Archive for the ‘Gene Therapy Research’ Category

Regulated information December 23, 2013

TiGenix secures EUR 10 million in financing from Kreos Capital Company ends year in strong position to fully leverage leading cell therapy platform

Leuven (BELGIUM) - December 23, 2013 - TiGenix (Euronext Brussels: TIG) announced today that it has signed a structured debt financing agreement of up to EUR10 million with Kreos Capital (Kreos), Europe's largest and leading provider of growth debt to high-growth companies.

The funds will supplement the EUR 12 million in equity financing TiGenix recently secured from Gri-Cel SA, and will be used for general growth purposes as TiGenix advances the development of its expanding pipeline of cell therapy products.

"In combination with the recent strategic investment by Gri-Cel/Grifols this funding significantly strengthens our financial position and allows us to aggressively expand our pipeline of proprietary cell therapy products," said Eduardo Bravo, CEO of TiGenix. "Importantly, it enables us to independently finalize the Phase III trial with our lead product Cx601 and file for European registration, and thus capture significantly more value from a potential partnering agreement. The addition of debt financing represents an attractive and, except for a limited warrant component, non-dilutive complement to our existing capital structure. We are delighted to finish the year with a solid balance sheet and additional resources to optimally leverage our world-leading cell therapy technology platform as we move forward."

"Kreos is very pleased to be able to support TiGenix as it further builds its strong cell therapy portfolio," said Maurizio PetitBon, General Partner of Kreos. "TiGenix constitutes one of our first investments in the public market, and we have been very impressed by the quality of the team, the technology platform and the underlying business."

About the loan agreement Draw down: three tranches at the Company's discretion: EUR 5 million in early February 2014; EUR 2.5 million by end of May, 2014; EUR 2.5 million by end of September, 2014 Term: four years Amortization: starts at first anniversary Interest: 12.5% fixed annual interest rate Structure: security over certain assets; no financial covenants Warrants:approximately 2 million warrants to be granted to Kreos, subject to shareholder approval; exercise price to equal 30-day average closing price of TiGenix share at date of issue of warrants; if shareholders do not approve the issue of warrants, Kreos is entitled to a payment of EUR 890,000 over 3 years

For more information: Eduardo Bravo Chief Executive Officer eduardo.bravo@tigenix.com

Claudia D'Augusta Chief Financial Officer claudia.daugusta@tigenix.com

Hans Herklots Director Investor & Media Relations hans.herklots@tigenix.com +32 16 39 60 97

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TiGenix : TiGenix secures EUR 10 million in financing from.

Much of today's cancer research is devoted to finding missing or defective genes that cause cancer or increase an individual's risk for certain types of cancer. Gene research at MDAnderson has resulted in many important discoveries. We identified the mutated multiple advanced cancers gene (MMAC1) involved in some common cancers. We also performed the first successful correction of a defective tumor suppressor gene (p53) in human lung cancer. Current gene therapies are experimental, and many are still tested only on animals. There are some clinical trials involving a very small number of human subjects.

The potential benefits of gene therapy are two-fold:

The focus of most gene therapy research is the replacement of a missing or defective gene with a functional, healthy copy, which is delivered to target cells with a "vector." Viruses are commonly used as vectors because of their ability to penetrate a cells DNA. These vector viruses are inactivated so they cannot reproduce and cause disease. Gene transfer therapy can be done outside the body (ex vivo) by extracting bone marrow or blood from the patient and growing the cells in a laboratory. The corrected copy of the gene is introduced and allowed to penetrate the cells DNA before being injected back into the body. Gene transfers can also be done directly inside the patients body (in vivo).

Other therapies include:

Gene therapy is a complicated area of research, and many questions remain unanswered. Some cancers are caused by more than one gene, and some vectors, if used incorrectly, can actually cause cancer or other diseases. Replacing faulty genes with working copies also brings up ethical issues that must be addressed before these therapies can be accepted for preventing cancer. Talk to your cancer specialist about the implications of gene therapy.

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Types of Treatment - Gene Therapy | MD Anderson Cancer Center

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23-Dec-2013

Contact: Lisa Newbern lisa.newbern@emory.edu 404-727-7709 Emory Health Sciences

New findings suggest the oxytocin receptor, a gene known to influence mother-infant bonding and pair bonding in monogamous species, also plays a special role in the ability to remember faces. This research has important implications for disorders in which social information processing is disrupted, including autism spectrum disorder. In addition, the finding may lead to new strategies for improving social cognition in several psychiatric disorders.

A team of researchers from Yerkes National Primate Research Center at Emory University in Atlanta, the University College London in the United Kingdom and University of Tampere in Finland made the discovery, which will be published in an online Early Edition of Proceedings of the National Academy of Sciences.

According to author Larry Young, PhD, of Yerkes, the Department of Psychiatry in Emory's School of Medicine and Emory's Center for Translational Social Neuroscience (CTSN), this is the first study to demonstrate that variation in the oxytocin receptor gene influences face recognition skills. He and co-author David Skuse point out the implication that oxytocin plays an important role in promoting our ability to recognize one another, yet about one-third of the population possesses only the genetic variant that negatively impacts that ability. They say this finding may help explain why a few people remember almost everyone they have met while others have difficulty recognizing members of their own family.

Skuse is with the Institute of Child Health, University College London, and the Great Ormond Street Hospital for Children, NHS Foundation Trust, London.

