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Archive for May, 2014

A Shot in the Arm for Evolutionary Genetics Research | Pew – Video


A Shot in the Arm for Evolutionary Genetics Research | Pew
http://www.pewscholars.org 2012 Pew biomedical scholar Nels Elde studies the evolution of conflict -- how the process of infection can drive some of the most dramatic adaptations seen in nature....

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A Shot in the Arm for Evolutionary Genetics Research | Pew - Video

Vision for Personalized Medicine: Princess Margaret 2014 Conference – Video


Vision for Personalized Medicine: Princess Margaret 2014 Conference
On Feb. 10-11, 2014, the Princess Margaret Cancer Centre - the largest comprehensive cancer centre in Canada and one of the world #39;s top five - hosted its inaugural conference, "Beyond the Genetic...

By: Princess Margaret Cancer Centre

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Vision for Personalized Medicine: Princess Margaret 2014 Conference - Video

Rick Hansen Difference Makers (2014) – Video


Rick Hansen Difference Makers (2014)
Share your difference making story and help create a barrier-free world. This video was created to support Rick Hansen Foundation #39;s campaign to promote the power of difference making and connect...

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Rick Hansen Difference Makers (2014) - Video

New insight into stem cell development

May 22, 2014

The world has great expectations that stem cell research one day will revolutionize medicine. But in order to exploit the potential of stem cells, we need to understand how their development is regulated. Now researchers from University of Southern Denmark offer new insight.

Stem cells are cells that are able to develop into different specialized cell types with specific functions in the body. In adult humans these cells play an important role in tissue regeneration. The potential to act as repair cells can be exploited for disease control of e.g. Parkinson's or diabetes, which are diseases caused by the death of specialized cells. By manipulating the stem cells, they can be directed to develop into various specialized cell types. This however, requires knowledge of the processes that regulate their development.

Now Danish researchers from University of Southern Denmark report a new discovery that provides valuable insight into basic mechanisms of stem cell differentiation. The discovery could lead to new ways of making stem cells develop into exactly the type of cells that a physician may need for treating a disease.

"We have discovered that proteins called transcription factors work together in a new and complex way to reprogram the DNA strand when a stem cell develops into a specific cell type. Until now we thought that only a few transcription factors were responsible for this reprogramming, but that is not the case", explain postdoc Rasmus Siersbaek, Professor Susanne Mandrup and ph.d. Atefeh Rabiee from Department of Biochemistry and Molecular Biology at the University of Southern Denmark.

"An incredibly complex and previously unknown interplay between transcription factors takes place at specific locations in the cell's DNA, which we call 'hotspots'. This interplay at 'hotspots' appears to be of great importance for the development of stem cells. In the future it will therefore be very important to explore these 'hotspots' and the interplay between transcription factors in these regions in order to better understand the mechanisms that control the development of stem cells", explains Rasmus Siersbaek.

"When we understand these mechanisms, we have much better tools to make a stem cell develop in the direction we wish", he says.

Siersbaek, Mandrup and their colleagues made the discovery while studying how stem cells develop into fat cells. The Mandrup research group is interested in this differentiation process, because fundamental understanding of this will allow researchers to manipulate fat cell formation.

"We know that there are two types of fat cells; brown and white. The white fat cells store fat, while brown fat cells actually increase combustion of fat. Brown fat cells are found in especially infants, but adults also have varying amounts of these cells.

"If we manage to find ways to make stem cells develop into brown rather than white fat cells, it may be possible to reduce the development of obesity. Our findings open new possibilities to do this by focusing on the specific sites on the DNA where proteins work together", the researchers explain.

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New insight into stem cell development

Whiplash headaches 11 months after stem cell therapy by Dr Harry Adelson – Video


Whiplash headaches 11 months after stem cell therapy by Dr Harry Adelson
Neil discusses his outcome 11 months after his stem cell therapy by Dr Harry Adelson for the treatment of his post-whiplash headache syndrome http://www.docereclinics.com.

By: Harry Adelson, N.D.

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Whiplash headaches 11 months after stem cell therapy by Dr Harry Adelson - Video

Bride Saves The Life Of Her Fianc Who Had Only 60 Days To Live By Finding A Stem Cell Donor

Mike Brandon was given just 60 days to find a bone marrow transplant. If he didn't, his leukaemia - cancer of white blood cells - was going to overwhelm his body.

Most people faced with such odds may have given up, but Brandon's fiance, Kate Robertson launched a desperate bid to find a matching donor for her husband-to-be.

