Lot 45
2014 Bull Sale.

By: Vertical Edge Genetics

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Lot 45 - Video

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Minecraft - Fun with Advanced Genetics
Me and Nyan have some fun with Advanced Genetics by ObsiLP Get Advanced Genetics Here: http://www.minecraftforum.net/topic/1988826-172164-forge-advanced-gene...

By: madogdan

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Carbs and Genetics
Why some people seem to be able to eat as much as they want and never gain weight.

By: Melanie Ash Fitness

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Carbs and Genetics - Video

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Lot 34
2014 Bull Sale.

By: Vertical Edge Genetics

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Lot 34 - Video

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All living things inherit their physical and biological characteristics from their parents through heredity. The study of heredity is a branch of science called genetics. Within living cells, tiny biochemical structure called DNA carries genetic information. DNA consists of segments, called genes, that carry instructions for the construction and functioning of every aspect of an organism. During cell divisions, random events called mutations can change the sequence of genes and lead to new characteristics and traits. This in turn drives evolution of life, leading to organisms that are better adapted to their environment.

Grade: 6 - 12

Learn about the basics of cells, chromosomes, and the genes contained in our DNA.

4:25

Grade: 6 - 12

Learn about the variations in our DNA called SNPs, and how they can help us understand relationships between people.

1:50

Grade: 6 - 12

Find out how chromosomes and genes are passed down from parent to child.

4:15

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Genetics - Kids Science Videos, Games and Lessons that ...

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Shares of Myriad Genetics Inc. ( MYGN ) slashed 8.34% at yesterday's close, soon after an unfavorable court bid, according to a Bloomberg report. The news narrated that the U.S. District Court for the District of Utah has denied Myriad's motion to block competitor Ambry Genetics from selling the competing tests, given that the patent infringement case between Myriad and Ambry Genetics is still pending at the court.

This court action also indicates Myriad's low chance of winning the lawsuits which alleged that the similar gene cancer tests related to the BRCA1 and BRCA2 genes being sold by Ambry Genetics Corp., violate its patents.

This comes as a major setback for Myriad Genetics which had recorded 43% growth in BracAnalysis - its flagship product in the last reported quarter. This is not the only standalone legal challenge facing Myriad. The company is currently entangled in several lawsuits with companies like Invitae Corporation and Laboratory Corporation of America Holdings ( LH ).

Notably, in Jun 2013, Supreme Court ruling declared that isolated human genes are not patentable. Following the decision, several companies have commenced offering clinical diagnostic and genomic laboratory services, including the testing and analysis of the BRCA1 and BRCA2 genes that compete with the BRACAnalysis testing and services of Myriad. Although Myriad has filed several patent infringement lawsuits against these companies, any defeat in these legal issues may create significant trouble for the company going forward.

We believe that emerging competitors and pricing pressure might plague the stock in the future. To add to the challenges faced by Myriad, macroeconomic uncertainty is another headwind. Also, with an extensive pipeline of some tests still under early-stage development, the company continues to face higher expenses. Although the company expects to increase its addressable market with myRisk in the near future, we remain cautious owing to the market conversion phase with the discontinuation of the successful legacy products.

Currently, the stock carries a Zacks Rank #2 (Buy). Some other well-placed stocks that are worth a look include Biogen Idec Inc. ( BIIB ) and Actelion Ltd. ( ALIOF ). Both the stocks carry a Zacks Rank #2.

ACTELION LTD (ALIOF): Get Free Report

BIOGEN IDEC INC (BIIB): Free Stock Analysis Report

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MYRIAD GENETICS (MYGN): Free Stock Analysis Report

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Myriad Genetics Down on Court Ruling - Analyst Blog

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saba crawl

By: ProjectWalkAtlanta Spinal Cord Injury Recovery

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saba crawl - Video

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Alex Valadka, MD, FAANS on running for TBI and spinal cord injury research

By: NREF

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Alex Valadka, MD, FAANS on running for TBI and spinal cord injury research - Video

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Prof. Alexander Smikodub

MD Ph.D

Alexander Smikodub jr.

