Two rare hereditary disorders, one of which kills children within the first few years of life, can be treated with gene therapy, new research from Italy suggests.

In children with the disorders, those who received gene therapy in which a "faulty" gene is replaced with a healthy one showed either improvement in their symptoms or a halt in the disease's progression. The children did not appear to experience serious side effects resulting from the gene therapy.

One disorder, called metachromatic leukodystrophy, causes a buildup of fatty acids in the brain, which leads to cognitive and movement problems and, ultimately, death at an early age.

The researchers treated three children with genetic mutations for metachromatic leukodystrophy, all of whom had older siblings with the condition. Because the patients were very young, ages 7 to 15 months at the study's start, they did not show full symptoms of the condition.

By age 3, one of the children treated with the gene therapy had a normal IQ score and language skills for his or her age, and was able to stand up voluntarily and walk holding someone's hand. In contrast, siblings of this patient who did not receive the therapy were incapable of speech and wheelchair bound by age 3.

The two other patients with the condition, who were also treated with gene therapy, did not show symptoms by age 2, an age at which researchers would have expected symptoms to appear.

Gene therapy was also used to treat three children with Wiskott-Aldrich syndrome, an immune system disorder caused by mutations in a gene called WAS. People with this condition are at increased risk for developing infections, as well as eczema. The children treated with the gene therapy saw their symptoms decrease or disappear within 20 to 30 months of undergoing treatment, the researchers said.

Though the results are promising, the study period was relatively short, and researchers said they need to continue to monitor all six children for changes in their conditions. [9 Most Bizarre Medical Conditions ]

Both groups of children (those with metachromatic leukodystrophy, and those with Wiskott-Aldrich syndrome) received very similar gene-therapy treatments. The researchers removed blood stem cells, called hematopoietic stem cells, from the patients, and used a virus to introduce a corrected form of each patient's faulty gene. These cells were then infused back into the patients.

In patients with Wiskott-Aldrich syndrome, blood stem cells are directly affected by the disease, so the newly infused stem cells replace the diseased cells, the researchers said. For patients with metachromatic leukodystrophy, the newly infused stem cells find their way to the brain, where they release the corrected form of the gene product (a protein), which, in turn, is taken in by the brian cells.

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6 children with rare disorders helped by gene therapy

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Italian researchers have used a defanged version of HIV to replace faulty genes and eliminate devastating symptoms in children suffering two rare and fatal genetic diseases.

Improved gene therapy techniques prevented the onset of metachromatic leukodystrophy in three young children and halted the progression of Wiskott-Aldrich syndrome in three others.

The advance represents a major stride for a field that has struggled to translate experimental successes in lab animals into safe and effective treatments for people, experts said. Researchers may be able to use the team's method as a template, modifying it to treat a variety of diseases.

This is "ammunition for the gene therapy world," said Dr. Theodore Friedmann, a pediatric gene therapist at UC San Diego, who was not part of the study. "The field is slowly but surely making impressive advances against quite untreatable diseases."

The scientists published results from the two clinical trials Thursday in the journal Science.

Metachromatic leukodystrophy affects just 1 in 40,000 to 1 in 160,000 people worldwide; Wiskott-Aldrich syndrome, only 1 to 10 per million males. But both illnesses are devastating. Children with late infantile metachromatic leukodystrophy, the most common form of that disease, begin having trouble walking about a year old and soon after experience muscle deterioration, developmental delays, paralysis and dementia. Most die within a few years of onset.

Kids with Wiskott-Aldrich syndrome suffer from eczema, bruising, nosebleeds and recurrent infections. Most develop at least one autoimmune disorder. A third get cancers, such as lymphoma and leukemia. Life expectancy ranges from 15 to 20 years.

The disorders are challenging when not impossible to treat. No therapy exists for metachromatic leukodystrophy. A bone marrow transplant can stop disease progression for the few Wiskott-Aldrich patients with an immunologically matched sibling, but they may experience severe side effects or death if the donor is not as close a match.

Both diseases are caused by inherited genetic mutations that disrupt the body's ability to produce crucial enzymes. In each trial, researchers took the normal form of the faulty gene and attached it to a virus derived from HIV that had been modified so that it could no longer cause AIDS.

The researchers removed bone marrow stem cells from the patients and then used the lentivirus to infect those cells with the normal genes.

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Gene therapy using HIV helps children with fatal diseases, study says

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How yoga helped me rehabilitate after a spinal cord injury
Mary-Jo Fetterly, is an established yoga teacher who trains people of all abilities. Since a skiing accident left her with a complete C4, C5 quadriplegic inj...