The odds paid off: less than a month after Miss Robertson's campaign was launched, a donor has been found.

Anthony Nolan said that her efforts has led to a 650% increase in people joining the bone marrow register. The blood cancer charity said that there was a particular surge among potential donors in the couple's home city of Bristol.

Miss Robertson, 31, said the response has been "astounding".

"It's been an incredibly difficult time so the relief we're feeling is overwhelming," she said.

"A matching donor means that we can go ahead with Mike's bone marrow transplant. We know we have a rocky road ahead as a transplant is a serious procedure, but knowing there is a good match for Mike is a fantastic boost that we desperately needed.

"We are hugely grateful to the selfless person who has stepped forward to help Mike, and to everyone who has pledged to do the same for someone else, by joining the Anthony Nolan register."

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Bride Saves The Life Of Her Fianc Who Had Only 60 Days To Live By Finding A Stem Cell Donor

After cancer rebound, Victoria's little Hannah Day back into life of pain as transplanted stem cells attack her body

The seemingly miraculous recovery of little Hannah Day who rebounded earlier this month after a rare bone marrow transplant cancer free for 60 days has suffered a major setback.

Mother Brooke Ervin said her stem cells, which were transplanted into her daughter on March 19, are attacking her four-year-old daughters body from the inside out, manifesting in a rash and third-degree-like burns.

She has burns to 90 per cent of her body and is now admitted back to [B.C. Childrens] hospital in hopes they can stop it.

Hannah is in immeasurable pain as her family watches, terrified and helpless, Ervin said Wednesday.

Hannah is not responding to oral antibiotics, and steroids being pumped into her body to stop the burning are suppressing her immune system, which is needed to fight off the cancer.

This is such a horrible life she got, a distraught Ervin said.

She has spent most of her life suffering just to stay alive. No one should have to fight so hard, especially an innocent child.

She wants to live so bad and she shows us every day with her fight and will to live, Ervin said. She wont give up and we cant either. We have to hold strong in the hopes one day this will end.

On May 6, Hannah was discharged from hospital in Vancouver after receiving stem cells from her mother in a haploidentical transplant.

Although only a half match, doctors hope Hannahs cells will recognize her moms cells which once protected her in the womb and allow them to kill off cancer cells in Hannahs body.

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After cancer rebound, Victoria's little Hannah Day back into life of pain as transplanted stem cells attack her body

Wyndmoor family remembers son with bone marrow drive at Springfield Township High School

Its an image Angie Lawn said shed never forget.

It was May of 2013, and she was at her 19-year-old sons funeral. PJ had just died after complications related to leukemia. The day was a blur, and she was distraught as she walked out of the service and into the parking lot.

On the periphery of the crowd at the edge of the parking lot was this long line of La Salle boys all standing with their jackets, very respectfully, with their hands folded, she recalled. They were some of the last to leave.

A year later, Angie and her family, who live in Wyndmoor, near La Salle College High Schools campus, are organizing a bone marrow donor drive at Springfield Township High School May 31 in honor of PJ.

Angie described her son as a brainiac who loved writing and computer science. He found pleasure in comic books, movies, TV shows, games like Monopoly and a good cheesesteak, she said. He played guitar and piano, and he was skilled at doing imitations and making people laugh.

He took very little for granted, partly because of the cancer, but partly because thats just who he was, she said.

PJ was 12 years old when he was first diagnosed with leukemia, a cancer of the blood cells.

Angie remembered they were going to get ice cream, and PJ didnt want any because he said his chin was bothering him. To be cautious, they went to the doctor, and a blood test determined it was acute lymphoblastic leukemia.

At that point, PJ was just about to head to St. Genevieves for the seventh grade, but instead he was tutored at CHOP as an inpatient that year. The doctors decided to try a bone marrow transplant, but neither Angie nor her husband, Patrick, were a match for PJ. Siblings have a slightly better chance at being a match, so the Lawns other children were tested: Shannon, a senior now at Springfield Township High School; Sean, a sophomore at the high school; and Owen, a seventh-grader at St. Genevieves currently.

Shannon was 10 years old when she was determined to be a match for her older brother. Continued...