MD Ph.D

Our clinic offers the advanced and patented methods of fetal stem cell treatment for various conditions and diseases. This method of treatment can be found in wikipedia: Stem cell therapy. Fetal stem cells are non-specialized cells that differentiate (turn) into any other cell type of the body that form organs and tissues. Fetal stem cells that we use for treatment have huge potential for growth, differentiation and are not rejected by the patients body, which allows to achieve unique long-term clinical effects.

We have more than 15 years of experience in stem cell therapy and are the leaders of the industry. Most of the methodic used in the clinic are unique and patent protected in many countries including USA. Since 1994 prof. Alexander Smikodub Sr. was the main researcher, doctor and administrator of the clinic. Now his son, Alexander Smikodub Jr. M.D. continues his fathers venture. During these years more than 6500 patients from all over the world received fetal stem cell treatment, resulting in significant improvement of their conditions, and in case of timely contact with us in complete cure of the diseases still considered lethal by most medical institutions.

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Stem cells are the new word in the medical science, possibly the new revolution. Their importance can be compared with antibiotics discovery or the first successful heart transplantation. They are the inner restorative and regenerative reserve of your body, found in blood, fat layer and bone marrow. After injection of a big stem cells doze, impaired tissues are recovered, regeneration speed is increased and overall condition is greatly improved. We use only material from healthy patients, which passes multiple security checks. They are a perfect material for treating a wide variety of neural and physical diseases.

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Stem cell therapy | Stem cell treatment | Unique Cell ...

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Health

Teruhiko Wakayama, a respected stem cell scientist from Japans RIKEN Institute, said he is not certain about the methods used in two studies he co-authored with lead investigator Haruko Obokata.

In the ground-breaking work, heralded by some in the field as a game-changer in the way stem cells are made, Obokata and her team, which included researchers from Harvard University and other international institutes, detailed how they were able to coax already developed cells to revert back to an embryonic-like state to become stem cells by simply exposing them to chemical solutions (mostly acidic) or physical stress. Stem cells can be manipulated to develop into any of the bodys tissues to repair or replace diseased cells.

The controversy erupted when Obokata and her team published a tips sheet for other researchers to follow to replicate their work. But inconsistencies between the newly released methods and the original protocol in the papers, as well as questions about images in the published work, led some to wonder about the validity of the results. Wakayama himself said he was able to repeat the study only once, with Obokatas assistance, but not on his own.

MORE: The Rise and Fall of the Cloning King

In a press conference in Japan last month, Wakayama, who is best known for using stem cell techniques to clone mice, said he asked all of the scientists involved to retract the papers, which were published in the journal Nature in January, and to have the data and results reviewed by other scientists. RIKEN is investigating the work, as is Nature.

The development adds another black eye to the field of stem cell science, which is ripe with possibility but has struggled to establish its credibility. In 2006, Korean researcher Woo Suk Hwang claimed he had become the first to successfully clone human cells, generating patient-specific lines of stem cells from a persons skin cell. The work turned out to be fraudulent, and the stem cells derived from an already established technique of extracting them from existing embryos.

Since then, both policy makers and those in the field have been more skeptical of milestone claims for good reason, as the latest study shows.

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Stem Cell Researcher Calls for Retraction of His Own Work

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Mouse cells exposed to an acidic environment turned into embryonic-like "STAP" cells. These were used to generate an entire fetus.

A coauthor of a disputed study on a new way generating stem cells through exposure to acid and other stresses has asked for its retraction, it was reported Monday.

Teruhiko Wakayama asked for retraction of two papers describing so-called STAP cells, according to the Yomiuri Shimbun. The papers had been published in the Jan. 30 edition of the journal Nature. Other news outlets, including the Wall Street Journal and the Boston Globe, quickly followed up with the call for retraction.

The original announcement gained worldwide attention because it promised an easy way to generate pluripotent stem cells, which act like embryonic stem cells. Simply immersing cells in an acid bath or squeezing them was enough to reprogram them to the embryonic-like state, the scientists reported. The acronym STAP stands for stimulus-triggered acquisition of pluripotency.