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Winning spinal cord injury cases
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Family & Child Health Home>>Family & Child>>Health news Written by: Ben Hirschler, REUTERS Jul. 3, 2013 HIV. (Wikimedia Commons/CDC/C. Goldsmith/HO)

LONDON - Two men with HIV have been off AIDS drugs for several months after receiving stem-cell transplants for cancer that appear to have cleared the virus from their bodies, researchers reported on Wednesday.

Both patients, who were treated in Boston and had been on long-term drug therapy to control their HIV, received stem-cell transplants after developing lymphoma, a type of blood cancer.

Since the transplants, doctors have been unable to find any evidence of HIV infection, Timothy Henrich of Harvard Medical School and Brigham and Womens Hospital in Boston told an International AIDS Society conference in Kuala Lumpur.

Last July Henrich first reported that the two men had undetectable levels of HIV in their blood after their stem-cell treatment, but at that time they were still taking medicines to suppress HIV.

Using stem-cell therapy is not seen as a viable option for widespread use, since it is extremely expensive, but the latest cases could open new avenues for fighting the disease, which infects about 34 million people worldwide.

The latest cases resemble that of Timothy Ray Brown, known as the Berlin patient, who became the first person to be cured of HIV after receiving a bone marrow transplant for leukemia in 2007. There are, however, important differences.

While Browns doctor used stem cells from a donor with a rare genetic mutation, known as CCR5 delta 32, which renders people virtually resistant to HIV, the two Boston patients received cells without this mutation.

Dr. Henrich is charting new territory in HIV eradication research, Kevin Robert Frost, chief executive officer of the Foundation for AIDS Research, which funded the study, said in a statement.

Scientific advances since HIV was first discovered more than 30 years ago mean the virus is no longer a death sentence and the latest antiretroviral AIDS drugs can control the virus for decades.

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SAN DIEGO, July 10, 2013 /PRNewswire/ --ViaCyte, Inc., a leading regenerative medicine company focused on developing new approaches to treat major diseases through the application of a stem cell-derived cell therapy, announced today that it completed a private equity financing transaction, providing the Company $10.6 million through the sale of Series C-1 Preferred Stock, together with warrants to purchase stock. The financing, conducted as a rights offering to ViaCyte Series B and C Preferred Stock holders, included the Company's largest existing investors - Johnson & Johnson Development Corporation, Sanderling Ventures and Asset Management Company (Johnson Trust).

(Logo: http://photos.prnewswire.com/prnh/20121026/LA00871LOGO-a)

This funding serves as a match for a $10.1 million Strategic Partnership Award (SPA) that was approved last October by the California Institute for Regenerative Medicine (CIRM) to support clinical evaluation of VC-01, ViaCyte's promising encapsulated cell-therapy product being developed as a transformative therapy for patients with type 1 and insulin-dependent type 2 diabetes. In addition, ViaCyte may sell additional shares of Series C-1 Preferred Stock and warrants in one or more subsequent closings that may occur during the remainder of 2013.

The Company will use the funds to pursue clinical development of VC-01. VC-01 is a development-stage product that consists of pancreatic precursor cells (designated PEC-01) manufactured through directed differentiation of stem cells sourced from a proprietary human embryonic stem cell line, and encapsulated in a proprietary, immune protective medical device (the ENCAPTRA device). When implanted under the skin, the encapsulated cells are designed to further differentiate into insulin and other hormone-producing pancreatic cells that regulate blood glucose in a manner similar or identical to a normal pancreas.

Kevin D'Amour, Ph.D., ViaCyte's Chief Scientific Officer, recently provided an update on VC-01 during a presentation at the 73rd Scientific Sessions of the American Diabetes Association entitled Development of an Encapsulated Stem Cell-Based Therapy for Diabetes. The presentation highlighted the tremendous progress ViaCyte has made in the preclinical development of VC-01. ViaCyte is currently planning to file its Investigational New Drug (IND) application with the Federal Drug Administration (FDA) to initiate clinical trials in patients with type 1 diabetes early next year. If VC-01 performs in humans as it has in preclinical studies, it could significantly alleviate or eliminate the challenges and complications for Type 1 diabetics who manage their disease through careful control of diet, monitoring of blood glucose levels and injection of insulin.

The SPA from CIRM provides ViaCyte with $10.1 million to support the clinical study planned for next year and complements previous funding from CIRM. This award reflects CIRM's commitment to follow promising science through the progressive stages of product development. The Company has also received and continues to receive both scientific and financial support for the development of VC-01 from JDRF.

Paul Laikind, Ph.D., ViaCyte's president and chief executive officer, said, "We are very gratified by the continued support of our investors and CIRM as we prepare to evaluate the safety and efficacy of VC-01. This promising product candidate has the potential to vastly improve the lives of millions of patients who currently require insulin injections to survive. Moreover, success of VC-01 will validate ViaCyte's proprietary, stem cell-derived cell therapy platform that has multiple applications, as well as demonstrate the full utility of the Company's encapsulation technology for enabling allogeneic cell therapy treatments."