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Wyndmoor family remembers son with bone marrow drive at Springfield Township High School

Stem Cells Market By Application (Regenerative Medicine), By Technology (Acquisition, Sub-Culture), By Product (Adult …

San Francisco, California (PRWEB) May 22, 2014

The global market for stem cells is expected to reach USD 170.15 billion by 2020, according to a new study by Grand View Research, Inc. Growing prevalence of chronic diseases such as cardiovascular and liver disease, diabetes and cancer coupled with the presence of high unmet medical needs in these disease segments is expected to drive market growth during the forecast period. Moreover, increasing government support pertaining to funding R&D initiatives and the growing demand for medical tourism and stem cell banking services is expected to boost the demand for stem cells over the next six years. The future of this market is expected to be driven by opportunities such as the growing global prevalence of neurodegenerative diseases, increasing demand for contract research outsourcing services and the substitution of animal tissues by stem cells in the

The stem cells technology market was valued at USD 12.88 billion in 2013 and is expected to grow at a CAGR of over 12.0% during the forecast period. This market was dominated by the cell acquisitions technology segment in terms of share in 2013 owing to the fact that this technology serves as the foremost step to process involving stem cells culture. The global stem cell acquisition technology market is expected to reach USD 10.88 billion by 2020, growing at a CAGR of over 14.0% over the next six years.

The report Stem Cells Market Analysis By Product (Adult Stem Cells, Human Embryonic Cells, Pluripotent Stem Cells), By Application (Regenerative Medicine, Drug Discovery and Development) And Segment Forecasts To 2020, is available now to Grand View Research customers at http://www.grandviewresearch.com/industry-analysis/stem-cells-market

Request Free Sample of this Report @ http://www.grandviewresearch.com/industry-analysis/stem-cells-market/request

Further key findings from the study suggest:

Browse All Biotechnology Market Reports @ http://www.grandviewresearch.com/industry/biotechnology

For the purpose of this study, Grand View Research has segmented the global stem cells market on the basis of product, application, technology and region:

Latest Reports Published By Grand View Research:

Global Polymethyl Methacrylate (PMMA) Market Expected to Reach USD 10.87 Billion by 2020 (https://www.grandviewresearch.com/industry-analysis/polymethyl-methacrylate-pmma-industry)

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Stem Cells Market By Application (Regenerative Medicine), By Technology (Acquisition, Sub-Culture), By Product (Adult ...

Stem Cell Therapy Provided by Pend Oreille Veterinary Service Helps Local Leonberger Get the Bounce Back in His Step …

Poway, California (PRWEB) May 22, 2014

Zeke was in pain from arthritis caused by an old injury and was facing possible surgery on both knees. Christine Ponsness-Wetzel, DVM, at Pend Oreille Veterinary Service determined that Zeke was a good candidate for stem cell therapy by Vet-Stem, Inc. as an alternative, and just a few months later, he now has a bounce back in his step.

Zeke is a 125-pound Leonberger who lives in Idaho and enjoys going on back country ski trips. Zekes hobbies came to a halt two years ago when he was diagnosed with a partial cruciate ligament tear. He had gone lame and two weeks of rest was recommended, but his owners did not see improvement. After a month of rest, x-rays revealed arthritis had developed in one of Zekes knees.

After a year of pain medications to control the discomfort and pain, Zeke started having more difficulties. He had a delayed ability to comfortably bend his leg, often needed help getting up from a laying position, and would whimper in pain. This time, x-rays would reveal arthritis in both knees. After a few months of increased pain medications and only mild improvement, Zekes owners opted for stem cell therapy with Dr. Ponsness-Wetzel.

Zeke was still quite active and happy, so the thought of double knee surgery and the long recovery time was not in my books, so we opted for stem cell therapy, Zekes owner explains. It has been four months since the stem cell injections (both knees and an IV dose) and Zeke has definitely improved. He no longer needs help getting up. He does not whimper in pain. His delay in bending his knee is non-existent, and his pain medication has been reduced by about 80%. Hikes are no longer sheer drudgery and he has a bounce in his step that I forgot existed.

Pend Oreille Veterinary Services celebrates its 50th anniversary in the Bonner County, providing basic health care services to small animals and reptiles, as well as cutting edge therapies such as acupuncture, laser, and stem cells. Pend Oreille Veterinary Services also offers boarding and grooming to the cities around their two locations in Ponderay and Bonners Ferry. To find out more about Pend Oreille Veterinary Service and Vet-Stem Cell Therapy with Dr. Ponsness-Wetzel, visit http://www.sandpointvets.com.

