Category Archives: Gene therapy

Four researchers from UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received Early Translational Research Awards totaling approximately $13 million from the California Institute for Regenerative Medicine, the state's stem cell agency. The UCLA researchers received four of the 12 total awards; no other institution received more than one.
 
The Independent Citizens Oversight Committee, CIRM’s governing body, announced at its Aug. 28 meeting in La Jolla, Calif., that grant recipients included Dr. Jerome Zack, professor of medicine and microbiology, immunology and molecular genetics; Dr. Robert Reiter, Bing Professor of Urologic Research; Dr. Donald Kohn, professor of pediatrics and microbiology, immunology and molecular genetics in th...

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Scientists have come a step closer to being able to repair the damage done by heart attacks, using a cocktail of genes to transform scar tissue into working heart muscles.

Novel techniques to mend broken hearts using gene therapy and stem cells represent a major new frontier in the treatment of heart disease.

In the latest breakthrough, achieved by researchers at the Gladstone Institute of Cardiovascular Disease in California, researchers were able to re-programme scar-forming cells into heart muscle cells, some of which were capable of transmitting the kind of electrical signals that make the heart beat, according to the latest issue of the Stem Cell Reports journal.

The same team demonstrated their technique last year in live mice, transforming scar-forming cells, called fibroblasts, into beating heart muscle cells, but this is the first time that human fibroblasts have been re-programmed in this way.

So far, the work with human fibroblasts has only been done in the lab, but it paves the way for new treatments for heart attack victims. Researchers said that the cocktail of genes used to regenerate cells could one day be replaced with small drug-like molecules that would offer safer and easier delivery.

We've now laid a solid foundation for developing a way to reverse the damage [done by a heart attack] something previously thought impossible and changing the way that doctors may treat heart attacks in the future, said Dr Deepak Srivastava, director of cardiovascular disease at the Gladstone Institutes. Our findings here serve as a proof of concept that human fibroblasts can be re-programmed successfully into beating heart cells.

In 2012, Dr Srivastava and his team reported in the journal Nature that, by injecting three genes into the hearts of live mice that had been damaged by heart attack, fibroblasts could be turned into working heart cells.

The scientists attempted the same technique using human fibroblasts from foetal heart cells, embryonic stem cells and neonatal skin cells, injected with genes in petri dishes in the lab. An increased number of genes was required to transform the human cells, and the efficiency of the transformed cells was low, but the team were encouraged by the results.

While almost all the cells in our study exhibited at least a partial transformation, about 20 per cent of them were capable of transmitting electrical signals a key feature of beating hearts, said Gladstone staff scientist Ji-dong Fu, the studys lead author.

The number of people who survive heart attacks has increased considerably in recent decades. The British Heart Foundation (BHF) said earlier this year that 70 per cent of women and 68 per cent of men were now surviving. However, success in keeping people alive after a heart attack has led to a rise in the number of people suffering from the long-term after-effects, which include debilitating heart failure.

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Scientists use gene therapy to repair muscles damaged in heart attacks

Scientists have come a step closer to being able to repair the damage done by heart attacks, using a cocktail of genes to transform scar tissue into working heart muscles.

Novel techniques to mend broken hearts using gene therapy and stem cells represent a major new frontier in the treatment of heart disease.

In the latest breakthrough, achieved by researchers at the Gladstone Institute of Cardiovascular Disease in California, researchers were able to re-programme scar-forming cells into heart muscle cells, some of which were capable of transmitting the kind of electrical signals that make the heart beat, according to the latest issue of the Stem Cell Reports journal.

The same team demonstrated their technique last year in live mice, transforming scar-forming cells, called fibroblasts, into beating heart muscle cells, but this is the first time that human fibroblasts have been re-programmed in this way.

So far, the work with human fibroblasts has only been done in the lab, but it paves the way for new treatments for heart attack victims. Researchers said that the cocktail of genes used to regenerate cells could one day be replaced with small drug-like molecules that would offer safer and easier delivery.