About ViaCyte

ViaCyte, a private company that has emerged as a leader in the field of regenerative medicine, is currently focused on developing a novel cell therapy for the treatment of diabetes. The Company's lead product is based on the production of pancreatic progenitors derived from human pluripotent stem cells. These cells are implanted in a durable and retrievable encapsulation device. Once implanted and matured, these cells are designed to secrete insulin and other regulatory factors in response to blood glucose levels. ViaCyte's goal for this potentially transformative diabetes product is long term insulin independence without immune suppression, and without risk of hypoglycemia and other diabetes-related complications.

ViaCyte is headquartered in San Diego, California with additional operations in Athens, Georgia. The Company is funded in part by CIRM and JDRF.

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ViaCyte, Inc. raises $10.6 Million in a Private Financing to Support Clinical Development of its Cell Therapy Product ...

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This post is sponsored by IMARC Research, Inc. As the cell therapy market continues to grow, its important to note the similarities between medical device and cell therapy clinical trials. To help illustrate this, weve created an infographic, Cell Therapy: The Fourth Pillar of Healthcare, which is available for download.

Currently three main pillars of medicine exist, including biologics, medical devices, and pharmaceuticals. These pillars of healthcare have strong guidelines and regulations in place. In the U.S., the Food and Drug Administration is beginning to piece together a foundation for an emerging medical industry.

History has shown the three pillar system has been highly successful, improving the lives of millions of people worldwide. However, there are still unmet medical needs.

An article by Chris Mason discusses an emerging fourth pillar in the industry is cell therapy. Cell therapy is innovative but certainly not new technology. In laymans terms, cell therapy consists of introducing new cells into an area of the body in order to treat disease. Cell therapy has 200 years rooted in medical history, including: organ and bone marrow transplants, blood transfusion, and in vitro fertilization, just to name a few.

With cell therapys roots in history and consisting of a unique therapeutic platform technology, this means there is potential for enormous growth in the industry. The spectrum of cell therapies is highly diverse but broadly broken down into two major components:

In simple terms, autologous cell therapy involves harvesting cells from that patient, or from a single person or group of people considered universal donors. Allogeneic cell therapy can involve harvesting cells from one or more universal donors. These cells would then be expanded in a manufacturing setting and cryopreserved for later use.

Currently there is a broad spectrum of activity in the industry with eight FDA/European Medicines Agency (EMA)-approved cell therapies. Will that number rise? In the last 10 years, more than 2,500 cell therapy trials have occurred, with the majority still enrolling and collecting data. These studies included more than 50 percent at the Phase II and Phase III stages, which are critical in determining effectiveness.

So where will we be in five to 10 years? The estimates based on the other pillars in the industry and better success rates than its counterparts (biologics and pharmaceuticals) would lead us to believe we could possibly see more than 100 regulatory approvals in that time.

The FDA continues the process of building a foundation for cell therapy, as it is now emerging as a major therapeutic force and fast-growing sector of healthcare across a wide range of medical indications. Even though cell therapy trials share similarities with medical device trials, cell therapy has its own requirements and challenges that the FDA will need to consider in the regulation of this sector.

Please take time to download IMARC Researchs new infographic depicting the similarities between medical device trials and cell therapy clinical trials.

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Cell therapy: the fourth pillar of healthcare

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TAMPA, Fla., July 9, 2013 /PRNewswire/ -- Stem Cell Therapy for knee joint pain has been involved in multiple clinical trials worldwide. In the United States trials for knee arthritis and back pain with degenerative disc disease have undergone safety trials and phase studies for effectiveness. The safety profile for stem cell therapy in joints has been proven. Adverse effects are not seen as related to the stem cells. These studies were conducted with allogenic (other people's) stem cells. Naturally, if using your own stem cells, the issues which may be raised from someone else's stem cells is not a concern, and are therefore even safer. There are no immune rejection issues or communicable diseases that can be obtained by using your own cells.

Stem Cell Therapy for joints also do not carry surgical risks such as anesthesia, there is no greater risk for other postoperative complications such as blood clots, infections, or need for revision surgery if it is unsuccessful. Dr. Dennis Lox, a Regenerative and Sports Medicine physician in the Tampa Bay, Florida area (www.drlox.com), comments, "Surgery for joint replacement does carry some significant risks, as this is a highly invasive surgery. Knee and other joint replacement surgery consent forms do include the complication of death. More common problems are infection and blood clots. Stem Cell Therapy injections for joints are no more difficult than injecting cortisone into the knee," states Dr. Lox."There is preparation involved to get to that point, however the injection can be a simple, same-day, office-based procedure."

