Archive for the ‘Gene Therapy Research’ Category

What #39;s the Future Like: Socio-Genetics
This is the second video in the "What #39;s the Future Like" series. Socio-genetics has been great for society and has solved a lot of long standing problems. I ...

By: Maxwell Hammer

The rest is here:
What's the Future Like: Socio-Genetics - Video

Genetics and Building Your Physique
http://www.gettingtoshredded.com Too many people use the excuse that their genetics are holding them back. I #39;m pretty depleted, but I #39;m forcing myself to do ...

By: Getting2Shredded

Follow this link:
Genetics and Building Your Physique - Video

Cy Humphreys - Simple Genetics
ARTHOUSE010: Cy Humphreys - Live Let Cy EP Hailing from Wrexham in North Wales. Cy is an outspoken DJ and club critic, plying his trade venues and parties ...

By: Jamie Anderson

Read the original post:
Cy Humphreys - Simple Genetics - Video

19 Population genetics
For more information, log on to- http://shomusbiology.weebly.com/ Download the study materials here- http://shomusbiology.weebly.com/bio-materials.html.

By: Suman Bhattacharjee

Read the rest here:
19 Population genetics - Video

Are your genetics keeping you skinny?
Download my FREE 12 week muscle building program: http://musclemonsters.com/massinaflash Follow me on facebook: http://facebook.com/musclemonsters Follow me ...

By: musclemonsters

The rest is here:
Are your genetics keeping you skinny? - Video

Bigger Butt Secrets
Bigger Butt Secrets Ebook by Jayna Davis Free Download And Discount Visit: http://www.makeoverbutt.com/ for more information. Bigger Butt Secrets Most women ...

By: makeoverbutt4u

Read this article:
Bigger Butt Secrets - Video

GENVIP - Genetics, Vaccines, Infections and Pediatrics research group
More information at: http://www.genvip.eu or @GENVIP_RESEARCH Infectious diseases are the main cause of morbidity and mortality among children and infants across the world. Five million children...

By: fmartor

Go here to see the original:
GENVIP - Genetics, Vaccines, Infections and Pediatrics research group - Video

Gene therapy: not just for mice.

Genetic traits, like a bulbous nose or balding, give some people reasons to moan about what they inherited from their parents. But more serious genetic flaws can cause debilitating disease. Now, Italian researchers have come up with a way of treating one such inherited disease and reversing another using a promising new method of gene therapy.

The idea behind gene therapy is to replace a faulty gene with a shiny new version that works properly. Modified versions of viruses, which have been sculpted by millions of years of evolution, perfectly penetrate human cells. They act as courriers delivering DNA payloads to defective cells and ensure they are stably inherited.

This deceptively simple idea, though, has been challenging to achieve in practice. The first commercial gene therapy product, Glybera, only received regulatory approval in 2012.

Part of the reason for this is the difficulty of successfully clearing three hurdles at once: delivering replacement genetic information to the exact cells that need help, getting this information safely translated in high enough volumes to overcome the defects, and stopping the immune system from reacting to normal genes when it has grown used to only seeing mangled ones.

Now, a team led by Alessandra Biffi at the San Raffaele Scientific Institute in Milan, Italy, reports inScience that they have developed a new approach that navigates each of these hurdles to treat three children with metachromatic leukodystrophy (MLD), a devastating inherited disease that affects around 1 in 40,000 people.

MLD usually manifests in early childhood and kills patients just a few years after the first symptoms appear. It is caused by a defect in a single gene, ARSA. This gene encodes information used by the lysosome, a piece of recycling machinery used by human cells to break down unwanted material. When this recycling process does not work properly in nerve cells, as is the case with MLD patients, they become filled with rubbish and begin to slowly decline, leading to brain and spinal cord degeneration, as well as sensory deprivation.

Supplying a replacement ARSA gene to affected cells in the nervous system is a tricky task, because these areas are heavily protected. To overcome these defenses, the team employed haematopoietic stem cells (HSCs), which can usually be found nestling quietly in the bone marrow, as stealthy genetic courriers. A tiny number of these cells were harvested from each patient, loaded with benign viruses carrying a working copy of ARSA, and put back into the bloodstream.

These engineered cells either lodged in the bone marrow or continued to travel around the body in the blood, where they corrected defective cells in the nervous system by supplying the normal version of ARSA. Because these were stem cells, they also reproduced to form new blood cells that themselves took on the same supportive roles.

Most MLD patients produce a garbled version of ARSA that has a very low level of activity, nowhere near enough to let the lysosome carry out its normal job. Restoring partial activity is not enough to make a clinical impactlevels must be hiked to make an obvious difference.

See the original post:
Two successful gene therapy trials block inherited diseases in humans

Public release date: 11-Jul-2013 [ | E-mail | Share ]

Contact: Glenna Picton picton@bcm.edu 713-798-4710 Baylor College of Medicine

HOUSTON -- Two Houston researchers from Baylor College of Medicine and Texas Children's Hospital were part of an international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency.

The new research, led by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy was published in Science Express.