However, researchers attempting to replicate the experiment, including those at the Salk Institute and The Scripps Research Institute in La Jolla, have so far reported failure. And errors in the papers, including duplication of images, have caused scientists to question the findings.

The first author of both papers is Haruko Obokata, 30, of the Riken Center for Developmental Biology in Japan. She was hailed as a scientific prodigy after the research was published. Another coauthor, Charles Vacanti, chairman of the Anesthesiology department at Brigham and Womens Hospital in Boston, had previously said the errors were caused by overwork and didn't affect the results.

As recently as Feb. 27, Wakayama said scientists should give replication efforts a year before passing judgment.

But on Monday, Wakayama said the irregularities render the findings questionable.

"Wakayama said images that show pluripotency of STAP cells look almost identical to those used in Obokatas doctoral thesis about pluripotent stem cells that exist in human body," the Yomiuri Shimbun reported.

Wakayama is probably acting out of a sense of duty, said Jeanne Loring, a stem cell scientist at The Scripps Research Institute.

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STAP cells paper coauthor asks for retraction

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PUBLIC RELEASE DATE:

11-Mar-2014

Contact: Dr. Markus M. Nthen markus.noethen@ukb.uni-bonn.de 49-228-287-51101 University of Bonn

First on top of the world and then in the depths of despair this is what the extreme mood changes for people with bipolar disorder are like. Under the direction of scientists from the University of Bonn Hospital, the Central Institute of Mental Health of Mannheim and the University of Basel Hospital, an international collaboration of researchers discovered two new gene regions which are connected with the prevalent disease. In addition, they were able to confirm three additional suspect genes. In this unparalleled worldwide study, the scientists are utilizing unprecedented numbers of patients. The results are now being published in the renowned journal "Nature Communications".

Throughout the course of their lives, about one percent of the population suffers from bipolar disorder, also known as manic-depressive disorder. The patients undergo a veritable rollercoaster of emotions: During extreme shifts, they experience manic phases with delusions of grandeur, increased drive and a decreased need for sleep as well as depressive episodes with a severely depressed mood to the point of suicidal thoughts. The causes of the disease are not yet fully understood, however in addition to psychosocial triggers, genetic factors play a large role. "There is no one gene that has a significant effect on the development of bipolar disorder," says Prof. Dr. Markus M. Nthen, Director of the Institute of Human Genetics of the University of Bonn Hospital. "Many different genes are evidently involved and these genes work together with environmental factors in a complex way."

Scale of the investigation is unparalleled worldwide

In recent years, scientists at the Institute of Human Genetics were already involved in decoding several genes associated with bipolar disorder. The researchers working with Prof. Dr. Marcella Rietschel from the Central Institute of Mental Health of Mannheim, Prof. Dr. Markus M. Nthen from the University of Bonn Hospital and Prof. Dr. Sven Cichon from the University of Basel Hospital are now using unprecedented numbers of patients in an international research collaboration: New genetic data from 2266 patients with manic-depressive disorder and 5028 control persons were obtained, merged with existing data sets and analyzed together. In total, data on the genetic material of 9747 patients were compared with data from 14,278 healthy persons. "The investigation of the genetic foundations of bipolar disorder on this scale is unique worldwide to date," says Prof. Rietschel from the Central Institute of Mental Health of Mannheim.

The search for genes involved in manic-depressive disorder is like looking for a needle in a haystack. "The contributions of individual genes are so minor that they normally cannot be identified in the 'background noise' of genetic differences," explains Prof. Cichon from the University of Basel Hospital. Only when the DNA from very large numbers of patients with bipolar disorder are compared to the genetic material from an equally large number of healthy persons can differences be confirmed statistically. Such suspect regions which indicate a disease are known by scientists as candidate genes.