Dr. Lox notes, "Stem Cell Therapy for joint repair has been used for acute and chronic injuries, knee meniscal tears, loss of knee joint cartilage, and to stop the progression of degenerative arthritis. Even avascular necrosis (AVN) or osteonecrosis has been treated with Stem Cell Therapy. The secondary arthritis from joint collapse in avascular necrosis (AVN) can be significant leading to knee joint replacement. The useof stem cells is becoming a more common alternative to joint replacement."

Dr. Lox further notes, "Some patients may have already had one knee joint replaced with a bad outcome, and wish to avoid a second knee replacement. Others may not be healthy enough. Some medical disorders such as bad hear ailments may preclude having a knee replacement. In these cases, having a regenerative medicine procedure is an attractive, conservative option. Patients who are not medically suited for joint or knee replacement are generally good candidates for Stem Cell Therapy. The pursuit of conservative options in patients who wish to avoid surgery for joint disorders, may find Stem Cell Therapy as an attractive alternative."

About Dr. Dennis Lox Dr. Lox practices in the Tampa Bay Florida area. Dr. Lox is a Sports and Regenerative Medicine Physician, who specializes in the use of regenerative and restorative medicine to assist in treating athletic and arthritis conditions. Dr. Lox may be reached at (727) 462-5582 or visit Drlox.com.

http://www.drlox.com

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Stem Cell Therapy for Knee Pain: Safer than Surgery

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CLARKSBURG, Md., July 10, 2013 /PRNewswire/ --BrightFocus Foundation, a nonprofit organization that funds innovative, early-stage research on Alzheimer's disease and the vision diseases of glaucoma and macular degeneration, today announced grant awards totaling more than $7.2 million to 53 scientists in 16 states and four foreign countries.

The funded research projects reflect the full range of new tools and innovationsin imaging technology, gene therapy, and cell regenerationthat scientists are using to better understand how diseases of mind and sight develop. Study results could lead to new therapies to prevent or treat these diseases.

"Investment in research has advanced our understanding of Alzheimer's and vision diseases," said BrightFocus President and CEO, Stacy Pagos Haller."Now, thanks to new developments in genetics, neurology and imaging, the potential for scientists to make groundbreaking research discoveries is taking off. BrightFocus Foundation is more committed than ever to making this cutting-edge research possible, particularly at a time when government research funding levels are stagnant."

BrightFocus has provided $130 million to date in research funding, awarding more than $26 million for research on diseases of mind and sight in the last four years alone. This year's grantees include researchers from across the U.S., as well as Australia, Great Britain, Ireland, and Israel.

Alzheimer's Disease Research

Alzheimer's is a devastating degenerative disease that irreversibly destroys memory and other brain function over time. BrightFocus-funded scientists are studying various ways in which the "memory pathways" in the brainthe systems by which brain cells communicatecan go awry in this disease.

Some researchers are investigating whether certain chemicals control the "switches" to these pathways; others are using highly refined brain imaging or magnetic brain stimulation techniques to learn more about pathways; and some are using cell-based therapies to try to restore the brain circuits made during memory formation. Still others are studying how problems with brain blood flow contribute to Alzheimer's disease.

Glaucoma Research

For all three diseases, scientists want to know how inflammation and the body's immune response system may be involved, making the body turn against healthy cells in the brain or eyes. Several glaucoma researchers are examining the mind-eye connection, and why changes in the brain may contribute to the development of glaucoma long before vision loss occurs. Early detection is particularly important for glaucoma: in the U.S., an estimated half of the three million people with glaucoma may not know they have the disease.

Macular Degeneration Research

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BrightFocus Foundation Announces $7.2 Million in New Grants For Alzheimer's and Vision Disease Research

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Public release date: 10-Jul-2013 [ | E-mail | Share ]

Contact: Alice L. Kirkman akirkman@brightfocus.org 301-556-9349 AHAF-American Health Assistance Foundation

Clarksburg, MDBrightFocus Foundation, a nonprofit organization that funds innovative, early-stage research on Alzheimer's disease and the vision diseases of glaucoma and macular degeneration, today announced grant awards totaling more than $7.2 million to 53 scientists in 16 states and four foreign countries.

The funded research projects reflect the full range of new tools and innovationsin imaging technology, gene therapy, and cell regenerationthat scientists are using to better understand how diseases of mind and sight develop. Study results could lead to new therapies to prevent or treat these diseases.

"Investment in research has advanced our understanding of Alzheimer's and vision diseases," said BrightFocus President and CEO, Stacy Pagos Haller. "Now, thanks to new developments in genetics, neurology and imaging, the potential for scientists to make groundbreaking research discoveries is taking off. BrightFocus Foundation is more committed than ever to making this cutting-edge research possible, particularly at a time when government research funding levels are stagnant."