About Vet-Stem, Inc. Vet-Stem, Inc. was formed in 2002 to bring regenerative medicine to the veterinary profession. The privately held company is working to develop therapies in veterinary medicine that apply regenerative technologies while utilizing the natural healing properties inherent in all animals. As the first company in the United States to provide an adipose-derived stem cell service to veterinarians for their patients, Vet-Stem, Inc. pioneered the use of regenerative stem cells in veterinary medicine. The company holds exclusive licenses to over 50 patents including world-wide veterinary rights for use of adipose derived stem cells. In the last decade over 10,000 animals have been treated using Vet-Stem, Inc.s services, and Vet-Stem is actively investigating stem cell therapy for immune-mediated and inflammatory disease, as well as organ disease and failure. For more on Vet-Stem, Inc. and Veterinary Regenerative Medicine visit http://www.vet-stem.com or call 858-748-2004.

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Stem Cell Therapy Provided by Pend Oreille Veterinary Service Helps Local Leonberger Get the Bounce Back in His Step ...

OHSU Scientist Pushes Forward With Stem Cell Research

Contributed By:

Dave Blanchard

OPB | May 22, 2014 12:06 p.m. | Updated: May 22, 2014 1:51 p.m.

An egg cell's nucleus is extracted by apipette.

OHSU

This March, Oregon Health & Science University (OHSU) created a new Center for Embryonic Cell and Gene Therapy. The facility will be focused in part on advancing the work of Shoukhrat Mitalipov, one of the worlds leading researchers on embryonic stem cells. Mitalipov has been working for years on two promising areas of stem cellscience.

The first research area is a gene therapy for women with diseases stored in DNA located in their mitochondria. Mitalipovs lab has developed a technique to extract the nucleus from a cell with damaged mitochondrial DNA, and implant it in a cell with healthy mitochondria. The process would allow most of the mothers DNA to be inherited by her child, without the risk of the mitochondrial diseases. Mitalipov hopes to begin clinical trials of the procedure, and the FDA is in the process of deciding whether to approve the technique soon. Some critics have ethical and medical concerns about creating an embryo with DNA from three differentpeople.

The second area, which has garnered even more attention, is the field of embryonic stem cell cloning. Last May, Mitalipovs lab became the first team to create human embryonic stem cells by cloning a breakthrough that was highlighted by Nature, Discover, Science, and National Geographic as one of the most significant science stories of the year. Now Miltalipovs lab is trying to figure out how to further that field ofresearch.

Well check in with Mitalipov to hear about his hopes for his areas of research, and where he thinks the future holds for stem cell science and genetherapy.

Rose E. Tucker Charitable Trust

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OHSU Scientist Pushes Forward With Stem Cell Research

Study shows how common obesity gene contributes to weight gain

PUBLIC RELEASE DATE:

22-May-2014

Contact: Karin Eskenazi ket2116@cumc.columbia.edu 212-342-0508 Columbia University Medical Center

NEW YORK, NY (May 22, 2014) Researchers have discovered how a gene commonly linked to obesityFTOcontributes to weight gain. The study shows that variations in FTO indirectly affect the function of the primary cilium, a little-understood hair-like appendage on brain and other cells. Specific abnormalities of cilium molecules, in turn, increase body weight, in some instances, by affecting the function of receptors for leptin, a hormone that suppresses appetite. The findings, made in mice, suggest that it might be possible to modify obesity through interventions that alter the function of the cilium, according to scientists at Columbia University Medical Center (CUMC).

"If our findings are confirmed, they could explain how common genetic variants in the gene FTO affect human body weight and lead to obesity," said study leader Rudolph L. Leibel, MD, the Christopher J. Murphy Memorial Professor of Diabetes Research, professor of pediatrics and medicine, and co-director of the Naomi Berrie Diabetes Center at CUMC. "The better we can understand the molecular machinery of obesity, the better we will be able to manipulate these mechanisms and help people lose weight."

The study was published on May 6 in the online edition of Cell Metabolism.

Since 2007, researchers have known that common variants in the fat mass and obesity-associated protein gene, also known as FTO, are strongly associated with increased body weight in adults. But it was not understood how alterations in FTO might contribute to obesity. "Studies have shown that knocking out FTO in mice doesn't necessarily lead to obesity, and not all humans with FTO variants are obese," said Dr. Leibel. "Something else is going on at this location that we were missing."

In experiments with mice, the CUMC team observed that as FTO expression increased or decreased, so did the expression of a nearby gene, RPGRIP1L. RPGRIP1L is known to play a role in regulating the primary cilium. "Aberrations in the cilium have been implicated in rare forms of obesity," said Dr. Leibel. "But it wasn't clear how this structure might be involved in garden-variety obesity."