Young, Skuse and their research team studied 198 families with a single autistic child because these families were known to show a wide range of variability in facial recognition skills; two-thirds of the families were from the United Kingdom, and the remainder from Finland.

The Emory researchers previously found the oxytocin receptor is essential for olfactory-based social recognition in rodents, like mice and voles, and wondered whether the same gene could also be involved in human face recognition. They examined the influence of subtle differences in oxytocin receptor gene structure on face memory competence in the parents, non-autistic siblings and autistic child, and discovered a single change in the DNA of the oxytocin receptor had a big impact on face memory skills in the families. According to Young, this finding implies that oxytocin likely plays an important role more generally in social information processing, which is disrupted in disorders such as autism.

Additionally, this study is remarkable for its evolutionary aspect. Rodents use odors for social recognition while humans use visual facial cues. This suggests an ancient conservation in genetic and neural architectures involved in social information processing that transcends the sensory modalities used from mouse to man.

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Researchers identify gene that influences the ability to remember faces

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Newswise BIRMINGHAM, Ala. Changes to a gene called LZTR1 predispose people to develop a rare disorder where multiple tumors called schwannomas form near nerve pathways, according to a study published today in the journal Nature Genetics and led by researchers from the University of Alabama at Birmingham.

The formation of multiple schwannomas is one sign that a person has the genetic disorder called schwannomatosis, which is one of the three major forms of neurofibromatosis, besides neurofibromatosis types 1 and 2. The condition is so named because the tumors originate in Schwann cells that form in sheaths that insulate nerves to cause severe, chronic pain in many patients.

To date, physicians cannot give most patients a confirmed diagnosis for schwannomatosis, even if they show symptoms, because changes in genes linked to the condition by past studies explain only about 50 percent of familial and less than 10 percent of sporadic cases.

Work in 2007 determined that inheritable mutations in SMARCB1 predisposed to schwannomatosis. In addition, the schwannomas showed a loss of the long arm of chromosome 22, and different mutations in the neurofibromatosis type 2 (NF2) gene were found in each tumor studied.

Despite these many known details, much of the risk for schwannomatosis remained unexplained going into the current study. Several research groups had proposed that other schwannomatosis-predisposing genes existed, but no one had found any. Specializing in genetic studies for all forms of the neurofibromatoses, the UAB Medical Genomics Laboratory chose to focus its research on a subset of schwannomatosis samples that did not harbor SMARCB1 mutations, which framed their experiments such that the role of LZTR1 was revealed.

We have been working urgently to identify the genetic mechanisms behind these diseases because doing so is central to efforts to understand schwannoma tumor development as well as to identify new drug treatments, said Ludwine Messiaen, Ph.D., director of the Medical Genomics Laboratory, professor in the Division of Clinical Genetics in the Department of Genetics within the UAB School of Medicine and corresponding study author. This is pertinent as only some of the schwannomas can be surgically removed without neurological consequences, and there is no widely accepted approach for treating the severe, chronic pain in these patients.

The study, conceived and coordinated by Arkadiusz Piotrowski of the University of Gdansk in Poland and Messiaen, resulted in the identification of LZTR1 on chromosome 22q as a novel tumor-suppressor gene predisposing to multiple schwannomas in patients without a mutation in SMARCB1. The results were seen in patients whose schwannomas also showed a loss of the long arm of chromosome 22 and a different somatic NF2 mutation in each tumor. The team found that in all 25 schwannomas studied from 16 unrelated schwannomatosis patients, all tumors showing a loss of the long arm of chromosome 22 and a different somatic NF2 mutation in each tumor also had LZTR1 mutations present, strongly supporting the contribution to the disease by the combination of these factors.

The LZTR1 mutations were found using massive parallel sequencing (e.g. next-generation sequencing) of highly evolutionary conserved sequences specifically on chromosome 22. LZTR1 mutations likely will be found in a high fraction of familial as well as sporadic schwannomatosis patients, whose predisposition is not caused by SMARCB1, says Messiaen. Indeed, LZTR1 mutations were found in 6/6 familial and 8/11 sporadic such patients. Both causal genes, LZTR1 and SMARCB1, show a potential functional link to chromatin remodeling mechanisms, which play a crucial role in cell differentiation and adaptation to environmental stimuli. Further, LZTR1 and SMARCB1 are known to interact with histone deacetylase 4 or HDAC4, which is a target for histone deacetylase inhibitors, a new class of anti-tumor drugs. The present findings will encourage further studies aiming at potential treatment for schwannomatosis.

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Changes in Gene Explain More of Inherited Risk for Rare Disease

Genetic engineering is a scientific development that involves the artificial manipulation of an organism's genes by using techniques such as molecular cloning and transformation in order to alter their nature and structure. Many of these transformations are achieved by manipulation of an organism's DNA, which effectively is the code inscribed in every cell to determine how it will function.

As with most scientific developments there are a number of arguments both for and against.

There has been a considerable amount of research into the genetic engineering of crops such as potatoes, tomatoes, soybean and rice, with the aim of obtaining new strains that have better nutritional qualities and better yields.

In a world where there is a continual need to produce more food; genetically engineered crops are being developed to grow on land that is currently not suitable for cultivation. By manipulating the genes in crops the aim is to improve their nutritional value, their rate of growth and their flavour.