BrightFocus has provided $130 million to date in research funding, awarding more than $26 million for research on diseases of mind and sight in the last four years alone. This year's grantees include researchers from across the U.S., as well as Australia, Great Britain, Ireland, and Israel.

Alzheimer's Disease Research

Alzheimer's is a devastating degenerative disease that irreversibly destroys memory and other brain function over time. BrightFocus-funded scientists are studying various ways in which the "memory pathways" in the brainthe systems by which brain cells communicatecan go awry in this disease.

Some researchers are investigating whether certain chemicals control the "switches" to these pathways; others are using highly refined brain imaging or magnetic brain stimulation techniques to learn more about pathways; and some are using cell-based therapies to try to restore the brain circuits made during memory formation. Still others are studying how problems with brain blood flow contribute to Alzheimer's disease.

Glaucoma Research

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BrightFocus Foundation announces $7.2 million in grants for Alzheimer's and vision disease research

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July 10, 2013 - Columnist, author, and small business owner Gene Marks will present opening keynote at IPC Technology Market Research Conference (TMRC), which will take place September 25 and feature "The Next Generation: Creating the Electronics Industry of the Future" theme. Keynote,"Economic, Political and Other Key Trends: 10 Things Happening Today That Will Affect Your Business Tomorrow," will examine how economy, Washington, and technology affect businesses. IPC-Association Connecting Electronics Industries 3000 Lakeside Drive Bannockburn, IL, 60015 USA Press release date: July 3, 2013

Columnist and Author to Discuss Economic and Political Trends Affecting Business

BANNOCKBURN, Ill., USA Columnist, author and small business owner Gene Marks will present the opening keynote at the IPC Technology Market Research Conference (TMRC), Wednesday, September 25, at the Allerton Hotel in Chicago. The event, themed The Next Generation: Creating the Electronics Industry of the Future, will be co-located with the IPC Management Meetings, which will be held on Tuesday, September 24.

In keeping with the forward-looking theme of the conference, Marks presentation, Economic, Political and Other Key Trends: 10 Things Happening Today That Will Affect Your Business Tomorrow, will take a thought-provoking and entertaining look at how the economy, Washington and technology affect businesses, and what successful companies are doing today to ensure future profitability.

A columnist for The New York Times, Forbes, Inc. Magazine, Fox Business and The Huffington Post, Marks is also the author of several books and often appears on Fox News, Bloomberg and CNBC to discuss business issues. In addition, he owns and operates The Marks Group PC, a 10-person firm that provides customer relationship management technology and consulting services to small- and medium-sized businesses.

Gene Marks insightful keynote will be an excellent kick-off to this highly interactive and empowering event, says Sanjay Huprikar, IPC vice president of member success. Hes very knowledgeable about business and economic issues, and has an engaging presentation style that will get conference participants excited about our industrys future and their role in creating it.

The focus and topics for the TMRC are determined in collaboration with a steering committee of industry professionals with a vested interest in a thriving electronics industry. Other sessions at the TMRC will address global economics, high-performance computing systems in the military, nanotechnology, graphene, 3-D packaging, new materials and an interactive session about how companies can position themselves for the future. Representatives from all areas of the electronics industry are invited to attend the TMRC.

More information about the TMRC and IPC Management Meetings is available at http://www.ipc.org/tmrc-mm or by contacting Susan Filz, IPC director of industry programs at +1 847-597-2884.

About IPC IPC (www.IPC.org) is a global industry association based in Bannockburn, Ill., dedicated to the competitive excellence and financial success of its 3,300 member companies which represent all facets of the electronics industry, including design, printed board manufacturing, electronics assembly and test. As a member-driven organization and leading source for industry standards, training, market research and public policy advocacy, IPC supports programs to meet the needs of an estimated $2.17 trillion global electronics industry. IPC maintains additional offices in Taos, N.M.; Arlington, Va.; Stockholm, Sweden; Moscow, Russia; Bangalore, India; Bangkok, Thailand; and Shanghai, Shenzhen, Chengdu, Suzhou and Beijing, China.

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Gene Marks to Keynote at IPC Technology Market Research Conference

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Public release date: 10-Jul-2013 [ | E-mail | Share ]

Contact: Margaret Mroziewicz mmroziewicz@cifar.ca 416-971-4876 Canadian Institute for Advanced Research

Every cell in an organism's body has the same copy of DNA, yet different cells do different things; for example, some function as brain cells, while others form muscle tissue. How can the same DNA make different things happen? A major step forward is being announced today that has implications for our understanding of many genetically-linked diseases, such as autism.