Dr. Leibel and his colleague, George Stratigopoulos, PhD, associate research scientist, hypothesized that common FTO variations in noncoding regions of the gene do not change its primary function, which is to produce an enzyme that modifies DNA and RNA. Instead, they suspected that FTO variations indirectly affect the expression of RPGRIP1L. "When Dr. Stratigopoulos analyzed the sequence of FTO's intronits noncoding, or nonprotein-producing, portionwe found that it serves as a binding site for a protein called CUX1," said Dr. Leibel. "CUX1 is a transcription factor that modifies the expression of RPGRIP1L."

Next, Dr. Stratigopoulos set out to determine whether RPGRIP1L plays a role in obesity. He created mice lacking one of their two RPGRIP1L genes, in effect, reducing but not eliminating the gene's function. (Mice that lack both copies of the gene have several serious defects that would obscure the effects on food intake.) Mice with one copy of RPGRIP1L had a higher food intake, gained significantly more weight, and had a higher percentage of body fat than controls.

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Study shows how common obesity gene contributes to weight gain

LW Genetic Engineering – Video


LW Genetic Engineering
This video is about Genetic engineering 2.

By: Liz Wigdahl

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LW Genetic Engineering - Video

Court decision keeps NZ GE-free

Court decision keeps NZ GE-free

A recent High Court decision is a victory for New Zealand consumers, farmers and foresters, says the Soil & Health Association. The High Court ruled against a decision by the Environmental Protection Authority (EPA) that two new plant breeding techniques were not genetic engineering techniques.

Soil & Health welcomes the court ruling. Without it New Zealand would have lost its status and international reputation as a producer of GE-free food and fibre.

If the EPAs decision had not been challenged and found wanting, plants bred using these GE techniques could have been planted in New Zealand without any public consultation or monitoring of their effects, said Marion Thomson, co-chair of Soil & Health.

There are other new techniques like this, and they must all be thoroughly and independently scrutinised and the precautionary principle applied. Otherwise, its an uncontrolled experiment that could have adverse effects for people, animals and the environment.

The High Court ruled that the EPA had misinterpreted the law and that it had failed to apply the precautionary principle.

Soil & Health is concerned about the ability of the EPA to protect our environment, says Thomson. We commend the Sustainability Council for challenging the EPAs decision, but we should not have to rely on not-for-profit watchdog organisations to protect our environment. That is the job of the EPA, and we urge them to apply the precautionary principle in any similar decisions in future.

Soil & Health has been informing its members and the public about genetic engineering for over 25 years. It has played a key role in advocating for a GE-free New Zealand for health, environmental, economic and other reasons. The New Zealand public, and our key markets, have consistently opposed genetic engineering in food and the environment.

ENDS

Scoop Media

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Court decision keeps NZ GE-free

The Sims 3 | Perfect Genetics Challenge Part 4: Preggers – Video


The Sims 3 | Perfect Genetics Challenge Part 4: Preggers
In this part, we get all dem woohooty woohoos going. Backstory: "Once upon a time, the Mighty Player sent a Sim to live in the world where all its creations were living happily. But this...

By: simplyapril

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The Sims 3 | Perfect Genetics Challenge Part 4: Preggers - Video

Why Atossa Genetics (ATOS) Stock Is Up Today

NEW YORK (TheStreet) --Atossa Genetics (ATOS)stock surged on Thursday after the Board of Directors approved a poison pill provision. The adoption of the stockholder rights agreement is designed to deter "coercive, unfair, or inadequate takeovers and other abusive tactics," the company said in a statement.

The developer of breast health solutions said the agreement would not prevent takeovers at a full and fair price, but would encourage any potential acquirers to first negotiate with the Board.

STOCKS TO BUY: TheStreet Quant Ratings has identified a handful of stocks that can potentially TRIPLE in the next 12 months. Learn more.

Under the terms of the agreement, the rights agent will distribute to stockholders a dividend distribution of one preferred stock purchase right for each share of common stock held at the close of business May 26. Rights will be exercisable if a person becomes an "acquiring person" by acquiring ownership of 15% or more of common stock or if a person commences a tender which would result in that person becoming an "acquiring person."

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Why Atossa Genetics (ATOS) Stock Is Up Today

Gene Therapy Cure Cancer – Video


Gene Therapy Cure Cancer
This is my final project for science subject at my school. I learning about biotechnology. My topic is about " How gene therapy can cure cancer?" and i have ...