We've now laid a solid foundation for developing a way to reverse the damage [done by a heart attack] something previously thought impossible and changing the way that doctors may treat heart attacks in the future, said Dr Deepak Srivastava, director of cardiovascular disease at the Gladstone Institutes. Our findings here serve as a proof of concept that human fibroblasts can be re-programmed successfully into beating heart cells.

In 2012, Dr Srivastava and his team reported in the journal Nature that, by injecting three genes into the hearts of live mice that had been damaged by heart attack, fibroblasts could be turned into working heart cells.

The scientists attempted the same technique using human fibroblasts from foetal heart cells, embryonic stem cells and neonatal skin cells, injected with genes in petri dishes in the lab. An increased number of genes was required to transform the human cells, and the efficiency of the transformed cells was low, but the team were encouraged by the results.

While almost all the cells in our study exhibited at least a partial transformation, about 20 per cent of them were capable of transmitting electrical signals a key feature of beating hearts, said Gladstone staff scientist Ji-dong Fu, the studys lead author.

The number of people who survive heart attacks has increased considerably in recent decades. The British Heart Foundation (BHF) said earlier this year that 70 per cent of women and 68 per cent of men were now surviving. However, success in keeping people alive after a heart attack has led to a rise in the number of people suffering from the long-term after-effects, which include debilitating heart failure.

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Scientists come closer to 'mending broken hearts' by using gene therapy to repair muscles damaged in heart attacks

Scientists have come a step closer to being able to repair the damage done by heart attacks, using a cocktail of genes to transform scar tissue into working heart muscles.

Novel techniques to mend broken hearts using gene therapy and stem cells represent a major new frontier in the treatment of heart disease.

In the latest breakthrough, achieved by researchers at the Gladstone Institute of Cardiovascular Disease in California, researchers were able to re-programme scar-forming cells into heart muscle cells, some of which were capable of transmitting the kind of electrical signals that make the heart beat, according to the latest issue of the Stem Cell Reports journal.

The same team demonstrated their technique last year in live mice, transforming scar-forming cells, called fibroblasts, into beating heart muscle cells, but this is the first time that human fibroblasts have been re-programmed in this way.

So far, the work with human fibroblasts has only been done in the lab, but it paves the way for new treatments for heart attack victims. Researchers said that the cocktail of genes used to regenerate cells could one day be replaced with small drug-like molecules that would offer safer and easier delivery.

We've now laid a solid foundation for developing a way to reverse the damage [done by a heart attack] something previously thought impossible and changing the way that doctors may treat heart attacks in the future, said Dr Deepak Srivastava, director of cardiovascular disease at the Gladstone Institutes. Our findings here serve as a proof of concept that human fibroblasts can be re-programmed successfully into beating heart cells.

In 2012, Dr Srivastava and his team reported in the journal Nature that, by injecting three genes into the hearts of live mice that had been damaged by heart attack, fibroblasts could be turned into working heart cells.

The scientists attempted the same technique using human fibroblasts from foetal heart cells, embryonic stem cells and neonatal skin cells, injected with genes in petri dishes in the lab. An increased number of genes was required to transform the human cells, and the efficiency of the transformed cells was low, but the team were encouraged by the results.

While almost all the cells in our study exhibited at least a partial transformation, about 20 per cent of them were capable of transmitting electrical signals a key feature of beating hearts, said Gladstone staff scientist Ji-dong Fu, the studys lead author.

The number of people who survive heart attacks has increased considerably in recent decades. The British Heart Foundation (BHF) said earlier this year that 70 per cent of women and 68 per cent of men were now surviving. However, success in keeping people alive after a heart attack has led to a rise in the number of people suffering from the long-term after-effects, which include debilitating heart failure.