Two new gene regions discovered and three known gene regions confirmed

Using automated analysis methods, the researchers recorded about 2.3 million different regions in the genetic material of patients and comparators, respectively. The subsequent evaluation using biostatistical methods revealed a total of five risk regions on the DNA associated with bipolar disorder. Two of these regions were newly discovered: The gene "ADCY2" on chromosome five and the so-called "MIR2113-POU3F2" region on chromosome six. The risk regions "ANK3", "ODZ4" and "TRANK1" have already been described in prior studies. "These gene regions were, however, statistically better confirmed in our current investigation - the connection with bipolar disorder has now become even clearer," says Prof. Nthen.

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New gene for bipolar disorder discovered

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PUBLIC RELEASE DATE:

11-Mar-2014

Contact: Jennifer Quigley jquigley@liebertpub.com 914-740-2100 x2149 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 11, 2014Several young children suffering from a severe degenerative genetic disease received injections of therapeutic genes packaged within a noninfectious viral delivery vector. Safety, tolerability, and efficacy results from this early stage clinical trial are reported in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Marc Tardieu, Universit Paris-Sud and INSERM, and a team of international researchers administered the adeno-associated viral (AAV) vector carrying a normal copy of the N-sulfoglycosamine sulfohydrolase (SGSH) gene into the brains of four children affected by mucopolysaccharidosis type IIIA (MPSIIIA), an inherited lysosomal storage disease in which the SGSH gene is defective. The AAV vector also delivered a sulfatase-modifying factor (SUMF1), needed to activate the SGSH protein.

In addition to measures of toxicity, adverse events, and tolerability, the researchers evaluated the children for brain shrinkage (a characteristic of MPSIIIA) and for changes in behavior, attention, sleep, and cognitive benefit. They describe their findings in the article "Intracerebral administration of AAV rh.10 carrying human SGSH and SUMF1 cDNAs in children with MPSIIIA disease: results of a phase I/II trial."

"This is an important new approach for treating CNS manifestations of lysosomal storage diseases that could be applied across a wide array of disorders," 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 and 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.

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Gene therapy for lysosomal storage disease shown to be safe and well tolerated

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March 12, 2014, 12:13 a.m.

A new technique looks set to soon allow consenting parents to have a child who will not suffer from a serious disability.

Parents in the UK look set to become the first in the world to use a radical IVF technique that some critics have condemned as eugenic engineering. If approved by parliament, so called "three person IVF" could be available on the National Health Service as early as next year.

Supporters hail the technique as a cure for the debilitating and incurable diseases caused by defective genetic material in a part of the mother's egg cell called the mitochondria. It involves implanting the nucleus of a woman's egg into another woman's egg cell which has healthy mitochondria and has had its nucleus removed. The process can take place before or after the egg is fertilised using a man's sperm.

Although the genetic contribution of the egg donor is very small (1 per cent) and won't be detectable in the child's appearance and psychological characteristics, the transfer of genetic material affects the genetic constitution of the egg and the embryo. This means that changes will not only affect the child but also the child's descendants, and there has been criticism of the risk of introducing bad traits through the generations though there have been government assurances that the process will be closely monitored in the UK.

One of the distinctions that ought to play a crucial role in this debate is between genetic engineering that aims to remove a serious disability and engineering designed to make people more intelligent, better looking, stronger or more assertive. And it is the latter that has raised the spectre of eugenics.

Eugenics, of course, is reviled because of the policies adopted by a number of states in the first part of the 20th century, most famously the Nazis, to build a more productive and healthy population by eliminating from the gene pool those regarded it regarded as unfit. And the debate over the ethical implications of mitochondrial transfer is very much alive.

In the Council of Europe, 34 member politicians declared that the creation of babies from the DNA of three parents was a form of eugenics "incompatible with human dignity and international law". They claimed it contravened a European Union human rights convention that forbids genetic interventions that affect the human germ-line by altering the genome of descendants.

The thinking behind this prohibition is that tampering of this kind is not only dangerous, but makes humans into a product of engineering. Even if intentions are good, the use of such techniques undermines the reasons we have for respecting human individuals. Humans are supposed to be valuable in themselves. Products are merely means to ends.