By: della lathifah

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Gene Therapy Cure Cancer - Video

Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 – 2019

Albany, New York (PRWEB) May 22, 2014

Gene therapy involves use of DNA as a pharmaceutical agent to treat diseases. It is one of the most important developments in the field of medicine that has potential to treat various lethal diseases such as HIV, cancer and cystic fibrosis. In the long run, biotechnology and clinical trial industries will benefit from developments in gene therapy and provide potential treatment solutions for various incurable diseases.

Browse the full report - http://www.transparencymarketresearch.com/gene-therapy-market.html.

In the present scenario, various pharmaceutical companies are using clinical data to validate the concept of gene therapy. Moreover, many venture capital investors are also showing their interest in gene therapy, and are investing heavily in its development. However, gene therapy is highly dependent on the regulatory approvals and most of the products are currently in clinical trial phase. Most of these gene therapy products are for cancer and cardiovascular diseases, and are in Phase III/ Phase II of clinical trials.

In addition, growing popularity of DNA vaccines boost advances in gene therapy and is likely to be practiced in clinics in the near future, with a number of therapy programs now in phase II/III trials, showing promising results.

Get report sample - http://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=1838.

Some of the major players operating in the market are AnGes MG, BioSante Pharmaceuticals, GenVec, Genzyme Corporation, Oxford BioMedica, Transgene, Urigen Pharmaceuticals and Vical.

This research report analyzes this market depending on its market segments, major geographies, and current market trends. Geographies analyzed under this research report include:

This report provides comprehensive analysis of:

This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porters five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.

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Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 - 2019

Gene therapy extends survival in an animal model of spinal muscular atrophy

PUBLIC RELEASE DATE:

22-May-2014

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

New Rochelle, NY, May 22, 2014To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA. A new study demonstrating that enough copies of the SMN1 gene can be delivered to the spinal cord motor neurons to extend the survival of the treated animals is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

Marco Passini and coauthors from Genzyme (Framingham, MA), University of California San Francisco, Emory University School of Medicine (Atlanta, GA), and Georgetown University Medical Center (Washington, DC) used an adeno-associated viral vector as the delivery vehicle to transport copies of the SMN1 gene into motor neurons in the spinal cord via intrathecal delivery. They report on the effectiveness of restoring the levels of functional SMN protein in normal pig and non-human primate SMA models that would predict efficacy based on gene transfer with the same vector in an authentic mouse model of SMA in the article "Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9Survival Motor Neuron 1 for Spinal Muscular Atrophy."

"This is a very promising and thorough set of preclinical studies that supports rapid translation to the clinic," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

###

About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

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Gene therapy extends survival in an animal model of spinal muscular atrophy

8th Annual Chair-Leader Event – Part 2 – Video


8th Annual Chair-Leader Event - Part 2
On May 7th, 2014 Members of Parliament representing all political parties cam together to participate in the 8th annual Chair-Leaders Campaign to raise awareness regarding accessibility barriers....

By: Spinal Cord Injury Canada TV

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8th Annual Chair-Leader Event - Part 2 - Video

A brave new world: Stem cell therapy in Lebanon

BEIRUT: Fat removal and a non-surgical facelift at the same time might sound like a two-for-one offer too good to be true. But that is a pretty common combination at the Innovi Stem Cell Therapy Clinic, where doctors extract stem cells from the bodys fat to do any number of cosmetic cleanups, from scar removal to diminishing fine lines and wrinkles.

The clinic opened five months ago in the Beirut neighborhood of Sodeco, bringing Lebanon its first specialized center in stem cell research.

Around the world at any given medical conference, from fields as diverse as orthopedics to dentistry, stem cells have become one of the main events, as researchers believe these undifferentiated cells hold the cure to some of the gravest human diseases: cancer, diabetes, multiple sclerosis, to name a few.

In a country like Lebanon, stem cell specialists figured the best way to support their research was to offer one of the most in-demand medical procedures: cosmetic surgery.

Walking through the halls of the elegant, albeit quaint, clinic, one will see top-of-the-line fat freezing technology, equipment for laser hair removal and facilities where doctors carry out medical face peels and stretch mark treatment.

They also offer Ozone therapy, which uses pure oxygen that can supposedly alleviate a range of maladies from skin disorders and premature aging to chronic pain.

But we are not a beauty clinic, said one of the doctors, who asked not to be identified due to Lebanons strict medical advertising laws.

