A pacemaker that may no longer be needed if the new gene therapy technique works. By Rachael Rettner, Senior Writer for LiveScience 2014-07-17 18:15:37 -0400

Electronic pacemakers can be lifesaving for people with abnormal or slow heart rhythms, but not everyone who needs a pacemaker is able to have an electronic device implanted in their heart.

Now, in experiments in pigs, researchers have come up with a new method for making a "biological pacemaker" that might one day serve as an alternative to electronic ones, the researchers said.

Making this pacemaker involves injecting a gene into heart muscle cells, which transforms these normal heart cells into special cells that can initiate a heartbeat.

This method could be useful for certain patients, such as those who develop infections from electronic pacemakers and need to have the devices temporarily removed, or fetuses with life-threatening heart disorders who cannot have an electronic pacemaker implanted, the researchers said.

"Babies still in the womb cannot have a pacemaker," study researcher Dr. Eugenio Cingolani, director of the Cardiogenetics-Familial Arrhythmia Clinic at Cedars-Sinai Heart Institute in Los Angeles, said in a statement. "It is possible that one day, we might be able to save lives by replacing [electronic] hardware with an injection of genes."

The researchers previously showed that this method worked in rodents, but pig hearts are similar to human hearts in their size and the way that they work, so there's reason to think the new findings could translate to humans. Still, more research is needed before the method could be tested in people to better understand the treatment's safety and effectiveness, the researchers said. The method relies on a virus to insert the gene into the heart cells, and although this virus cannot replicate itself or integrate into the genome, the pig experiments showed that a small amount of virus did end up in other organs in the animals besides the heart, according to the study published July 16 in the journal Science Translational Medicine.

In healthy people, a small region of the heart, called the sinoatrial node, fires the electrical impulses that determine heart rate. If this region is not working properly, people can develop heart rhythm problems, and have symptoms such as fatigue, fainting or even cardiac arrest. Such patients may have electronic pacemakers put in to monitor the heart rhythm, which sends electrical pulses to keep the heart beating normally.

In the study, the researchers used pigs with a condition called complete heart block, in which the heart beats very slowly. The researchers injected a gene called TBX18 into a small area of the heart muscle. This gene converted this area of heart muscle cells into sinoatrial node cells.

"In essence, we create a new sinoatrial node in a part of the heart that ordinarily spreads the impulse, but does not originate it," study researcher Dr. Eduardo Marbn, director of the Cedars-Sinai Heart Institute, said in news conference about the findings. "The newly created node then takes over as a functional pacemaker, bypassing the need for implanted electronics and hardware."

Read more here:
Gene Therapy in Pigs Creates Temporary 'Biological Pacemaker'

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WASHINGTON --

The study, published Wednesday, is one step toward developing an alternative to electronic pacemakers that are implanted into 300,000 Americans a year.

"There are people who desperately need a pacemaker but can't get one safely," said Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, who led the work. "This development heralds a new era of gene therapy" that one day might offer them an option.

Your heartbeat depends on a natural pacemaker, a small cluster of cells - it's about the size of a peppercorn, Marban says - that generates electrical activity. Called the sinoatrial node, it acts like a metronome to keep the heart pulsing at 60 to 100 beats a minute or so, more when you're active. If that node quits working correctly, hooking the heart to an electronic pacemaker works very well for most people.

But about 2 percent of recipients develop an infection that requires the pacemaker to be removed for weeks until antibiotics wipe out the germs, Marban said. And some fetuses are at risk of stillbirth when their heartbeat falters, a condition called congenital heart block.

For over a decade, teams of researchers have worked to create a biological alternative that might help those kinds of patients, trying such approaches as using stem cells to spur the growth of a new sinoatrial node.

Marban's newest attempt uses gene therapy to reprogram a small number of existing heart muscle cells so that they start looking and acting like natural pacemaker cells instead.

Because pigs' hearts are so similar to human hearts, Marban's team studied the approach in 12 laboratory pigs with a defective heart rhythm.

They used a gene named TBX18 that plays a role in the embryonic development of the sinoatrial node. Working through a vein, they injected the gene into some of the pigs' hearts - in a spot that doesn't normally initiate heartbeats - and tracked them for two weeks.