Scientists know that much of what a gene does and produces is regulated after it is turned on. A gene first produces a molecule called RNA, to which tiny proteins called RNA binding proteins (RBPs) bind and control its fate. For instance, some of these proteins cut out parts of the RNA molecule so that it makes a particular protein, while other RBPs help destroy the RNA before it even produces a protein.

But these mechanisms are not well understood because the RNA sequences, which the RBPs bind to, have been so difficult to decipher. To fully understand gene regulation (and disregulation, as in the case of disease), scientists have needed to employ advanced lab techniques and data analysis to identify the patterns of the RNA sequences.

This gap in knowledge motivated a team of researchers co-led by Senior Fellow Tim Hughes (University of Toronto and the Canadian Institute for Advanced Research) to produce the first-ever compendium of RNA-binding sequences, which was published in Nature on July 11, 2013.

"It took us a long time to generate and analyze the data," explains Hughes. "After spending years developing and perfecting a method, we started looking at all the proteins in humans, fruit flies and other complex organisms that look like they may bind RNA and found which sequences they like to bind to. Our compendium of RNA-binding sequences will become a resource for researchers in this field, and will be especially useful in human genetic analysis."

The team found that humans and fruit flies have similar RBPs, since they derive from a common ancestor, and that in many cases they essentially bind the same sequences. The researchers anticipate that this is the case for proteins in other organisms.

"We looked at just over 200 proteins in total, but can probably infer the preference for tens of thousands of proteins in many other organisms," says Hughes.

In addition, many of the sequences similar across species were at the end of the RNA transcript, which is a region associated with regulation of RNA decay or movement of the RNA to another part of the cell. "This indicates that there is probably more regulation of gene expression itself at the level of stability or destruction of RNA," explains Hughes.

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Scientists decode mystery sequences involved in gene regulation

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Mini Documentary - The Hedonistic Imperative - David Pearce (Take 1)
http://www.hedweb.com/ - The Hedonistic Imperative outlines how genetic engineering and nanotechnology will abolish suffering in all sentient life. Some foot...

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Mini Documentary - The Hedonistic Imperative - David Pearce (Take 1) - Video

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Battle Purple Tomato: Genetically Engineered vs. Non-GMO

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In5D Radio - Jordan Maxwell - Do Your Homework! Episode 16 | In5D.com
Gregg Prescott and Kendra Gilbert interviewed our returning guest, Jordan Maxwell, on In5D Radio as we covered a wide range of topics including astrotheology...

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In5D Radio - Jordan Maxwell - Do Your Homework! Episode 16 | In5D.com - Video

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Using a novel form of immune-genetic therapy, researchers from Yale School of Medicine and the Jagiellonian University College of Medicine in Poland have successfully inhibited a strong immune allergic inflammatory response in the skin of mice. The results suggest the technique could be used to combat a variety of diseases.

We use an antibody coating we chose to deliver therapeutic genetic material we selected to target cells, said Dr. Philip Askenase, professor of medicine and senior author of the study published July8 in the Journal of Allergy and Clinical Immunology.

The delivery system consists of naturally occurring nanoparticles called exosomes that are about one thousandth the size of donor cells that release them. These tiny vesicles were once thought to contain only unneeded cellular debris. However, in the last decade, scientists have shown that there are billions of exosomes in the circulation and that they carry genetic instructions in the form of micro-RNAs (miRNA) to regulate the functions of nearby and distant cells.

Askenase and colleagues found that exosomes could be coated with an antibody of their choosing. These nanovesicles were able to deliver therapeutic miRNA to specific cells targeted by the antibody. In the current study, the coated exosomes delivered their miRNA cargo to immune system cells, inhibiting an active allergic disease response in the skin of mice.

These natural nanoparticles are present throughout the body, said Dr. Krzysztof Bryniarski of Jagiellonian University and lead author of the paper. They seem to be a superior delivery system compared to artificial nanoparticles currently in use, which often are eliminated from the body because they are sensed as artificial.

In theory, the researchers said, the natural nanoparticles coated with chosen specific antibodies and loaded with selected miRNAs could be used to specifically target and then genetically alter crucial cells involved in allergic conditions such as asthma, autoimmune responses, and potentially even cancers and neurological diseases.

The research was funded by the National Institute of Allergy and Infectious Diseases at the NIH (AI-00714 and 076366), and the Polish Ministry of Science and Higher Education (N401 092 31/2176 and K/ZDS/001429).

(Image via Shutterstock)

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Nature’s own nanoparticles harnessed to target disease

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Awareness of genetics, ovarian cancer link could be lifesaver
Angelina Jolie #39;s double mastectomy made genetics and breast cancer a hot topic. But women also need to focus on the links between genetics and the "below-the-belt cancers."