Seeds can be engineered so that they are resistant to pests and can survive cultivation in relatively harsh climatic conditions. Biotechnology can also be used to slow down the process of food spoilage so that fruit and vegetables can have a longer shelf life.

Although on the face of it genetic engineering might appear to bring a number of very positive benefits, there is by no means a universal approval of this practice.

Greenpeace International is very firm in its opposition, pointing out that there is no adequate scientific understanding of the impact that genetically modified organisms might have on the world's environment and on human health.

Undesirable genetic mutations can lead to allergies in crops and critics believe that while genetic engineering might enhance taste and appearance of foodstuffs, it could also hamper the nutritional value. At the very least, in order to inform consumers, all foodstuffs or products that have been made from genetically modified food should be clearly labelled as such at point of sale.

Whole new substances such as proteins and other food nutrients can be produced as a result of genetic engineering. The genetic modification of foods can be used to increase their medicinal value, thus making available a range of homegrown medical vaccines.

Greenpeace maintains that commercial interests are the prime movers to introduce genetically modified organisms into the food chain and stresses that once these organisms have been released into the environment they cannot be recalled.

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Genetic Engineering | The Earth Times | Encyclopaedia

Learn the Fundamentals of Biotechnology

1. What is biotechnology?

Biotechnology is the application of biological knowledge to obtain new techniques, materials and compounds of pharmaceutical, medical, agrarian, industrial and scientific use, i.e., of practical use.

The pioneer fields of biotechnology were agriculture and the food industry but nowadays many other practical fields use its techniques.

2. What is genetic engineering?

Genetic engineering is the use of genetic knowledge to artificially manipulate genes: It is one of the fields of biotechnology.

3. At the present level of the biotechnology what are the main techniques of genetic engineering?

The main techniques of genetic engineering today are: the recombinant DNA technology (also called genetic engineering itself) in which pieces of genes from an organism are inserted into the genetic material of another organism producing recombinant beings; the nucleus transplantation technology, popularly known as cloning, in which a nucleus of a cell is grafted into a enucleated egg cell of the same species to create a genetic copy of the donor (of the nucleus) individual; the technology of DNA amplification, or PCR (polymerase chain reaction), that allows millions replications of chosen fragments of a DNA molecule.

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Genetic Engineering - Biology Questions and Answers

ANN ARBOR Many primary care pediatricians say they feel uncomfortable providing health care to patients with genetic disorders. Also, many do not consistently discuss all risks and benefits of genetic tests with patients, according to research published recently in the American Journal of Medical Genetics.

Investigators from the University of Michigans C.S. Mott Childrens Hospital and The Childrens Hospital at Montefiore (CHAM) conducted a national survey of 88 physicians who are part of the American Academy of Pediatrics Quality Improvement Innovation Networks, assessing their comfort level ordering genetic tests for their pediatric patients, their attitudes toward genetic medical care and their choices regarding taking family histories. The majority of those physicians reported ordering few genetic tests (three or less times) per year, excluding newborn screening which is federally mandated for all newborns; few (13 percent) strongly agreed that they discussed the potential risks, benefits, and limitations of genetic tests with all their patients and only half felt competent in providing healthcare to patients with genetic disorders.

While genetics has historically been viewed as a discipline focused on rare conditions, recent genomic advances have highlighted that genetics has a role in common conditions encountered in primary care medicine, said Dr. Beth Tarini, senior author, assistant professor of pediatrics, Child Health Evaluation & Research (CHEAR) Unit, Division of General Pediatrics, University of Michigan and co-medical director of the Genetics in Primary Care Institute (GPCI), a project of the American Academy of Pediatrics. Unfortunately, most PCPs have received insufficient education and training about genetics, which has left them uncertain about their role in providing genetics related care.

The study found that 100 percent of study participants stated that taking a family history is important, but less than one-third stated that they gather a minimum of a three-generation family history, a basic component of a genetic medical evaluation. Previous studies have shown that using family history and genetic information greatly improved outcomes for patients so researchers encourage patients to know their family history and share this with their providers in order to optimize their health care.

PCPs play an integral role in caring for children with genetic conditions and it is vital that they feel comfortable identifying issues and providing comprehensive care to suit their patients unique needs, said Dr. Michael L. Rinke, lead author and assistant medical director for quality, CHAM, and assistant professor of pediatrics at Albert Einstein College of Medicine of Yeshiva University. Thousands of children in the U.S. are diagnosed with genetic disorders annually and in order to optimize outcomes for these patients early identification and medical intervention is essential.

The researchers say that robust education, increased access to resources, improved electronic health records systems to document family histories and rigorous quality improvement efforts are key to enhancing integration of genetic medicine into routine primary preventative care.

Tarini says that the national Genetics in Primary Care Institute Quality Improvement Project hopes to identify effective strategies so that physicians who are at the forefront of diagnosing and managing patients with genetic disorders feel confident and competent in their abilities to provide care for these patients.

Investigators from the University of Michigans C.S. Mott Childrens Hospital and The Childrens Hospital at Montefiore (CHAM) conducted a national survey of 88 physicians who are part of the American Academy of Pediatrics Quality Improvement Innovation Networks, assessing their comfort level ordering genetic tests for their pediatric patients, their attitudes toward genetic medical care and their choices regarding taking family histories. The majority of those physicians reported ordering few genetic tests (three or less times) per year, excluding newborn screening which is federally mandated for all newborns; few (13 percent) strongly agreed that they discussed the potential risks, benefits, and limitations of genetic tests with all their patients and only half felt competent in providing healthcare to patients with genetic disorders.