Two days later, treated pigs had faster heartbeats than control pigs who didn't receive the gene, the researchers reported in the journal Science Translational Medicine. That heart rate automatically fluctuated, faster during the day. The treated animals also became more active, without signs of side effects.

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Scientists using gene therapy to create biological pacemaker

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........................................................................................................................................................................................

WASHINGTON No batteries required: Scientists are creating a biological pacemaker by injecting a gene into the hearts of sick pigs that changed ordinary cardiac cells into a special kind that induces a steady heartbeat.

The study, published Wednesday, is one step toward developing an alternative to electronic pacemakers that are implanted into 300,000 Americans a year.

There are people who desperately need a pacemaker but cant get one safely, said Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, who led the work. This development heralds a new era of gene therapy that one day might offer them an option.

Your heartbeat depends on a natural pacemaker, a small cluster of cells its about the size of a peppercorn, Marban says that generates electrical activity. Called the sinoatrial node, it acts like a metronome to keep the heart pulsing at 60 to 100 beats a minute or so, more when youre active. If that node quits working correctly, hooking the heart to an electronic pacemaker works very well for most people.

But about 2 percent of recipients develop an infection that requires the pacemaker to be removed for weeks until antibiotics wipe out the germs, Marban said. And some fetuses are at risk of stillbirth when their heartbeat falters, a condition called congenital heart block.

For over a decade, teams of researchers have worked to create a biological alternative that might help those kinds of patients, trying such approaches as using stem cells to spur the growth of a new sinoatrial node.

Marbans newest attempt uses gene therapy to reprogram a small number of existing heart muscle cells so that they start looking and acting like natural pacemaker cells instead.

Because pigs hearts are so similar to human hearts, Marbans team studied the approach in 12 laboratory pigs with a defective heart rhythm.

They used a gene named TBX18 that plays a role in the embryonic development of the sinoatrial node. Working through a vein, they injected the gene into some of the pigs hearts in a spot that doesnt normally initiate heartbeats and tracked them for two weeks.

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Scientists creating a biological pacemaker

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More than 100 people from Tayside have signed up for the bone marrow register since the Tele published the story of tragic tot Faith Cushnie.

The nine-month-old from Menzieshill needed a bone marrow donation to beat leukaemia, but the donor backed out and doctors have told Faiths parents that there is now nothing they can do for her.

But 109 of you were so touched by Faiths story you immediately registered to be donors at the bone marrow and stem cell charity Anthony Nolan.

Over the same period last year the charity did not have a single registration from Tayside.

Incredibly, Dundee is currently sending the second highest number of visitors to the charitys website, after London, with 658 sessions on Tuesday and Wednesday.

Charities like Anthony Nolan typically struggle for donors, in comparison to campaigns like Give Blood.

Blood was donated in Tayside 21,000 times in the last year but only 4,000 people in the region are on the list of bone marrow donors.

Thats despite an average of around 600 people being diagnosed with leukaemia in Scotland during that time.

Dr David Meiklejohn, a consultant in the department of haematology in Ninewells Hospital, said nearly all donors were volunteers.

He said: Its important to raise awareness as we cant get donors otherwise.

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Tele readers rush to save lives: Faith Cushnies plight highlights importance of bone marrow donors

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PUBLIC RELEASE DATE:

15-Jul-2014

Contact: Jenny Eriksen Leary jenny.eriksen@bmc.org 617-638-6841 Boston University Medical Center

Using experimental models, researchers at Boston University School of Medicine (BUSM) showed that adenosine, a metabolite released when the body is under stress or during an inflammatory response, stops the process of adipogenesis, when adipose (fat) stem cells differentiate into adult fat cells.

Previous studies have indicated adipogenesis plays a central role in maintaining healthy fat homeostasis by properly storing fat within cells so that it does not accumulate at high levels in the bloodstream. The current findings indicate that the body's response to stress, potentially stopping the production of fat cell development, might be doing more harm than good under conditions of obesity and/or high levels of circulating blood fat.

The process is halted due to a newly identified signaling from an adenosine receptor, the A2b adenosine receptor (A2bAR) to a stem cell factor, known as KLF4, which regulates stem cell maintenance. When A2bAR is expressed, KLF4 level is augmented, leading to inhibition of differentiation of fat stem cells. The correlation between these two factors leads to an interruption of fat cell development, which could result in issues with fat storage within the cells and it getting into the bloodstream.