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Awareness of genetics, ovarian cancer link could be lifesaver - Video

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Newswise BETHESDA, MD July 9, 2013 Six graduate students and one undergraduate were named as recipients of Genetics Society of America (GSA) poster awards at the 19th International C. elegans Meeting, held June 2630 on the campus of the University of California, Los Angeles. More than 1,750 scientists attended the worlds largest gathering of those conducting research using the nematode worm, Caenorhabditis elegans, a model organism that lends itself to easy investigation where findings can easily be translated to humans.

Recipients were selected from almost 400 eligible posters presented at the meeting. The 93 faculty who judged the poster presenters had quite a challenge to select the best ones, because there were so many excellent posters. They did a great job judging and selecting the finalists, said Tina L. Gumienny, PhD, co-chair of the poster competition. The caliber of science at this year's poster session was amazing and bodes well for the future of C. elegans research, added Erin J. Cram, PhD, poster co-chair.

One winner was selected in each topic area:

Cell Biology Tisha E. Bohr University of California, Santa Cruz, CA Spindle assembly checkpoint proteins regulate and monitor meiotic synapsis in C. elegans

Development and Evolution Tulsi Patel Columbia University Medical Center, New York, NY Cell Fate Restriction and Reprogramming in C. elegans

Gene Regulation and Genomics Ashlyn D. Ritter University of Massachusetts Medical School, Worcester, MA Complex expression dynamics and robustness in C. elegans insulin networks

Methods and Technology Valeriya Laskova Samuel Lunenfeld Research Institute, Toronto, ON, Canada Mapping the entire connectome of C. elegans L1 larvae

Neurobiology Julie E. Grimm Technion Institute of Technology, Haifa, Israel How to Fix a Broken Neuron

Physiology Kurt J. Warnhoff Washington University, St. Louis, MO natc-1 mediates stress resistance and dauer formation as a downstream effector of the insulin/IGF-1 signaling pathway

Undergraduate Michael James Hoy College of the Holy Cross, Worcester, MA The C. elegans Insulin Signaling Response to Glucose Stress Requires Unique Regulators

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Seven Receive Genetics Society of America Poster Awards at Worm Meeting

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Myriad sues competitor over cancer gene test

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Home Mail News Sports Finance Weather Games Groups Answers Flickr More omg! Shine Movies Music TV Health Shopping Travel Autos Homes Search News Search Web Sign In Mail Help Account Info Help Suggestions Yahoo! Home Video Photos GMA Year in Review LiveRoom Odd Comics Travel Opinion Trending Now Who Knew? Weather The Upbeat U.S. U.S. Video GMA Education Religion Crimes and Trials The Lookout Local Contributor Network Year In Review World World Video Middle East Europe Latin America Africa Asia Canada Australia/Antarctica Business Video Exclusives Today's Markets Stocks Personal Finance Marketplace Entertainment Video Clinton Concert Celebrity TV Movies Music Fashion Books Arts Theater Dear Abby Comics Odd News Sports Video NFL MLB NBA NCAAF NCAAB Soccer Cycling NHL Tennis Golf Boxing Motor Sports MMA Olympics Tech Gadgets Wireless Apple Social Media Security Open Source Gaming Apps This Could Be Big Upgrade Your Life Politics Remake America The Issues Women and Politics Press Releases Video Science Science Video Weather News Space / Astronomy Pets Dinosaurs / Fossils Biotech Energy Green Health Video Weight Loss Cancer Sexual Health Medications/Drugs Parenting/Kids Seniors/Aging Diseases/Conditions Blogs The Lookout The Sideshow Around the World Katie's Take Power Players This Could Be Big Newsmakers Trending Now Just Explain It The Upbeat Local Popular Search Keyword News Search Featured Videos Photos Just Explain It Katie's Take Weather The Upbeat Newsmakers

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Myriad sues competitor overs cancer gene test

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ALISO VIEJO, Calif.--(BUSINESS WIRE)--

Ambry Genetics announced today that it intends to vigorously defend itself against the patent infringement suit filed yesterday in the United States District Court for the District of Utah by Myriad Genetics, the University of Utah Research Foundation, the Trustees of the University of Pennsylvania, HSC Research and Development LP, and Endorecherche, Inc. The complaint alleges that Ambry Genetics infringes on certain Myriad BRCA1 and BRCA2 patents by offering diagnostic BRCA1 and BRCA2 testing to women.

Ambry Genetics began offering BRCA1 and BRCA2 diagnostic testing following the United States Supreme Courts landmark June 13, 2013 decision in AMP et al v. Myriad Genetics, et al. That lawsuit was brought by a coalition of patients, physicians and health care groups to challenge Myriads patents directed at BRCA1 and BRCA2 genes. As alleged in that lawsuit, Myriad maintained a monopoly over diagnostic testing of BRCA1 and BRCA2 genes under the Myriad patents and threatened legal actions against entities that wished to provide BRCA1 and BRCA2 gene testing to women. Following a multi-year legal battle, the unanimous Supreme Court decision held that genomic sequences, whether isolated or not, may not be patented and declared invalid the Myriad BRCA1 and BRCA2 patent claims challenged in that suit.