While genetics has historically been viewed as a discipline focused on rare conditions, recent genomic advances have highlighted that genetics has a role in common conditions encountered in primary care medicine, said Dr. Beth Tarini, senior author, assistant professor of pediatrics, Child Health Evaluation & Research (CHEAR) Unit, Division of General Pediatrics, University of Michigan and co-medical director of the Genetics in Primary Care Institute (GPCI), a project of the American Academy of Pediatrics. Unfortunately, most PCPs have received insufficient education and training about genetics, which has left them uncertain about their role in providing genetics related care.

The study found that 100 percent of study participants stated that taking a family history is important, but less than one-third stated that they gather a minimum of a three-generation family history, a basic component of a genetic medical evaluation. Previous studies have shown that using family history and genetic information greatly improved outcomes for patients so researchers encourage patients to know their family history and share this with their providers in order to optimize their health care.

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ANN ARBOR: Study: Many pediatricians uncomfortable providing care to kids with genetic conditions

Mendel Genetics Project
Western Guilford High School 2013 Genetics class project video.

By: furmanbud

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Mendel Genetics Project - Video

Forbidden Secrets Of Genetics Israel Eden And The Great Pyramid Revealed By Dr. Scott McQuate
http://www.Pyranosis.com - Recently-discovered information from the Sumerian records by Dr. Scott McQuate exposes secrets that have been hidden for thousands of yea...

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Forbidden Secrets Of Genetics Israel Eden And The Great Pyramid Revealed By Dr. Scott McQuate - Video

Experimental Physiology meeting report: Genomes, genes and genetics
Steve Lolait reports on the meeting from July 2013 at The University of Bristol, UK. Read full reports at http://ep.physoc.org/content/99/1.toc#SymposiumReports.

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Experimental Physiology meeting report: Genomes, genes and genetics - Video

BIO. "Biotechnology in Perspective." Washington, D.C.: Biotechnology Industry Organization, 1990. Altered Genes Each of us carries about half a dozen defective genes. We remain blissfully unaware of this fact unless we, or one of our close relatives, are amongst the many millions who suffer from a genetic disease. About one in ten people has, or will develop at some later stage, an inherited genetic disorder, and approximately 2,800 specific conditions are known to be caused by defects (mutations) in just one of the patient's genes. Some single gene disorders are quite common - cystic fibrosis is found in one out of every 2,500 babies born in the Western World - and in total, diseases that can be traced to single gene defects account for about 5% of all admissions to children's hospitals.

In the U.S. and Europe, there are exciting new programs to 'map' the entire human genome - all of our genes. This work will enable scientists and doctors to understand the genes that control all diseases to which the human race is prone, and hopefully develop new therapies to treat and predict diseases.

On the other hand, if the gene is dominant, it alone can produce the disease, even if its counterpart is normal. Clearly only the children of a parent with the disease can be affected, and then on average only half the children will be affected. Huntington's chorea, a severe disease of the nervous system, which becomes apparent only in adulthood, is an example of a dominant genetic disease.

Finally, there are the X chromosome-linked genetic diseases. As males have only one copy of the genes from this chromosome, there are no others available to fulfill the defective gene's function. Examples of such diseases are Duchenne muscular dystrophy and, perhaps most well known of all, hemophilia.

Queen Victoria was a carrier of the defective gene responsible for hemophilia, and through her it was transmitted to the royal families of Russia, Spain, and Prussia. Minor cuts and bruises, which would do little harm to most people, can prove fatal to hemophiliacs, who lack the proteins (Factors VIII and IX) involved in the clotting of blood, which are coded for by the defective genes. Sadly, before these proteins were made available through genetic engineering, hemophiliacs were treated with proteins isolated from human blood. Some of this blood was contaminated with the AIDS virus, and has resulted in tragic consequences for many hemophiliacs. Use of genetically engineered proteins in therapeutic applications, rather than blood products, will avoid these problems in the future.

Not all defective genes necessarily produce detrimental effects, since the environment in which the gene operates is also of importance. A classic example of a genetic disease having a beneficial effect on survival is illustrated by the relationship between sickle-cell anemia and malaria. Only individuals having two copies of the sickle-cell gene, which produces a defective blood protein, suffer from the disease. Those with one sickle-cell gene and one normal gene are unaffected and, more importantly, are able to resist infection by malarial parasites. The clear advantage, in this case, of having one defective gene explains why this gene is common in populations in those areas of the world where malaria is endemic.

The most likely candidates for future gene therapy trials will be rare diseases such as Lesch-Nyhan syndrome, a distressing disease in which the patients are unable to manufacture a particular enzyme. This leads to a bizarre impulse for self-mutilation, including very severe biting of the lips and fingers. The normal version of the defective gene in this disease has now been cloned.

If gene therapy does become practicable, the biggest impact would be on the treatment of diseases where the normal gene needs to be introduced into only one organ. One such disease is phenylketonuria (PKU). PKU affects about one in 12,000 white children, and if not treated early can result in severe mental retardation. The disease is caused by a defect in a gene producing a liver enzyme. If detected early enough, the child can be placed on a special diet for their first few years, but this is very unpleasant and can lead to many problems within the family.