While the majority of the study was carried out in experimental models, the group also showed that A2bAR activation inhibits adipogenesis in a human primary preadipocyte culture system. Finally, analysis of adipose tissue of obese subjects showed a strong association between A2bAR and KLF4 expression in both subcutaneous (under the skin) and visceral (internal organ) human fat.

"It may seem counterintuitive, but our body needs fat tissue in order to function properly, and certain biochemical cellular processes are necessary for this to happen," said Katya Ravid, DSc/PhD, professor of medicine and biochemistry at BUSM and director of the Evans Center for Interdisciplinary Biomedical Research who led the study. "Our study indicates that a dysfunction resulting from stress or inflammation can disrupt the process of fat tissue development, which could have a negative impact on processes dependent on proper fat cell homeostasis."

This study is part of ongoing research interest and investigations by researchers in Ravid's lab examining the differentiation of bone marrow and tissue stem cells and the role of adenosine receptors in this process.

###

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BUSM study: Obesity may be impacted by stress

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DR ANDREW J ROCHMAN: ON STEM CELL THERAPY

By: Len Promoter

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DR ANDREW J ROCHMAN: ON STEM CELL THERAPY - Video

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PCP: Stem cell therapy untested, expensive, and experimental

MANILA - The Lung Center of the Philippines (LCP) defended the P70 million it received as funding from the Disbursement Acceleration Program for a stem cell research project.

LCP Executive Director Jose Luis J. Danguilan admitted receiving P105 million in total allocations from the DAP, which he said was spent wisely. He said P35 million was spent for the LCP's pediatric unit while the rest went to stem cell research.

The stem cell research program was listed as the 15th item in the Department of Budget and Management list. It was named as the LCPs Bio-Regenerative Technology Program.

The project intends to "fund the Bio-Regenerative Program aimed at harnessing stem cell research and technology to reconstruct new healthy cells, replacing cancer or dead cells."

Danguilan said the equipment bought for both the stem cell and pediatric unit programs have other uses.

"To spend the money wisely, it was decided that pieces of equipment needed for the Bioregenerative Program and the Pediatric Unit could also be used by the Department of Pathology and Laboratory, the Department of Thoracic Surgery and Surgery, the Department of Pulmonary Medicine and the Department of Radiology," he said.

He also noted the money was used to purchase equipment and supplies for the LCP Molecular Diagnostics and Cellular Therapeutics Laboratory for research "mainly on dendritic cell vaccine for use in cancer treatment, specifically lung cancer."

"To place things in perspective, the LCP is a tertiary hospital for pulmonary and other chest diseases, and as far as I know, it is the only tertiary pulmonary specialty hospital in the Philippines. As such, it should take the lead, not only in cutting edge treatment but also in advocacies," he added.

On Tuesday, members of the Philippine College of Physicians (PCP) said the priorities of the government when it comes to funding projects under the DAP seem to be misplaced.

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Lung Center defends DAP-funded stem cell program

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Let #39;s Play The Sims 3 - Perfect Genetics Challenge: Cowgirl and Horse Edition Episode 18
Come join me on my latest journey into the complex world of sims 3 genetics, as I try to get perfect foals and perfect children. Will I succeed in getting pe...

By: GamerGirlsNetwork

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Let's Play The Sims 3 - Perfect Genetics Challenge: Cowgirl and Horse Edition Episode 18 - Video

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Genetics of Regeneration describes the Believer #39;s Adoption in Messiah..
Dormant D.N.A. becomes active when a person puts his or her trust in Messiah because each is made alive by the Holy Spirit. Paul said that the Almighty is in everything. He is. But not in a...

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Let #39;s Play The Sims 3 - Perfect Genetics Challenge: Cowgirl and Horse Edition Episode 19
Come join me on my latest journey into the complex world of sims 3 genetics, as I try to get perfect foals and perfect children. Will I succeed in getting pe...

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Let's Play The Sims 3 - Perfect Genetics Challenge: Cowgirl and Horse Edition Episode 19 - Video

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Let #39;s Play The Sims 3 - Perfect Genetics Challenge: Cowgirl and Horse Edition Episode 20
Come join me on my latest journey into the complex world of sims 3 genetics, as I try to get perfect foals and perfect children. Will I succeed in getting pe...