Ambry Genetics supports the Supreme Courts decision and will vigorously defend its position, said Charles Dunlop, Chief Executive Officer of Ambry Genetics. We have had an overwhelming response from our clients seeking an alternative laboratory to perform BRCA testing and Ambry is fully committed to supporting our clients and patients moving forward.

Ambry Genetics is the leader in hereditary cancer diagnostics. With the addition of BRCA1 and BRCA2 testing to its menu, Ambry Genetics now offers the most comprehensive germline cancer-testing menu in the industry, providing patients with the most accurate diagnosis currently available. Menu highlights include: BRCAplus, BreastNext, OvaNext, ColoNext, CancerNext and coming soon multi-gene NGS panels aimed to analyze genes implicated in hereditary renal, pancreatic and PGL-PCC cancers.

About Ambry Genetics

Ambry Genetics is a College of American Pathologists (CAP)-accredited and Clinical Laboratory Improvement Amendments (CLIA)-certified commercial clinical laboratory with headquarters in Aliso Viejo, Orange County, California. Since its founding in 1999, it has become a leader in providing genetic services focused on clinical diagnostics and genomic services, particularly in sequencing and array services. Ambry has established a reputation for unparalleled service and for over a decade has been at the forefront of applying new technologies to the clinical molecular diagnostics market and to the advancement of disease research. To learn more about testing and services available through Ambry Genetics, visit http://www.ambrygen.com.

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Ambry Genetics Will Vigorously Defend Against BRCA1 and BRCA2 Gene Patent Infringement Suit

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Scientists have created genetically-engineered mice with artificial human chromosomes in every cell of their bodies, as part of a series of studies showing that it may be possible to treat genetic diseases with a radically new form of gene therapy.

Click image above to enlarge graphic

In one of the unpublished studies, researchers made a human artificial chromosome in the laboratory from chemical building blocks rather than chipping away at an existing human chromosome, indicating the increasingly powerful technology behind the new field of synthetic biology.

The development comes as the Government announces today that it will invest tens of millions of pounds in synthetic biology research in Britain, including an international project to construct all the 16 individual chromosomes of the yeast fungus in order to produce the first synthetic organism with a complex genome.

A synthetic yeast with man-made chromosomes could eventually be used as a platform for making new kinds of biological materials, such as antibiotics or vaccines, while human artificial chromosomes could be used to introduce healthy copies of genes into the diseased organs or tissues of people with genetic illnesses, scientists said.

Researchers involved in the synthetic yeast project emphasised at a briefing in London earlier this week that there are no plans to build human chromosomes and create synthetic human cells in the same way as the artificial yeast project. A project to build human artificial chromosomes is unlikely to win ethical approval in the UK, they said.

However, researchers in the US and Japan are already well advanced in making mini human chromosomes called HACs (human artificial chromosomes), by either paring down an existing human chromosome or making them de novo in the lab from smaller chemical building blocks.

Natalay Kouprina of the US National Cancer Institute in Bethesda, Maryland, is part of the team that has successfully produced genetically engineered mice with an extra human artificial chromosome in their cells. It is the first time such an advanced form of a synthetic human chromosome made from scratch has been shown to work in an animal model, Dr Kouprina said.

The purpose of developing the human artificial chromosome project is to create a shuttle vector for gene delivery into human cells to study gene function in human cells, she told The Independent. Potentially it has applications for gene therapy, for correction of gene deficiency in humans. It is known that there are lots of hereditary diseases due to the mutation of certain genes.

Synthetic biology is loosely defined as designing new kinds of life-forms or making new genetic arrangements that do not exist in nature, which could provide practical benefits to society, notably in medicine, manufacturing or environmental monitoring.

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Exclusive: Scientists create human chromosome in breakthrough that could revolutionise medicine

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Natural Molecular Testing Canada: Benefits of Personalized Medicine
Testing done by NMT provides physicians with the state of the art tool that gives them data to help them understand which medications and therapies are worki...

By: NMTCanadaLtd

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Natural Molecular Testing Canada: Benefits of Personalized Medicine - Video

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19. From Genes to Personalized Medicine: Innovations in Bladder Cancer Research - Chin - LA
Arnold Chin, MD, PhD, UCLA LA Patient Forum.

By: Bladder Cancer Advocacy Network

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19. From Genes to Personalized Medicine: Innovations in Bladder Cancer Research - Chin - LA - Video

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