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Scientists come closer to 'mending broken hearts' by using gene therapy to repair muscles damaged in heart attacks

Shortly after a baby’s first wail at birth she or he receives a tiny prick on the heel. A few drops of blood are caught on special filter paper to screen for myriad diseases. [More]

-- Read more on ScientificAmerican.com

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Earlier this year Yaniv Erlich of the Whitehead Institute for Biomedical Research at M.I.T. sent bioethicists into a frenzy when he and his team uncovered the names of people whose anonymous genome...

-- Read more on ScientificAmerican.com

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Stem Cell and Gene Therapy for CV Disease: Lessons learned and future directions
Les Miller, MD Director of the USF Heart Institute Professor of Cardiovascular Sciences at the USF Health Morsani College of Medicine Cardiology Grand Rounds...

By: DukeClinicalResearch

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Stem Cell and Gene Therapy for CV Disease: Lessons learned and future directions - Video

Earlier this year Yaniv Erlich of the Whitehead Institute for Biomedical Research at M.I.T. sent bioethicists into a frenzy when he and his team uncovered the names of people whose anonymous genome...

-- Read more on ScientificAmerican.com

Source:
http://rss.sciam.com/~r/sciam/topic/gene-therapy/~3/c3dvzwuFAUI/article.cfm

Macular Degeneration And Acupuncture: Gene Therapy and Stem Cell for AMD Safe?
http://www.MacularDegenerationSupport.com or (908) 264-5484 Download the FULL webinar for free by clicking the link. Dr. Andy Rosenfarb conducted an hour lon...

By: Dr. Andy Rosenfarb

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Macular Degeneration And Acupuncture: Gene Therapy and Stem Cell for AMD Safe? - Video

July 24, 2013 Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

The method was used to treat Sanfilippo -- a fatal inherited condition which causes progressive dementia in children -- but could also benefit several neurological, genetic diseases.

Researchers behind the study, published in the journal Molecular Therapy this month, are now hoping to bring a treatment to trial in patients within two years.

Sanfilippo, a currently untreatable mucopolysaccharide (MPS) disease, affects one in 89,000 children in the United Kingdom, with sufferers usually dying in their mid-twenties. It is caused by the lack of SGSH enzyme in the body which helps to breakdown and recycle long chain sugars, such as heparan sulphate (HS). Children with the condition build up and store excess HS throughout their body from birth which affects their brain and results in progressive dementia and hyperactivity, followed by losing the ability to walk and swallow.

Dr Brian Bigger, from the University of Manchester's Institute of Human Development who led the research, said bone marrow transplants had been used to correct similar HS storage diseases, such as Hurler syndrome, by transplanting normal cells with the missing enzyme but the technique did not work with Sanfilippo disease. This is because monocytes, a type of white blood cell, from the bone marrow, did not produce enough enzyme to correct the levels in the brain.

Dr Bigger said: "To increase SGSH enzyme from bone marrow transplants, and to target it to the cells that traffic into the brain, we have developed a stem cell gene therapy which overproduces the SGSH enzyme specifically in bone marrow white blood cells. "We have shown that mice treated by this method produce five times the normal SGSH enzyme levels in the bone marrow and 11 per cent of normal levels in the brain.

"The enzyme is taken up by affected brain cells and is enough to correct brain HS storage and neuro inflammation to near normal levels and completely corrects the hyperactive behaviour in mice with Sanfilippo.

"This is extremely exciting and could have huge implications for treatments. We now hope to work to a clinical trial in Manchester in 2015."

The University of Manchester team is now manufacturing a vector -- a tool commonly used by molecular biologists to deliver genetic material into cells -- for use in humans and hope to use this in a clinical trial with patients at Central Manchester University Hospital NHS Foundation Trust by 2015.

The stem cell gene therapy approach was recently shown by Italian scientists to improve conditions in patients with a similar genetic disease affecting the brain called metachromatic leukodystrophy, with results published in the journal Science earlier this month. Manchester scientists refined the vector used by the Italian scientists. "This approach has the potential to treat several neurological genetic diseases," Dr Bigger added.

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New stem cell gene therapy gives hope to prevent inherited neurological disease