By: GamerGirlsNetwork

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Let's Play The Sims 3 - Perfect Genetics Challenge: Cowgirl and Horse Edition Episode 20 - Video

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Let #39;s Play: Sims 3 Perfect Genetics Challenge - Part 13
More additions to the household... let #39;s hope for a perfect genetic match! Thank you so much for watching! I greatly appreciate it! Please comment, like and ...

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The Sims 3 | Perfect Genetics Challenge Part 13: Teenager!
In this part, we give birth to our next baby and Ashley ages up. Backstory: "Once upon a time, the Mighty Player sent a Sim to live in the world where all its creations were living happily....

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The Sims 3 | Perfect Genetics Challenge Part 13: Teenager! - Video

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Washington No batteries required: Scientists are creating a biological pacemaker by injecting a gene into the hearts of sick pigs that changed ordinary cardiac cells into a special kind that induces a steady heartbeat.

The study, published Wednesday, is one step toward developing an alternative to electronic pacemakers that are implanted into 300,000 Americans a year.

There are people who desperately need a pacemaker but cant get one safely, said Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, who led the work. This development heralds a new era of gene therapy that one day might offer them an option.

Your heartbeat depends on a natural pacemaker, a small cluster of cells its about the size of a peppercorn, Marban says that generates electrical activity. Called the sinoatrial node, it acts like a metronome to keep the heart pulsing at 60 to 100 beats a minute or so, more when youre active. If that node quits working correctly, hooking the heart to an electronic pacemaker works very well for most people.

But about 2 percent of recipients develop an infection that requires the pacemaker to be removed for weeks until antibiotics wipe out the germs, Marban said. And some fetuses are at risk of stillbirth when their heartbeat falters, a condition called congenital heart block.

For over a decade, teams of researchers have worked to create a biological alternative that might help those kinds of patients, trying such approaches as using stem cells to spur the growth of a new sinoatrial node.

Marbans newest attempt uses gene therapy to reprogram a small number of existing heart muscle cells so that they start looking and acting like natural pacemaker cells instead.

Because pigs hearts are so similar to human hearts, Marbans team studied the approach in 12 laboratory pigs with a defective heart rhythm.

They used a gene named TBX18 that plays a role in the embryonic development of the sinoatrial node. Working through a vein, they injected the gene into some of the pigs hearts in a spot that doesnt normally initiate heartbeats and tracked them for two weeks.

Two days later, treated pigs had faster heartbeats than control pigs who didnt receive the gene, the researchers reported in the journal Science Translational Medicine. That heart rate automatically fluctuated, faster during the day. The treated animals also became more active, without signs of side effects.

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Scientists use gene therapy to create biological pacemaker

Recommendation and review posted by Bethany Smith

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Newswise Researchers at the National Institutes of Health have developed a technique that will speed up the production of stem-cell derived tissues. The method simultaneously measures the expression of multiple genes, allowing scientists to quickly characterize cells according to their function and stage of development. The technique will help the researchers in their efforts to use patients skin cells to regenerate retinal pigment epithelium (RPE)a tissue in the back of the eye that is affected in several blinding eye diseases. It will also help the scientists search for drugs for personalized treatments.

Progress in stem cell-based therapies has been limited by our capacity to authenticate cells and tissues, said Kapil Bharti, Ph.D., a Stadtman Investigator in the Unit on Ocular and Stem Cell Translational Research at the National Eye Institute (NEI), a part of NIH. This assay expands that capacity and streamlines the process.

The assay was described in a recent issue of Stem Cells Translational Medicine.

The RPE is a single layer of cells that lies adjacent to the retina, where the light-sensitive photoreceptors commonly called rods and cones are located. The RPE supports photoreceptor function. Several diseases cause the RPE to break down, which in turn leads to the loss of photoreceptors and vision.

The stem cells Dr. Bharti is using to make RPE are induced pluripotent (iPS) stem cells, which are produced by reverting mature cells to an immature state, akin to embryonic stem cells. iPS cells can be derived from a patients skin or blood cells, coaxed into other cell types (such as neurons or muscle), and in theory, re-implanted without causing immune rejection.

To verify the identity of RPE made from iPS cells, scientists use microscopy to ensure the tissue looks like RPE and physiological assays to ensure the tissue behaves like RPE. They also use a technique called quantitative RT-PCR to measure the expression of genes that indicate ongoing cell development and function. For example, expression of the gene SOX2 is much higher in iPS cells than mature RPE.