The types of gene therapy described thus far all have one factor in common: that is, that the tissues being treated are somatic (somatic cells include all the cells of the body, excluding sperm cells and egg cells). In contrast to this is the replacement of defective genes in the germline cells (which contribute to the genetic heritage of the offspring). Gene therapy in germline cells has the potential to affect not only the individual being treated, but also his or her children as well. Germline therapy would change the genetic pool of the entire human species, and future generations would have to live with that change. In addition to these ethical problems, a number of technical difficulties would make it unlikely that germline therapy would be tried on humans in the near future.

Before treatment for a genetic disease can begin, an accurate diagnosis of the genetic defect needs to be made. It is here that biotechnology is also likely to have a great impact in the near future. Genetic engineering research has produced a powerful tool for pinpointing specific diseases rapidly and accurately. Short pieces of DNA called DNA probes can be designed to stick very specifically to certain other pieces of DNA. The technique relies upon the fact that complementary pieces of DNA stick together. DNA probes are more specific and have the potential to be more sensitive than conventional diagnostic methods, and it should be possible in the near future to distinguish between defective genes and their normal counterparts, an important development.

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Gene Therapy - An Overview - Access Excellence

Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a persons genes. Gene therapy is in its infancy, and current therapies are primarily experimental, with most human clinical trials still in the research stages.

How does gene therapy work? Genes are composed of DNA that carries information needed to make proteins the building blocks of our bodies. Variations in the DNA sequence or code of a gene are called mutations, which often are harmless but sometimes can lead to serious disease. Gene therapy treats disease by repairing dysfunctional genes or by providing copies of missing genes.

To reverse disease caused by genetic damage, researchers isolate normal DNA and package it into a vehicle known as a vector, which acts as a molecular delivery truck. Vectors composed of viral DNA sequences have been used successfully in human gene therapy trials. Doctors infect a target cell usually from a tissue affected by the illness, such as liver or lung cellswith the vector. The vector unloads its DNA cargo, which then begins producing the proper proteins and restores the cell to normal. Problems can arise if the DNA is inserted into the wrong place in the genome. For example, in rare instances the DNA may be inserted into a regulatory gene, improperly turning it on or off, leading to cancer.

Researchers continue to optimize viral vectors as well as develop non-viral vectors that may have fewer unexpected side effects. Nonviral gene delivery involves complexing DNA with an agent that allows it to enter a cell nonspecifically. DNA delivered in this manner is usually expressed for only a limited time because it rarely integrates into the host cell genome.

Initial efforts in gene therapy focused on delivering a normal copy of a missing or defective gene, but current programs are applying gene delivery technology across a broader spectrum of conditions. Researchers are now utilizing gene therapy to :

What diseases could be treated with gene therapy? About 4,000 diseases have been traced to gene disorders. Current and possible candidates for gene therapy include cancer, AIDS, cystic fibrosis, Parkinsons and Alzheimers diseases, amyotrophic lateral sclerosis (Lou Gehrig's disease), cardiovascular disease and arthritis.

In cases such as cystic fibrosis or hemophilia, disease results from a mutation in a single gene. In other scenarios like hypertension or high cholesterol, certain genetic variations may interact with environmental stimuli to cause disease.

Has gene therapy been successfully used in humans? Gene therapy is likely to be most successful with diseases caused by single gene defects. The first successful gene therapy on humans was performed in 1990 by researchers at the National Institutes of Health. The therapy treated a four-year-old child for adenosine deaminase (ADA) deficiency, a rare genetic disease in which children are born with severe immunodeficiency and are prone to repeated serious infections.

Since 1990, gene therapy had been tested in human clinical trials for treating such diseases as severe combined immunodeficiency disease (SCID), cystic fibrosis, Canavan's disease, and Gaucher's disease. In 2003, more than 600 gene therapy clinical trials were under way in the United States but only a handful of these are in advanced stages. SCID, in which children lack natural defenses against infection and can only survive in isolated environments, remains the only disease cured by gene therapy.

Are genetic alterations from gene therapy passed on to children? Gene therapy can be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene therapy, the patients genome is changed, but the change is not passed along to the next generation. In germline gene therapy, the patients egg or sperm cells are changed with the goal of passing on changes to their offspring. Existing gene therapy treatments and experiments are all somatic.

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Gene Therapy - American Medical Association

What is gene therapy?

Gene therapy is the use of genetic instructions to produce a protein to treat a disorder or deficiency. It can aid in a disease even if the therapy is not directly targeting a gene defect that causes the disease. In amyotrophic lateral sclerosis (ALS), gene therapy may help if it can deliver a beneficial protein, to salvage dying nerve cells. The gene therapy simply is a means to boost on site production of a trophic (growth enhancing) factor, at places where nerve cells are in trouble.

Genes are the molecules in all cells of our bodies that carry the instructions to make all of the materials that comprise the body. In the 1950s, scientists determined that genes code precisely for proteins, with a sequence that specifies the order of the building blocks of proteins, the amino acids. Each gene corresponds to a protein. Each base in a gene codes for an amino acid. The order of bases in a gene produces the ordered chain of amino acids that produce a working protein.

At the turn of the current century, scientists determined, in rough draft form, the sequence of all of the human genes. By this time, they also knew how to create a gene construct, and move that construct into cells, to get the cells to make the corresponding protein.