The bad history of eugenics is a good reason why a state should not be allowed to use genetic technology for its purposes. But the practice of mitochondrial transfer and the motivation behind it have nothing to do with eugenics as it was once practiced. It would allow consenting parents to have a child who will not suffer from a serious disability.

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Three-person IVF has nothing to do with eugenics

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Advanced Genetics! - Minecraft Modpacks: Attack of the B-Team - Episode 2
Attack of The B-Team is an awesome new modpack by Technic, that #39;s getting massive attention right now, mainly due to the fact that many popular YouTubers lik...

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Advanced Genetics! - Minecraft Modpacks: Attack of the B-Team - Episode 2 - Video

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How to use Hardy-Weinberg formula in order to solve genetics problems
Hardy-Weinberg equilibrium The Hardy-Weinberg equilibrium is a principle stating that the genetic variation in a population will remain constant from one gen...

By: GeneticsLessons

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How to use Hardy-Weinberg formula in order to solve genetics problems - Video

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Hermitcraft FTB Monster Ep. 11 - Advanced Genetics
Please remember to "Like" the video if you enjoyed 🙂 Playlist - http://www.youtube.com/playlist?list=PL7vFECXWtNMGotvG1sIO8zD32gbgyTFQv Watch More Vids He...

By: KingDaddyDMAC

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Hermitcraft FTB Monster Ep. 11 - Advanced Genetics - Video

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Minecraft Attack of the B Team SMP E06 "Intermediate Advanced Genetics"
Hello from the IntricateAffect SMP Attack of the B Team Server! -we inject ourselves with even MORE Cells -Invite Agent along to gather some wither skin -the...

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Intro to Genetics
Question link: http://quizegg.com/q/82928.

By: Michael Post

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Intro to Genetics - Video

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PUBLIC RELEASE DATE:

11-Mar-2014

Contact: Kristina Goel goel@aaos.org 312-388-5241 American Academy of Orthopaedic Surgeons

NEW ORLEANSGenetics may explain why some senior athletes are high functioning despite having one or both hip abnormalities typically associated with early onset osteoarthritis (OA): developmental dislocation of the hip (dysplasia), a loose hip joint; or femoroacetabular impingement (FAI), a condition in which the hip bones are abnormally shaped, according to new research presented today at the 2014 Annual Meeting of the American Academy of Orthopaedic Surgeons (AAOS).

In the study, "Prevalence of Radiographic Abnormalities in Senior Athletes with Well-functioning Hips," researchers evaluated the hips of 546 senior athletes (1,087 hips) with an average age of 67 (57 percent were male) for radiographic signs of FAI and dysplasia. Eighty-two percent of hips had radiographic evidence of FAI; 67 percent had at least one sign of cam FAI, in which the alpha angle of the bone was 50 on either hip; and 8 percent had isolated pincer impingement FAI, in which an extra bone extends out over the normal rim of the hip socket. Twenty-four percent of the senior athletes had signs of both cam and pincer FAI. Osteoarthritis was present in 17 percent of the hips. Ninety-three percent of the hips with OA had evidence of FAI, and 10 percent, dysplasia.

While hips with FAI were more likely to have OA, 72 percent of the hips with FAI showed little to no evidence of OA. According to the study authors, the findings may indicate that other factors, possibly genetics or the patient's type of cartilage, may play a role in preserving the hip joints in these high functioning senior athletes.

###

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Genetics may explain high-functioning senior athletes with hip abnormalities

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Myriad Genetics Inc. (MYGN), the biggest maker of tests for hereditary risks of breast and ovarian cancer, fell as much as 13 percent after losing its bid to block competing products while a patent-infringement case is pending.

Myriad is unlikely to win lawsuits claiming tests sold by Ambry Genetics Corp. violate its patents and hurt business, U.S. District Judge Robert Shelby in Salt Lake City said yesterday in a decision. There are substantial questions as to whether Myriads ideas cover something eligible for a patent, he said.

Myriad dropped 8.6 percent to $34.49 at 10:01 a.m. New York time, after falling as low as $32.76.