But quantitative RT-PCR only permits the simultaneous measurement of a few genes per sample. Dr. Bharti teamed up with Marc Ferrer, Ph.D., of NIHs National Center for Advancing Translational Sciences (NCATS) to develop a multiplex assaya method for simultaneously measuring multiple genes per RPE sample in a highly automated fashion. The assay is based on a commercially available platform from the biotech company Affymetrix. In the assay, tiny snippets of DNA tethered to beads are used to capture RNA moleculescreated when genes are expressed by cells in the RPE sample. Once captured, the RNA from distinct genes is labeled with a fluorescent tag.

Starting with cells from a skin biopsy, the researchers generated iPS-derived RPE and then measured the expression of eight genes that are markers of development, function, and disease. They measured RNA levels of each gene one at a time using quantitative RT-PCR and then all genes simultaneously using the multiplex assay. When compared, the results correlated.

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Gene Profiling Technique to Accelerate Stem Cell Therapies for Eye Diseases

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Spinal Cord Injury Variations
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Basic Home Process
A brief discussion on the first steps towards the application process.

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By LAURAN NEERGAARD AP Medical Writer

WASHINGTON (AP) - No batteries required: Scientists are creating a biological pacemaker by injecting a gene into the hearts of sick pigs that changed ordinary cardiac cells into a special kind that induces a steady heartbeat.

The study, published Wednesday, is one step toward developing an alternative to electronic pacemakers that are implanted into 300,000 Americans a year.

"There are people who desperately need a pacemaker but can't get one safely," said Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, who led the work. "This development heralds a new era of gene therapy" that one day might offer them an option.

Your heartbeat depends on a natural pacemaker, a small cluster of cells - it's about the size of a peppercorn, Marban says - that generates electrical activity. Called the sinoatrial node, it acts like a metronome to keep the heart pulsing at 60 to 100 beats a minute or so, more when you're active. If that node quits working correctly, hooking the heart to an electronic pacemaker works very well for most people.

But about 2 percent of recipients develop an infection that requires the pacemaker to be removed for weeks until antibiotics wipe out the germs, Marban said. And some fetuses are at risk of stillbirth when their heartbeat falters, a condition called congenital heart block.

For over a decade, teams of researchers have worked to create a biological alternative that might help those kinds of patients, trying such approaches as using stem cells to spur the growth of a new sinoatrial node.

Marban's newest attempt uses gene therapy to reprogram a small number of existing heart muscle cells so that they start looking and acting like natural pacemaker cells instead.

Because pigs' hearts are so similar to human hearts, Marban's team studied the approach in 12 laboratory pigs with a defective heart rhythm.

They used a gene named TBX18 that plays a role in the embryonic development of the sinoatrial node. Working through a vein, they injected the gene into some of the pigs' hearts - in a spot that doesn't normally initiate heartbeats - and tracked them for two weeks.

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Trying gene therapy to create biological pacemaker - Quincy Herald-Whig | Illinois & Missouri News, Sports

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PUBLIC RELEASE DATE:

15-Jul-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

The brain has a low renewable capacity for self-repair and generation of new functional neurons in the treatment of trauma, inflammation and cerebral diseases. Cytotherapy is one option to regenerate central nervous system that aim at replacing the functional depleted cells due to traumatic brain injury (TBI). Bone marrow mesenchymal stem cells (BMSCs) are also considered a candidate for cytotherapy because they can differentiate into neurons/nerve cells, pass across blood-brain barrier, migrate into the injured region, secrete neurotrophic factor, and provide microenvironment for neural regeneration. Prof. Mohammad Ali Khalili, Research and Clinical Center for Infertility, Shahid Sadoughi University of Medical Sciences, Iran and his team administered TBI rats 3106 BMSCs via the tail vein and found that the BMSCs transplanted via the tail vein promoted nerve cell regeneration in injured cerebral cortex, which supplement the lost nerve cells. Related results were published in Neural Regeneration Research (Vol. 9, No. 9, 2014).