In some diseases, researchers already know that a defective gene is not able to work. They have the potential means to cure the disease, by replacing the defective gene with a correct, working copy. In ALS, only a few percent of patients have a known gene defect. For the rest, it may be one undiscovered gene that is the problem, or it may be several. But gene therapy can still be designed to aid patients with ALS by providing supportive proteins for nerve cells.

Vectors deliver genes Genes normally reside in the nucleus, the core of a cell, separated from the surrounding materials by a membrane. The chromosomes are the structures within the cell nucleus that contain the DNA that comprises the genes. It is very challenging to get a gene made in the lab to cross both the outer envelope of a cell, and the nuclear membrane as well, to reach the chromosomes.

Scientists studying viruses have discovered natures own solution to the problem of moving genes. Viruses are essentially genes that have evolved to hijack cells, instead of forming cells for themselves. So viruses have strategies to enter cells and take over the protein production process, to produce instead, the virus. Researchers have figured out how to use viruses as Trojan horses, to bring in genes that can then carry out genetic repairs, replacing defective DNA.

For many viruses, researchers can disarm the genes responsible for the damaging properties and put in, instead, genetic instructions to make therapeutic proteins. These viruses, redesigned by researchers, are called vectors. They are simply a means to smuggle in therapeutic genes.

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Gene Therapy - The ALS Association

In 1872, the American physician George Huntington wrote about an illness that he called "an heirloom from generations away back in the dim past." He was not the first to describe the disorder, which has been traced back to the Middle Ages at least. One of its earliest names was chorea,* which, as in "choreography," is the Greek word for dance. The term chorea describes how people affected with the disorder writhe, twist, and turn in a constant, uncontrollable dance-like motion. Later, other descriptive names evolved. "Hereditary chorea" emphasizes how the disease is passed from parent to child. "Chronic progressive chorea" stresses how symptoms of the disease worsen over time. Today, physicians commonly use the simple term Huntington's disease (HD) to describe this highly complex disorder that causes untold suffering for thousands of families.

More than 15,000 Americans have HD. At least 150,000 others have a 50 percent risk of developing the disease and thousands more of their relatives live with the possibility that they, too, might develop HD.

Until recently, scientists understood very little about HD and could only watch as the disease continued to pass from generation to generation. Families saw the disease destroy their loved ones' ability to feel, think, and move. In the last several years, scientists working with support from the National Institute of Neurological Disorders and Stroke (NINDS) have made several breakthroughs in the area of HD research. With these advances, our understanding of the disease continues to improve.

This brochure presents information about HD, and about current research progress, to health professionals, scientists, caregivers, and, most important, to those already too familiar with the disorder: the many families who are affected by HD.

HD results from genetically programmed degeneration of nerve cells, called neurons,* in certain areas of the brain. This degeneration causes uncontrolled movements, loss of intellectual faculties, and emotional disturbance. Specifically affected are cells of the basal ganglia, structures deep within the brain that have many important functions, including coordinating movement. Within the basal ganglia, HD especially targets neurons of the striatum, particularly those in the caudate nuclei and the pallidum. Also affected is the brain's outer surface, or cortex, which controls thought, perception, and memory.

HD is found in every country of the world. It is a familial disease, passed from parent to child through a mutation or misspelling in the normal gene.

A single abnormal gene, the basic biological unit of heredity, produces HD. Genes are composed of deoxyribonucleic acid (DNA), a molecule shaped like a spiral ladder. Each rung of this ladder is composed of two paired chemicals called bases. There are four types of basesadenine, thymine, cytosine, and guanineeach abbreviated by the first letter of its name: A, T, C, and G. Certain bases always "pair" together, and different combinations of base pairs join to form coded messages. A gene is a long string of this DNA in various combinations of A, T, C, and G. These unique combinations determine the gene's function, much like letters join together to form words. Each person has about 30,000 genesa billion base pairs of DNA or bits of information repeated in the nuclei of human cellswhich determine individual characteristics or traits.

Genes are arranged in precise locations along 23 rod-like pairs of chromosomes. One chromosome from each pair comes from an individual's mother, the other from the father. Each half of a chromosome pair is similar to the other, except for one pair, which determines the sex of the individual. This pair has two X chromosomes in females and one X and one Y chromosome in males. The gene that produces HD lies on chromosome 4, one of the 22 non-sex-linked, or "autosomal," pairs of chromosomes, placing men and women at equal risk of acquiring the disease.

The impact of a gene depends partly on whether it is dominant or recessive. If a gene is dominant, then only one of the paired chromosomes is required to produce its called-for effect. If the gene is recessive, both parents must provide chromosomal copies for the trait to be present. HD is called an autosomal dominant disorder because only one copy of the defective gene, inherited from one parent, is necessary to produce the disease.

The genetic defect responsible for HD is a small sequence of DNA on chromosome 4 in which several base pairs are repeated many, many times. The normal gene has three DNA bases, composed of the sequence CAG. In people with HD, the sequence abnormally repeats itself dozens of times. Over timeand with each successive generationthe number of CAG repeats may expand further.

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Huntington's Disease: Hope Through Research: National ...

IRVINE, CA (PRWEB) December 20, 2013

Proove Biosciences, the leader in providing personalized genetic pain medicine services, participated and presented clinical data and research at the Eastern Pain Associations Annual Meeting on December 7, 2013. The meeting covered current topics in pain medicine and opioid treatment, and took place at the New York Marriott Downtown, in Manhattan.

