcu.edu 804-628-1550 Virginia Commonwealth University @vcunews

Targeted therapies are a growing and groundbreaking field in cancer care in which drugs or other substances are designed to interfere with genes or molecules that control the growth and survival of cancer cells. Now, scientists at Virginia Commonwealth University Massey Cancer Center and VCU Institute of Molecular Medicine (VIMM) have identified a novel interaction between a microRNA and a gene that could lead to new therapies for the most common and deadly form of brain tumor, malignant glioma.

In a study recently published in the journal Neuro-Oncology, a team of scientists led by Luni Emdad, M.B.B.S., Ph.D., and Paul B. Fisher, M.Ph., Ph.D., provided the first evidence of an important link between a specific microRNA, miR-184, and a cancer promoting gene, SND1, in the regulation of malignant glioma. miR-184 is known to suppress tumor development by regulating a variety of genes involved in cancer growth, while SND1 has been shown to play a significant role in the development of breast, colon, prostate and liver cancers. Through a variety of preclinical experiments, the team demonstrated that increasing the expression of miR-184 slows the growth and invasive characteristics of glioma cells through direct regulation of SND1. Additionally, they showed that reduced levels of SND1 led to reduced levels of STAT3, a gene that has been shown to promote the most lethal characteristics of brain cancer.

"Patients suffering from brain tumors are in desperate need of improved therapies," says Fisher, Thelma Newmeyer Corman Endowed Chair in Cancer Research and co-leader of the Cancer Molecular Genetics research program at VCU Massey Cancer Center, chairman of the Department of Human and Molecular Genetics at VCU School of Medicine and director of the VIMM. "We're hopeful that this new understanding of the relationship between miR-184 and SND1 ultimately will lead to the development of new drugs that reduce SND1 expression and improve patient outcomes."

Prior studies have shown that levels of miR-184 are unusually low in tissue samples from patients with malignant gliomas. Using advanced computer analysis techniques designed to study and process biological data, the researchers identified SND1 among a handful of other genes that miR-184 helps regulate. Knowing SND1 is implicated in a variety of cancers and having previously defined its role in liver cancer, Emdad, Fisher and their colleagues explored this relationship further. They confirmed low levels of miR-184 expression in human glioma tissue samples and cultured cell lines as well as an increase in the expression of SND1 compared to normal brain tissue. Using data from a large public brain tumor database called REMBRANDT, the researchers confirmed that patients with lower levels of SND1 survived longer than those with elevated SND1 expression.

"We still have a long way to go and many challenges to overcome before we will have therapies that are ready for clinical use, but this is a significant first step in the process," says Emdad, member of the Cancer Molecular Genetics research program at Massey, assistant professor in the VCU Department of Human and Molecular Genetics and member of the VIMM. "Future studies will aim to explore the relationship between SND1 and STAT3, identify additional microRNAs that may be relevant to malignant glioma and explore the effects of drugs that block SND1 expression in more advanced preclinical models."

###

Fisher and Emdad collaborated with Devanand Sarkar, M.B.B.S., Ph.D., Harrison Scholar and member of the Cancer Molecular Genetics research program at Massey, Blick Scholar and associate professor in the Department of Human and Molecular Genetics at the VCU School of Medicine and member of the VIMM; Swadesh K. Das, Ph.D., member of the Cancer Molecular Genetics research program at Massey, member of the VIMM and assistant professor in the VCU Department of Human and Molecular Genetics; Mitchell E. Menezes, Ph.D., Prasanna K. Santhekadur, Ph.D., and Bin Hu, Ph.D., all postdoctoral research scientists in the VCU Department of Human and Molecular Genetics; and Aleksandar Janjic, Mohammad Al-Zubi and Xue-Ning Shen, all research technicians in the VCU Department of Human and Molecular Genetics and the VIMM.

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PUBLIC RELEASE DATE:

11-Dec-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News @LiebertOnline

New Rochelle, NY, December 11, 2014--In cases of traumatic brain injury (TBI), predicting the likelihood of a cranial lesion and determining the need for head computed tomography (CT) can be aided by measuring markers of bone injury in the blood. The results of a new study comparing the usefulness of two biomarkers released into the blood following a TBI are presented in Journal of Neurotrauma, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Neurotrauma website at http://online.liebertpub.com/doi/full/10.1089/neu.2013.3245 until January 11, 2015.

The article "GFAP Out-Performs S100 in Detecting Traumatic Intracranial Lesions on Computed Tomography in Trauma Patients with Mild Traumatic Brain Injury and Those with Extracranial Lesions," describes a study of adult trauma patients with and without mild or moderate TBI. The authors, Linda Papa and colleagues from Orlando Regional Medical Center, North Florida Veteran's Health System and University of Florida (Gainesville), University of Central Florida (Orlando), Banyan Biomarkers Inc. (Alachua, FL), Virginia Commonwealth University (Richmond, VA), and Baylor College of Medicine (Houston, TX), showed that increased blood levels of glial fibrillary acidic protein (GFAP) following TBI was a good predictor of intracranial lesions, whether or not the patient had fractures elsewhere in the body. Whereas S100 levels in the blood of were significantly higher in trauma patients with fractures than without fractures, it was not as useful as GFAP in distinguishing between intracranial and extracranial lesions.

John T. Povlishock, PhD, Editor-in-Chief of Journal of Neurotrauma and Professor, Medical College of Virginia Campus of Virginia Commonwealth University, Richmond, notes that "This is an extremely important paper because of its relatively large sample size and its singular focus upon mild traumatic brain injury complicated by the presence of extracranial lesions. This study convincingly demonstrates the efficacy and brain specific nature of GFAP and its ability to detect traumatic intracranial lesions while also calling into question the overall utility of S100 in the same patient population. Importantly, the superior performance of GFAP in the mild brain injured population is an important observation consistent with other reports emerging in the field. Lastly, the observation that these GFAP elevations occur relatively early in a posttraumatic course speaks to the potential utility of using these biomarkers to screen brain injured patients who then may require more extensive and/or long term imaging studies."

###

About the Journal

Journal of Neurotrauma is an authoritative peer-reviewed journal published 24 times per year in print and online that focuses on the latest advances in the clinical and laboratory investigation of traumatic brain and spinal cord injury. Emphasis is on the basic pathobiology of injury to the nervous system, and the papers and reviews evaluate preclinical and clinical trials targeted at improving the early management and long-term care and recovery of patients with traumatic brain injury. Journal of Neurotrauma is the official journal of the National Neurotrauma Society and the International Neurotrauma Society. Complete tables of content and a sample issue may be viewed on the Journal of Neurotrauma website at http://www.liebertpub.com/neu.

About the Publisher

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Can a biomarker in the blood predict head fracture after traumatic brain injury?

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The Future of Genetic Medicine - Maureen Turner
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CHAPTER 49 - Crohn #39;s disease and genetics IBD Sensor

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Epilepsy and Cerebral Palsy; How Much Genetics? -Dr. Nor Azni Yahya
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