Article: " Intravenous transplantation of bone marrow mesenchymal stem cells promotes neural regeneration after traumatic brain injury" by Fatemeh Anbari1, Mohammad Ali Khalili1, Ahmad Reza Bahrami2, Arezoo Khoradmehr1, Fatemeh Sadeghian1, Farzaneh Fesahat1, Ali Nabi1 (1 Research and Clinical Center for Infertility, Shahid Sadoughi University of Medical Sciences, Yazd, Iran; 2 Institute of Biotechnology, Ferdowsi University of Mashhad, Mashhad, Iran)

Anbari F, Khalili MA, Bahrami AR, Khoradmehr A, Sadeghian F, Fesahat F, Nabi A. Intravenous transplantation of bone marrow mesenchymal stem cells promotes neural regeneration after traumatic brain injury. Neural Regen Res. 2014;9(9):919-923.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

###

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

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Does intravenous transplantation of BMSCs promote neural regeneration after TBI?

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Nine-month-old Faith Cushnie was born with a rare form of leukaemia.

Only a bone marrow transplant could save her life.

And mum and dad Amber Cushnie and Ryan Skelly were over the moon when Faith went into remission and a donor came forward.

Then, the unthinkable: The anonymous donor backed out and Faiths cancer relapsed.

Now doctors have said there is nothing more they can do for Menzieshill tot Faith.

Amber and Ryan hope their daughters tragic case can help drive home the importance of bone marrow donations.

There is currently a shortage of bone marrow donors in the UK.

As a result, Faiths family had to rely on just one individual to save their baby daughter.

Amber, 25, said: People give blood, but theres so little awareness of bone marrow donation. Its really simple to register.

If the transplant had taken place obviously theres no knowing what the situation would have been, but we wouldnt be in this position.

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Tragedy of Dundee tot Faith as bone marrow donor hopes dashed

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Scientists have corrected an irregular heart beat in pigs using gene therapy, a finding they say may one day offer a replacement for the electronic pacemakers now used in humans.

In the study, a gene known as TBX-18 that expresses a protein normally involved in regulating heart rhythm, was injected into the hearts of seven pigs who carried a medical condition that made their heart beat too slowly or irregularly. The inserted DNA reprogrammed cells in the animals hearts in a way that corrected the beat, according to the report published in the journal Science Translational Method.

The findings, which wore off after about 10 days, offer promise for creating bridge pacemakers for those who get infections or fetuses with congenital heart block, where early surgery isnt an option, said Eduardo Marbn, a study author.

Biology takes over and creates a functioning pacemaker, said Marbn, who is also director of the Cedars-Sinai Heart Institute in Los Angeles, in a telephone interview. Its one of the penultimate steps to getting this to clinical trials in humans.

About 300,000 Americans get electronic pacemakers yearly, according to the research. The devices send electrical shocks that shock the heart to restore regular rhythm.

About 2 percent of people with pacemakers get infections during the process and need to get the devices removed, according to Marbn. The gene was injected using a catheter, and researchers are continuing to study whether the procedure can have longer-lasting results, he said.

Human trials are at least 2 to 3 years away, Marban said.

The research offers an intriguing proof of concept that should be pursued, said Nikhil Munshi, a cardiovascular researcher at the University of Texas Southwestern Medical Center in Dallas, in a telephone interview.

Its exciting, said Munshi, who wrote an accompanying commentary in the journal. Its a step in the right direction toward developing a biological pacemaker to complement existing pacemakers.

He identified other methods being studied that included the use of stem cells that help rebuild the hearts ability to operate normally, and manipulating currents in the hearts ion channels, pores within the heart that open or close in response to chemical signals, creating electrical charges.

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Biological Pacemaker That Works in Pigs Offers Promise

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PUBLIC RELEASE DATE:

16-Jul-2014

Contact: Trish Reynolds reynoldsp2@mail.nih.gov 301-496-8190 NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases

Investigators have identified a gene that underlies a very rare but devastating autoinflammatory condition in children. Several existing drugs have shown therapeutic potential in laboratory studies, and one is currently being studied in children with the disease, which the researchers named STING-associated vasculopathy with onset in infancy (SAVI). The findings appeared online today in the New England Journal of Medicine. The research was done at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the National Institutes of Health.

"Not only do these discoveries have profound implications for children with SAVI, but they could have a broader impact by helping us to understand other, more common inflammatory conditions," said NIAMS Director Stephen I. Katz, M.D., Ph.D. "Diseases such as lupus share some characteristics with SAVI, so this work may lead to novel insights and possibly new treatments for these debilitating conditions, as well."