These cosmetic procedures complement their work in stem cells, a far less understood and rapidly evolving area of medicine. Innovi, for example, has built the Middle Easts only stem cell bank, where up to 19,000 vials can be frozen and preserved with liquid nitrogen. The closet housing the bank, which looks like an enormous washing machine, now holds the stem cells of a modest 10 clients.

The clinic has become a hub for various stem cells research. Doctors have visited from Europe and a Syrian doctor is now working with a couple to try and grow sperm from the stem cells of a man with aspermia.

But cosmetic treatments and stem cells go well together as doctors have been using fat-derived cells, also called adipose stem cells, as a Botox-like filler for almost a decade.

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A brave new world: Stem cell therapy in Lebanon

Alameda: Girl, 10, needs bone marrow donor

ALAMEDA -- Ten-year-old Myla Cunanan, a fourth-grader at Ruby Bridges Elementary School in Alameda, has been in Kaiser Oakland since March 24, undergoing her second round of chemotherapy for myeloid sarcoma, a form of acute myeloid leukemia.

Ruby Bridges Elementary hosted a "Myla Needs a Match" bone marrow donor registration drive Saturday that was attended by school parents, Myla's classmates, Kaiser Oakland staff, volunteers from the Asian-American Donor Program (AADP), congregants from the Church of Christ in Alameda, which Myla's family attends, the Alameda Police Department and many others. By day's end, 97 adults had registered to donate bone marrow.

"She's in good spirits; she's very brave," said Myla's mother, Leyna Cunanan, whose daughter was diagnosed with the disease in March. "She needs a bone-marrow transplant in order to survive."

Myla's cousin, 13-year-old Kaitlyn Francisco, who attends Lincoln Middle School, welcomed visitors as they arrived at the bone marrow drive.

"I want to work hard in order for my cousin to get better," Kaitlyn said. "Myla is very sweet, funny and considerate. We visit her every Sunday. Sometimes she's very cheerful, and sometimes she's weak and tired. When we visit her, she always tries to be active."

Myla's sister, Marielle, 13, and her brother, Matthew, 15, also welcomed visitors to the bone marrow drive, standing outside the school where dozens of purple balloons danced in the wind.

"She's really playful and talkative," said Marielle of her little sister. "I want to help find a match for my sister so she can heal."

Myla's aunt, Sarlea Atizado, described her niece as "a very sweet, caring and God-fearing child."

"She has a strong faith in God and does so many selfless acts," Atizado said. "Even in times of sickness, she worries about other people, especially her loved ones. One of her prayers was, 'God, please heal me so that my loved ones will not worry about me, especially my grandmother.' Myla has not only given us the opportunity to take care and pray for her, but has made our family even stronger despite this ordeal. God has a purpose for everything."

Myla is of Filipino descent, which makes it more difficult to find a bone marrow match, according to AADP volunteer Jaydeep Pathak, who explained bone marrow donation procedures to potential donors at the event.

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Alameda: Girl, 10, needs bone marrow donor

Functional nerve cells from skin cells

20 hours ago These are mature nerve cells generated from human cells using enhanced transcription factors. Credit: Fahad Ali

A new method of generating mature nerve cells from skin cells could greatly enhance understanding of neurodegenerative diseases, and could accelerate the development of new drugs and stem cell-based regenerative medicine.

The nerve cells generated by this new method show the same functional characteristics as the mature cells found in the body, making them much better models for the study of age-related diseases such as Parkinson's and Alzheimer's, and for the testing of new drugs.

Eventually, the technique could also be used to generate mature nerve cells for transplantation into patients with a range of neurodegenerative diseases.

By studying how nerves form in developing tadpoles, researchers from the University of Cambridge were able to identify ways to speed up the cellular processes by which human nerve cells mature. The findings are reported in the May 27th edition of the journal Development.

Stem cells are our master cells, which can develop into almost any cell type within the body. Within a stem cell, there are mechanisms that tell it when to divide, and when to stop dividing and transform into another cell type, a process known as cell differentiation. Several years ago, researchers determined that a group of proteins known as transcription factors, which are found in many tissues throughout the body, regulate both mechanisms.

More recently, it was found that by adding these proteins to skin cells, they can be reprogrammed to form other cell types, including nerve cells. These cells are known as induced neurons, or iN cells. However, this method generates a low number of cells, and those that are produced are not fully functional, which is a requirement in order to be useful models of disease: for example, cortical neurons for stroke, or motor neurons for motor neuron disease.