The tests check genes known as BRCA to determine if there is a hereditary risk of developing the diseases. Public awareness of the tests has grown since Academy Award-winning actress Angelina Jolie said last year that she had a double mastectomy after Myriad tests showed she had a mutation linked to the cancer that killed her mother at 56. Salt Lake City-based Myriad gets about three-quarters of its revenue from the tests.

The practical result of Myriads patents has been to hinder or halt follow-up research, data sharing, patient testing, and the creation of additional and more affordable technologies for BRCA1 and BRCA2 testing, Shelby said in his 106-page decision.

Myriads policy of keeping its database private distorts rather than serves the patent systems goal of public disclosure in exchange for exclusive rights, the judge said.

Ambry began offering tests after a June 13 U.S. Supreme Court ruling invalidated some of Myriads patents on genes linked to the diseases. Myriads July 10 lawsuits claim infringement of other patent claims that werent part of that high-court ruling.

Other companies, including Quest Diagnostics Inc., (DGX) also have entered the market and are challenging the Myriad patents. Gene by Gene Ltd. reached an agreement last month that included it stopping sales of its test.

Myriad wanted the competing products halted until a court decision on its patent-infringement claims, which could take a year or more. Shelby had to consider Myriads chance of winning the case and which side would experience the greater harm.

The other owners of the patents -- the University of Utah, the University of Pennsylvania, the Hospital for Sick Children in Toronto and Endorecherche Inc. -- joined in the suits.

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Myriad Falls After Losing Bid to Block Competing Tests

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local injection of gene therapy

By: Loi Do

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local injection of gene therapy - Video

Recommendation and review posted by Bethany Smith

PUBLIC RELEASE DATE:

11-Mar-2014

Contact: M. Angels Valls avalls@esteve.es 34-934-466-286 Universitat Autonoma de Barcelona

ESTEVE has announced the signing of two agreements that will enable it to progress the development of its gene therapeutic for the treatment of Mucopolysaccharidosis type IIIA (MPSIIIA or Sanfilippo A Syndrome) and begin a phase I/II clinical trial in 2015. The agreements are with the North American biotechnology company REGENX Biosciences, LLC (REGENX) and with the French non for profit organization GNTHON.

The license agreement with REGENX grants ESTEVE the right to use the adeno-associated viral vector, NAV rAAV9, in the development and commercialization of its investigational gene therapy for the treatment of Sanfilippo A Syndrome. The vector NAV rAAV9 is an integral part of the investigational therapeutic and enables the gene for the enzyme Sulfamidase, missing or defective in patients with Sanfilippo A Syndrome, to be delivered to and enter cells such as neurons and hepatocytes. Once inside the cells the gene expresses the Sulfamidase enzyme stably, compensating for its absence hence addressing the cause of the disease. The agreement with GNTHON is for the development of the manufacturing process of the investigational gene therapeutic and its production for clinical trial use. The process to be developed will allow the production of the therapeutic for preclinical toxicology studies, the clinical trial and eventually for commercial use.

Public-private partnership ESTEVE-UAB

The Sanfilippo project was initiated by the research team of Dr. Ftima Bosch at the Center for Biotechnology and Gene Therapy (CBATEG) of UAB and since 2009 is being developed within the framework of a public-private partnership between ESTEVE and the University, aimed at developing gene therapies for the treatment of this syndrome and related diseases called mucopolysaccharidoses. This research project was initiated at the CBATEG due to the petition of the Asociacin MPS-Fabry Espaa.

In this partnership, ESTEVE leads all activities associated with the management and protection of intellectual property, regulatory activities, the coordination and supervision of GMP manufacturing, the preclinical toxicology studies as well as all clinical development. The CBATEG research team at the UAB brings to the partnership their scientific know-how and expertise in gene therapy including viral vector design and the development of preclinical disease models.