The EPA offers local and regional scientific meetings to foster an exchange of clinical and scientific information among multidisciplinary health professionals and researchers interested in the field of pain. The Annual EPA Scientific Meeting offered symposia and lectures given by nationally recognized speakers, posters and exhibits designed to appeal to a wide spectrum of specialists and interests.

The EPAs Annual Meeting concluded a successful and busy year for Proove presenting research throughout the country. Most recently, Proove presented data and findings at the ASRAs Pain Medicine Meeting, The National Workers Compensation Conference and Expo, and the Common Sense Pain Management Conference.

Our research and clinical team, along with our aggressive business development efforts have allowed Proove to experience outstanding growth throughout the year, stated Proove Biosciences President and Founder,Brian Meshkin. We are the only company providing proprietary testing services in personalized pain medicine. We are happy to have been a part of the EPAs regional meeting, and exhibiting our work with physicians, psychologists, nurses, and scientists dedicated to pain research.

About Proove Biosciences Proove Biosciences is the leading Personalized Pain Medicine laboratory that provides proprietary genetic testing services to help physicians improve outcomes for patients and contain costs for insurers. With offices in Southern California and the Baltimore-Washington metropolitan area, the Company is the research leader investigating and publishing data on the genetics of pain medicine with clinical research sites across the United States. Physicians use Proove Biosciences testing to improve pain medicine selection, dosing, and evaluation of medications they prescribe. From a simple cheek swab collected in the office, Proove performs proprietary genetic tests in its CLIA-certified laboratory to identify patients at risk for misuse of prescription pain medications and evaluate their metabolism of medications. For more information, please visit http://www.proovebio.com or call toll free 855-PROOVE-BIO (855-776-6832).

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Proove Biosciences Successfully Presents Data and Exhibits at the Eastern Pain Association’s Annual Meeting in New ...

IV International Symposium of Genetics and Breeding - 1/7
Session: Pioneer/GenMelhor partnership PhD Tabare Abadie e Leonardo de Azevedo Peixoto.

By: GenMelhor UFV

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IV International Symposium of Genetics and Breeding - 1/7 - Video

Innovation Campaign - Genetics Research

By: GovNL

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Innovation Campaign - Genetics Research - Video

PhD Student Showcase: Chung Ching Chu, Division of Genetics and Molecular Medicine

By: kingsmedicine

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PhD Student Showcase: Chung Ching Chu, Division of Genetics and Molecular Medicine - Video

Dr. Janet Rowley, a pioneer in cancer genetics research, has died at age 88.

Rowley spent most of her career at the University of Chicago, where she also obtained her medical degree. She died Tuesday of ovarian cancer complications at her home nearby, the university said in a statement.

Rowley conducted landmark research with leukemia in the 1970s, linking cancer with genetic abnormalities work that led to targeted drug treatment for leukemia. She identified a genetic process called translocation, now widely accepted. By 1990, more than 70 translocations had been identified in various cancers, according to her biography on the National Library of Medicine's website.

She is a recipient of the National Medal of Science, the nation's highest scientific honor and the Presidential Medal of Freedom, the nation's highest civilian honor.

"Janet Rowley's work established that cancer is a genetic disease," Mary-Claire King, president of the American Society of Human Genetics, said recently. "We are still working from her paradigm."

Rowley, known among colleagues for her intelligence and humility, called receiving the presidential award, in 2009, "quite remarkable."

"I've never regretted being in science and being in research," Rowley said at the time. "The exhilaration that one gets in making new discoveries is beyond description."

With her silvery hair and twinkling eyes, Rowley was a recognizable figure at the University of Chicago, often seen riding her bike around the South Side campus, even up until a few months ago despite her disease. She remained active in research until close to her death and hoped that her own cancer could contribute to understanding of the disease.

Just last month, she was well enough to attend a celebration of the 50th anniversary of the presidential medal in Washington alongside other previous recipients and this year's winners, who include several scientists, former President Bill Clinton, Oprah Winfrey, baseball's Ernie Banks and Loretta Lynn.

Rowley was born in New York City in 1925 and at age 15 won a scholarship to an advanced academic program at the University of Chicago. She went to medical school there when the quota was just three women in a class of 65, the university said.

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Janet Rowley, Cancer Genetics Pioneer, Dies at 88 - ABC News

Gene therapy for inherited disorders
Gene therapy will be of great significance for patients with hereditary disorders and society at large, says Gerard Wagemaker.

By: youris.com - European Research Media Center

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Gene therapy for inherited disorders - Video

Mayo Clinic to Grow Human Cells in Space to Test Treatment for Stroke
Abba Zubair, M.D., Ph.D, medical and scientific director of the Cell Therapy Laboratory at Mayo Clinic in Florida, talks about the $300000 his research team...

By: Mayo Clinic

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Mayo Clinic to Grow Human Cells in Space to Test Treatment for Stroke - Video

(PRWEB) December 21, 2013

Stem cell therapy has a tremendous potential to cure various illnesses and injuries. Recent news items have highlighted possibilities that it could treat damaged spinal cords or revitalize hip joints. Scientists are working on stem cell remedies for dementia, heart disease and diabetes. Doctors in some countries have begun using this therapy to grow replacement body tissue and treat leukemia.