In addition, for age-related diseases such as Parkinson's and Alzheimer's, both of which affect millions worldwide, mature nerve cells which show the same characteristics as those found in the body are crucial in order to enhance understanding of the disease and ultimately determine the best way to treat it.

"When you reprogramme cells, you're essentially converting them from one form to another but often the cells you end up with look like they come from embryos rather than looking and acting like more mature adult cells," said Dr Anna Philpott of the Department of Oncology, who led the research. "In order to increase our understanding of diseases like Alzheimer's, we need to be able to work with cells that look and behave like those you would see in older individuals who have developed the disease, so producing more 'adult' cells after reprogramming is really important."

By manipulating the signals which transcription factors send to the cells, Dr Philpott and her collaborators were able to promote cell differentiation and maturation, even in the presence of conflicting signals that were directing the cell to continue dividing.

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Functional nerve cells from skin cells

Scientists find more genes tied to alcoholism risk

By Randy Dotinga HealthDay Reporter

(HealthDay News) -- New research pinpoints 11 gene variations that appear to be linked to a higher risk of alcoholism.

However, their impact seems to be small in individuals, and the findings may be too limited to be immediately useful in terms of prevention or treatment, an expert said.

Still, the results could provide more insight into the mysterious role that genes play in determining why some people drink and don't get addicted while others become alcoholics, the researchers said.

It may be possible someday for young people to take a blood test and learn if they're susceptible to alcoholism, said study co-author Dr. Alexander Niculescu III, an associate professor of psychiatry and medical neuroscience at Indiana University School of Medicine.

"Genes are not destiny, but knowing your genetic risk profile can empower you to make smart lifestyle choices," such as avoiding alcohol, he said.

According to the U.S. National Institute on Alcohol Abuse and Alcoholism, research suggests that genetics account for about half the risk that someone will become an alcoholic. Environment is responsible for the rest.

Scientists know about one genetic trait that makes some Asians ill when they drink, making it less likely that they'll abuse alcohol. But what about other genes that affect alcoholism?

In the new study, Niculescu and his colleagues linked 11 genetic variations to alcoholism after exploring genetic data from human and animal studies. It's not clear how the traits influence the risk of alcoholism, Niculescu said, but he thinks they may have something to do with the part of your brain that tells you that you feel good.

The researchers came up with a "genetic risk score" based on how many of the variations a person had. The investigators determined that the average person had a score of 47 out of 100, while 75 percent of those with scores above 48 were alcoholics.

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Scientists find more genes tied to alcoholism risk

Gene discovery may halt a deep-rooted pepper disease

May 21, 2014

Scientists have identified a promising candidate gene that encodes resistance to a root rot disease that severely diminishes chili pepper crop yields. (UC Davis photo)

For more than a century, the global hot pepper industry has been dealing with a problem. A funguslike pathogen, known as Phytophthora capsici, has spread a root rot disease that severely diminishes crop yields. Despite highly adaptive management practices and the availability of wild pepper varieties that have evolved resistance, the pathogen continues to thrive.

Now, scientists from the University of California, Davis, have identified a promising candidate gene that encodes resistance to P. capsici in peppers. The work is published this month in the journal The Plant Genome, available on line at http://bit.ly/1ocomlF.

Under the direction of plant scientist Allen Van Deynze, the director of research at UC Davis Seed Biotechnology Center, doctoral candidate Zeb Rehrig had begun the project by screening 31,000 genes in a population of pathogen-resistant chili peppers and jalapeos a number far surpassing the standard 1,000 genes screened in this type of test. This allowed the researchers to build a high-density genetic map of 3,600 genes.

They then tested their findings by introducing the peppers to P. capsici samples collected from across Mexico, New Mexico, New Jersey, California, Michigan and Tennessee. Analyses incorporating the pepper genome, from a study Van Deynze recently co-authored, ultimately led them to the P5 chromosome and to the gene related to resistance, CaDMR1.

While breeders have long known of a resistant pepper gene in the area of this chromosome, no one has been able to zero in on it the way the UC Davis team has, Van Deynze said.

The goal of when you get the gene is of course that you can have the perfect marker, he explained, which theoretically should be useful in really any population you test it in.

The new DNA markers will pave the way for breeders to selectively target the exact gene and turn on the resistance mechanism, giving an industry worth $29.1 billion worldwide a much-needed new tool in the battle over root rot.

This paper is the first chance to show all the work Ive been doing these last couple of years, said Rehrig. Its showing things you can apply directly to breeding right away.

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Gene discovery may halt a deep-rooted pepper disease

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