The disorder that weakens the body's immune system is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donoroften a sibling or relative. It can be curative for some patients mostly those who have a strongly matching donor.

An alternative is to obtain blood stem cells from patient, and, in the laboratory, use gene therapy involving a form of retrovirus to take the normal gene into the cells to correct the defect. The patients are then given back the genetically changed blood cells back.

This approach has been successful in a number of diseases, including who had Wiskott-Aldrich Syndrome. However, over the long term, some patients with immune deficiencies, including those with Wiskott-Aldrich Syndrome, developed blood cancers.

Researchers believe the viral vector used to take the gene into the cell inserted itself next to a oncogene in the DNA, turning it on and causing the cancers.

In this new research, the team used a partially inactivated lentivirus to take the normal gene into the cell, while reducing the risk of the gene inserting next to a cancer-promoting gene.

In these cases for whom there was no matching donor, the researchers led by Alessandro Aiuti of San Raffaele Telethon Institute for Gene Therapy in Milan, Italy; and including Dr. Jordan Orange, professor of pediatrics--rheumatology and Pinaki Banerjee, assistant professor of pediatrics human immunology at BCM and Texas Children's, took the patients' own blood stem cells and, in the laboratory, used the lentiviral vector combined with the normal WASP gene to correct the genetic defect in the blood. After a special treatment to eliminate their defective immune system, they received their own blood cells that had been altered to contain the normal WASP gene.

Continue reading here:
Gene therapy using lentivirus promising in 3 youngsters

WASHINGTON: A new type of gene therapy has shown promise in wiping out two rare childhood diseases, apparently without the risks of causing cancer, international researchers said Thursday.

The method used an HIV virus vector and the patients' own blood stem cells to deliver a corrected version of a faulty gene, said the report in the US journal Science.

As a result, six children are doing well, 18 to 32 months after their operations, said lead scientist Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan.

"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging. The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases."

Three of the children suffer from metachromatic leukodystrophy, a disease of the nervous system which is caused by mutations in the ARSA gene.

Babies with the disease appear healthy when they are born, but as they grow, they start to lose cognitive and motor skills. There is no cure.

The new gene therapy approach has halted progression of the disease in these three children, researchers said.

The other three children in the study have Wiskott-Aldrich syndrome, which is caused by mutations in the WAS gene and results in recurring infections, autoimmune diseases, frequent bleeding, and a high risk of cancer.

The treatment has caused the children's symptoms to lessen or vanish altogether, the researchers said.

Previous attempts at gene therapy for a range of diseases, including Wiskott-Aldrich Syndrome, have shown some success, but over the long term it was discovered that immune-compromised patients sometimes developed blood cancer.

Read the original here:
New gene therapy hope for rare childhood diseases

Six kids are healthy, up to three years after treatment

By Tina Hesman Saey

Web edition: July 11, 2013

A virus derived from HIV can safely fix broken immune systems and correct genetic diseases, suggest two new studies involving children with rare conditions.

For both studies, researchers put healthy genes into the childrens own DNA using lentiviruses, in this case genetically engineered versions of HIV that can no longer cause disease. Earlier gene therapy trials using different viruses had a flaw: When the viruses plunked themselves into the patients DNA, they sometimes amped up activity of neighboring cancer-causing genes, leading to leukemia. That side effect, along with the death of a young man participating in another clinical trial, nearly halted gene therapy in the United States in the early 2000s.

Now, researchers led by Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan have altered the lentiviruses so that they wont accidently turn on nearby genes. The researchers then infect bone marrow stem cells with lentiviruses carrying the appropriate gene and transplant the stem cells into patients.

In one study, three boys with Wiskott-Aldrich syndrome, an inherited disease that disables the immune system, received gene therapy. Now, two to three years after the therapy, the former bubble boys have healthy immune systems, Naldini and colleagues report July 11 in Science. The boys also show no signs of developing leukemia which should help allay concerns about the teams gene therapy approach, says Todd Rosengart, a surgeon and gene therapy researcher at Baylor College of Medicine in Houston.

In the second trial, Naldini and his colleagues treated three children with a metabolic disease called metachromatic leukodystrophy. Children with the disease lack an important enzyme. As a result, they gradually become paralyzed and suffer damage to their ability to think, dying within a couple of years. Up to two years after the therapy, the children in the study are still making enough of the enzyme to keep their brain and spinal cord working normally with no sign of leukemia, the researchers report in the same issue of Science.

The results are encouraging, says Uta Griesenbach, a gene therapist at Imperial College London. Even after fairly long-term follow up, it appears to be safe and effective. The boys arent out of the woods yet some of the patients in the original gene therapy trials didnt develop cancer until four years after treatment. But Griesenbach says that the children in the new studies dont have warning signs of cancer.

Because the lentiviruses appear safe and work so well, scientists may start doing gene therapy for more common conditions such as Parkinsons disease, says Senlin Li, a medical researcher at the University of Texas Health Science Center at San Antonio.

Read more here:
Gene therapy treats children with rare diseases

A new type of gene therapy has shown promise in wiping out two rare childhood diseases, apparently without the risks of causing cancer, international researchers said Thursday.