The investigational gene therapeutic consists of the viral NAV rAAV9, licensed from REGENX, which contains a version of the gene that codes for Sulfamidase that has been optimized to improve its expression levels. Experimentation using preclinical disease models performed by the CBATEG have validated the potential efficacy of this therapeutic approach. The treatment consists in the administration of gene therapy in the cerebrospinal fluid, the fluid that bathes the brain and spinal cord. The viral vector NAV rAAV9 has the advantage of its high affinity for the brain (main organ affected in this disease) than many of the other adeno-associated viral vectors, is harmless, not being known to cause any disease in humans. For its part, once the gene for the Sulfamidase enzyme reaches the cytoplasm of the neuron, it begins the production of the enzyme. Thus, it is produced enzyme which is secreted into spinal fluid, allowing its distribution throughout the brain and spinal cord and also reaching those neurons in which does not incorporate any viral vector. Furthermore, a small proportion of the gene therapeutic passes from the CSF into the peripheral circulatory system, thereby reaching organs such as the liver where it can enter hepatocytes and subsequently produce and secrete the Sulfamidase enzyme which then distributes throughout the body with the aid of the bloodstream.

In the preclinical disease model studied, after administering the gene therapeutic, the levels of Sulfamidase activity significantly increase both in the brain and the rest of the body, the accumulated glycosaminoglycans (substances that build up as a consequence of the disease) are eliminated from within cells, and signs of neuroinflammation disappear. Finally, and most importantly, the behavior is restored and the lifespan is prolonged close to normal.

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ESTEVE and UAB advance in their program to develop a cure for Sanfilippo A Syndrome

Recommendation and review posted by Bethany Smith

PUBLIC RELEASE DATE:

11-Mar-2014

Contact: Press Office presse@afm.genethon.fr AFM-Tlthon

France March 11, 2014 ESTEVE, a Spanish pharmaceutical company devoted to the research, development, manufacturing and commercialization of novel medicines and Genethon, a nonprofit organization dedicated to the research and development of gene therapies for orphan genetic diseases, announce that they have entered into an agreement to manufacture Esteves investigational gene therapy for the treatment of Sanfilippo A Syndrome, AAV9-hsulfamidase, under GMP.

"We are very happy to contribute to the development of the treatment for Sanfillippo A developed by Esteve. The quality of the project and of the teams both academic and industrial participating in the program is outstanding and it is our goal to support clinical development with our biomanufacturing expertise, in the most efficient way, and in the best interest of patients" explained Frdric Revah, Chief Executive Officer of Genethon.

"The signature of this agreement with Genethon is a new milestone for ESTEVE, as it enables us to advance the development of our gene therapeutic for Sanfilippo A towards clinical trials. We are very pleased to collaborate with Genethon, a reference center in manufacturing of gene therapies for rare diseases. Our mission at ESTEVE R&D is the development of innovative products to meet patient needs and that is why this is one of our highest priority projects today." said Albert Esteve, CEO of ESTEVE.

ESTEVE is developing the Sanfilippo project in a Public and Private Partnership (PPP) with the Universitat Autnoma de Barcelona (UAB) for the development of gene therapies for mucopolysaccharidoses. The program relies on state-of-the art science developed at the CBATEG (Center of Animal Biotechnology and Gene Therapy) of the UAB. The most advanced project in this program is the development of a novel gene therapy treatment for Sanfilippo A Syndrome.

###

About MPS III A (Sanfilippo syndrome Type A)

Sanfilippo A Syndrome is a devastating disease that leads to progressive and significant deterioration in mental status of children who rarely live beyond their twenties. Diagnosis of many rare diseases are lengthy and time consuming and is only initiated once the symptoms have begun to appear like the Sanfilippo Syndrome, a lysosomal storage disease caused by the loss of the activity of the enzyme sulfamidase. It affects approximately 1 in 100,000 births and is still largely underdiagnosed.

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GENETHON and ESTEVE announce agreement to manufacture the gene therapy for the treatment of Sanfilippo syndrome

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Best Local Personal Injury Attorneys Oklahoma City
Looking for a Lawyer? Watch Free Legal Advice on How to Obtain Legal Forms, Legal Help Free Legal Aid to Find the BestLocal Attorneys Near You. http://www....

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Best Local Personal Injury Attorneys Oklahoma City - Video

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