However, stem cell treatments remain controversial. Some people object to them on ethical or religious grounds. Others express concern about the safety of these newfound cures. Animal testing has revealed that minor mistakes can result in impurities that cause cells to produce tumors and other ill effects. Some patients have died after receiving experimental therapies that weren't adequately tested.

The producers of the "Leading Edge" TV series plan to release a new segment that examines this fascinating yet contentious health topic. Presenter Jimmy Johnson will offer an update on important facts and recent developments in the world of stem cell research. Viewers can benefit from the program's concise and unbiased perspective on an issue that many people have yet to learn about.

"Leading Edge" is independently distributed to local public TV broadcasters across the U.S. The national Public Broadcasting Service does not act as its distributor. To learn more about this informational series, please browse http://www.leadingedgeseries.com or send an email message to the program's producers. They can be reached at info(at)leadingedgeseries(dot)com.

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"Leading Edge" Set to Produce New Content Featuring Stem Cell Therapy, with Host Jimmy Johnson

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Newswise DALLAS Dec. 19, 2013 UTSouthwestern neuroscience researchers have identified a gene that controls the response to cocaine by comparing closely related strains of mice often used to study addiction and behavior patterns.

The researchers suspect that the newly identified gene, Cyfip2, determines how mammals respond to cocaine, although it is too soon to tell what the indications are for humans or for addiction, said Dr. Joseph Takahashi, chair of neuroscience and a Howard Hughes Medical Institute investigator at UTSouthwestern and the senior author of the study.

The findings, reported in Science, evolved from examining the genetic differences between two substrains of the standard C57BL/6 mouse strain: a J strain from the Jackson Laboratory (C57BL/6J) and an N strain from the National Institutes of Health (C57BL/6N). Researchers compared the two strains of mice and used their differential responses to cocaine to identify the causative gene.

We found that the N strain has accumulated mutations over time, one of which has a very strong effect on cocaine response, Dr. Takahashi said. We propose that CYFIP2 the protein produced by the Cyfip2 gene is a key regulator of cocaine response in mammals.

The Takahashi laboratory has identified about 100 genetic differences that affect protein sequences between the two mouse strains, meaning that there are many genetic differences whose effects are not yet known, he added.

We identified this gene by first using a forward genetics strategy to search for differences in traits between the two mouse strains. We found a difference in cocaine response between them, with the C57BL/6N strain showing a reduced behavioral response, Dr. Takahashi said. We then carried out genetic mapping and whole genome sequencing, which allowed us to pinpoint the Cyfip2 gene as the causative one in a rapid and unambiguous way.

The C57BL/6J J mouse is the gold-standard strain for most research involving the mouse. For example, the reference sequence for the mouse genome, as well as most behavioral and physiological experiments, are based on the J strain. However, the International Knockout Mouse Consortium will be shifting emphasis to the N strain since they have created 17,000 embryonic stem cell lines with gene mutations that originate from the N strain. Thus, identifying genetic differences between these two mouse strains is important, Dr. Takahashi said.

Although mouse geneticists pay close attention to the specific strains of mice that they use, it has not been generally appreciated that sublines of the same strain of mouse might differ so profoundly. Thus, a C57BL/6 mouse might appear to be the same, but in fact there are many, many sublines of this laboratory mouse, and it is important to know which exact one you are using. Since the knockout mouse project has produced so many mutations (17,000) derived from the N strain, it will be even more important to keep in mind that not all C57BL/6 mice are the same.

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UT Southwestern Neuroscience Researchers Identify Gene Involved in Response to Cocaine

Are GMOs Safe Medical Course
For Educational Use Only - Fair Use - E.R. physician Dr. Travis Stork explains how GMOs Genetically Modified Organisms amplify crop production and sustainabi...

By: Abiasaph Abiathar

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Are GMOs Safe Medical Course - Video

An American activist opposing genetic engineering has praised Nelson as the first part of New Zealand to declare itself free of genetically modified organisms.

Self-published author and speaker Jeffrey Smith gave a talk at the Free House pub this week emphasising the value in keeping genetically engineered products out of New Zealand. It was one of only two talks he gave nationwide.

"New Zealand is very well-poised to take advantage of the economics of going non-GM."

He said there was a growing sentiment in his homeland that genetically modified products should be avoided. He expected a consumer-driven "tipping point" to occur within the next 18 months, saying this would see products containing GM ingredients becoming a "commercial liability".

"At that point, the clean, green image of New Zealand will translate better into economic premiums."

Mr Smith said there was a particularly receptive market available for meat and dairy products which originated from animals that had not eaten GM feed. New Zealand farmers should phase out the use of GM feed and market their meat and dairy in the US, claiming the GE free products would command a premium.

In New Zealand, processed foods can contain GM ingredients but must be labelled accordingly. No GM crops are grown commercially and no GM fruit, vegetables or meat are sold, but meat and other products from animals that have been fed GM food are not required to be labelled.

Mr Smith claimed GE foods had been found to cause health problems, but said studies into this area had been suppressed.

He was not all praise for New Zealand, criticising the local processes in place for the approval of GE products. He said the process was "nowhere near" rigorous enough and did not protect the public, saying it was widely cited internationally as an example of "how regulations should not be conducted".

Based in Iowa, Mr Smith was hosted in New Zealand by non-profit organisation GE Free New Zealand. President Claire Bleakley said it would be enlightening for a local audience to gain insights on the international experience with genetic modification.

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Activist lauds GE-free city