The method used an HIV virus vector and the patients' own blood stem cells to deliver a corrected version of a faulty gene, said the report in the US journal Science.

As a result, six children are doing well, 18 to 32 months after their operations, said lead scientist Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan.

"Three years after the start of the clinical trial, the results obtained from the first six patients are very encouraging. The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases."

Three of the children suffer from metachromatic leukodystrophy, a disease of the nervous system which is caused by mutations in the ARSA gene.

Babies with the disease appear healthy when they are born, but as they grow, they start to lose cognitive and motor skills. There is no cure.

The new gene therapy approach has halted progression of the disease in these three children, researchers said.

The other three children in the study have Wiskott-Aldrich syndrome, which is caused by mutations in the WAS gene and results in recurring infections, autoimmune diseases, frequent bleeding, and a high risk of cancer.

The treatment has caused the children's symptoms to lessen or vanish altogether, the researchers said.

Previous attempts at gene therapy for a range of diseases, including Wiskott-Aldrich Syndrome, have shown some success, but over the long term it was discovered that immune-compromised patients sometimes developed blood cancer.

Link:
New gene therapy hope for rare childhood disease

July 11, 2013 Two Houston researchers from Baylor College of Medicine and Texas Children's Hospital were part of an international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency.

The new research, led by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy was published in Science Express.

The disorder that weakens the body's immune system is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donor -- often a sibling or relative. It can be curative for some patients -- mostly those who have a strongly matching donor.

An alternative is to obtain blood stem cells from patient, and, in the laboratory, use gene therapy involving a form of retrovirus to take the normal gene into the cells to correct the defect. The patients are then given back the genetically changed blood cells back.

This approach has been successful in a number of diseases, including who had Wiskott-Aldrich Syndrome. However, over the long term, some patients with immune deficiencies, including those with Wiskott-Aldrich Syndrome, developed blood cancers.

Researchers believe the viral vector used to take the gene into the cell inserted itself next to a oncogene in the DNA, turning it on and causing the cancers.

In this new research, the team used a partially inactivated lentivirus to take the normal gene into the cell, while reducing the risk of the gene inserting next to a cancer-promoting gene.

In these cases for whom there was no matching donor, the researchers led by Alessandro Aiuti of San Raffaele Telethon Institute for Gene Therapy in Milan, Italy; and including Dr. Jordan Orange, professor of pediatrics--rheumatology and Pinaki Banerjee, assistant professor of pediatrics -- human immunology at BCM and Texas Children's, took the patients' own blood stem cells and, in the laboratory, used the lentiviral vector combined with the normal WASP gene to correct the genetic defect in the blood. After a special treatment to eliminate their defective immune system, they received their own blood cells that had been altered to contain the normal WASP gene.

After 20 to 30 months, the three children showed significant improvement. New blood cells had the corrected WASP gene.

Orange referred one of the patients for treatment and saw one of them at a recent meeting. He and Banerjee contributed to the analysis of the gene corrected patient cells at the laboratory level using highly quantitative high-resolution imaging Orange maintains a high-and super resolution imaging facility in the Center for Human Immunobiology at Texas Children's Hospital.

The rest is here:
Gene therapy using lentivirus to treat Wiskott-Aldrich Syndrome promising

11 July 2013 Last updated at 14:04 ET By James Gallagher Health and science reporter, BBC News

A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors.

The study, in the journal Science, showed the three patients were now going to school.

A second study published at the same time has shown a similar therapy reversing a severe genetic disease affecting the immune system.

Gene therapy researchers said it was a "really exciting" development.

Both diseases are caused by errors in the patient's genetic code - the manual for building and running their bodies.

Babies born with metachromatic leukodystrophy appear healthy, but their development starts to reverse between the ages of one and two as part of their brain is destroyed.

Wiskott-Aldrich syndrome leads to a defective immune system. It makes patients more susceptible to infections, cancers and the immune system can also attack other parts of the body.

The technique, developed by a team of researchers at the San Raffaele Scientific Institute in Milan, Italy, used a genetically modified virus to correct the damaging mutations in a patient's genes.

The hype around gene therapy was huge - nipping into the genome and tweaking a bit of DNA was supposed to change medicine.

Originally posted here:
Gene therapy trial 'cures children'

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.

Follow this link:
6 children with rare disorders helped by gene therapy

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.

See more here:
Gene therapy using HIV helps children with fatal diseases, study says

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.

Read this article:
Stem-cell therapy wipes out HIV in two patients

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.

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

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.

View original post here:
Cell therapy: the fourth pillar of healthcare

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

Follow this link:
Stem Cell Therapy for Knee Pain: Safer than Surgery

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

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

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

See the article here:
BrightFocus Foundation announces $7.2 million in grants for Alzheimer's and vision disease research

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.

Read the rest here:
Gene Marks to Keynote at IPC Technology Market Research Conference

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.

Read the original post:
Scientists decode mystery sequences involved in gene regulation

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...

By: Adam Ford

See the original post:
Mini Documentary - The Hedonistic Imperative - David Pearce (Take 1) - Video