The senior author of the study, Raphaela Goldbach-Mansky, M.D., and the co-lead authors, Yin Liu, M.D., Ph.D., Adriana A. Jesus, M.D., Ph.D., and Bernadette Marrero, Ph.D., are in the NIAMS Translational Autoinflammatory Disease Section.

Autoinflammatory diseases are a class of conditions in which the immune system, seemingly unprovoked, becomes activated and triggers inflammation. Normally, the inflammatory response helps quell infections, but the prolonged inflammation that occurs in these diseases can damage the body.

In 2004, Dr. Goldbach-Mansky was called upon to advise on a patient with a baffling problema 10-year-old girl with signs of systemic inflammation, especially in the blood vessels, who had not responded to any of the medications her doctors had used to treat her.

She had blistering rashes on her fingers, toes, ears, nose and cheeks, and had lost parts of her fingers to the disease. The child also had severe scarring in her lungs and was having trouble breathing. She had shown signs of the disease as an infant and had progressively worsened. She died a few years later.

By 2010, Dr. Goldbach-Mansky had seen two other patients with the same symptoms. She suspected that all three had the same disease, and that it was caused by a genetic defect that arose in the children themselves, rather than having been inherited from their parents, who were not affected. Her hunch suggested a strategy for identifying the genetic defect. By comparing the DNA of an affected child with the DNA of the child's parents, scientists would be able to spot the differences and possibly identify the disease-causing mutation.

Continued here:
NIH scientists identify gene linked to fatal inflammatory disease in children

Recommendation and review posted by Bethany Smith

PUBLIC RELEASE DATE:

16-Jul-2014

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

New Rochelle, NY, July 16, 2014Experimental drugs proven safe but perhaps not sufficiently effective in initial testing or against a first disease target may sit gathering dust on the shelves of pharmaceutical companies. An NIH-sponsored effort based on a crowdsourcing strategy to establish collaborations between industrial and academic partners to test and develop these therapeutic compounds was met with an overwhelming response and has led to clinical testing of a broad range of pilot projects and a newly announced round of funding opportunities. These findings are described in a Review article in the preview issue of the new journal Drug Repurposing, Rescue, and Repositioning, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. The article is available free on the Drug Repurposing, Rescue, and Repositioning website.

Christine M. Colvis, PhD and Christopher P. Austin, MD, National Center for Advancing Translational Sciences, National Institutes of Health (NIH), Bethesda, MD, explain that the Center does not focus on a particular disease or organ system, allowing it to support a broad scope of projects that link indications with unmet medical needs to the mechanisms of action of new drug compounds that are ready to move into patient testing. In the article "The NIH-Industry New Therapeutic Uses Pilot Program: Demonstrating the Power of Crowdsourcing") the authors state that among the new funding opportunity announcements released by the Center in May were 12 therapeutic agents for pediatric indication consideration.

"This article describes not only how targeted crowdsourcing can link up the assets, the know-how, and the creativity that drug repurposing needs, but also how such a program can be organized to serve the best interests of all concerned parties," says journal Editor Hermann Mucke, PhD, H.M. Pharma Consultancy, Vienna, Austria. "Pharmaceutical companies and academia must collaborate to leverage their huge potential synergies in compound re-development, and by arranging and mentoring this pilot program NCATS has firmly established its role as a mediator in drug repurposing."

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About the Journal

Drug Repurposing, Rescue, and Repositioning, a dynamic new peer-reviewed journal, presents techniques and tools for finding new uses for approved drugs particularly for disorders where no animal model, physiologic abnormality, biochemical pathway, or molecular target has been identified. Led by Editor-in-Chief Aris Persidis, Biovista, Inc., and Editor Hermann Mucke, H.M. Pharma Consultancy e.U, the Journal provides a new interdisciplinary platform for scientific contributions to the field of drug repurposing including original research papers, reviews, case studies, application-oriented technology assessments, and reports in methodology and technology application. The Journal is published quarterly online with Open Access options and in print. A sample issue may be viewed on the Drug Repurposing, Rescue, and Repositioning website

About the Publisher

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NIH turns to crowdsourcing to repurpose drugs

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