For patients with choroideremia a rare form of progressive blindness there are no current treatment options that can help stop their visual degeneration. But now a new innovative procedure may be the key.

In a new study published in The Lancet, researchers used a novel gene therapy technique on choroideremia patients, which helped restore some of the sight they had already lost over the years. Gene therapy involves injecting patients with a vital gene that is either missing or defective in their genetic code.

Gene therapy is exciting; its a new type of medicine, lead author Robert MacLaren, a professor at the University of Oxford, told FoxNews.com. And what were doing is it on a very small scale, because were looking at a very straightforward gene to replace.

Caused by a mutation in the CHM gene on the X chromosome, choroideremia causes progressive blindness due to degeneration of the choroid, retinal pigment epithelium and retina. Patients with this disease can start their lives with perfect vision, but eventually start to experience problems with light sensitivity and peripheral vision as they age.

The condition, which affects 1 in every 50,000 people, ultimately leads to the death of the photoreceptor cells in the retina causing complete blindness in middle age.

Its like looking down through a telescope at a small central island of vision, MacLaren explained of the disorder. And by the time theyre in their 40s and 50s, they lose vision completely.

Because choroideremia is caused by a defect in a single gene, MacLaren believed that gene therapy could hold promise for patients with this form of progressive blindness. Additionally, because the cellular degeneration occurs so slowly, the researchers had a large window of opportunity in which they could test their treatment before complete visual loss occurred.

In order to fix the mutation found in choroideremia patients, MacLaren and his colleagues genetically altered an adeno-associated virus (AAV), so that it carried a corrective copy of the CHM gene.

The virus is a small biological organism, and its very good at getting into cells, MacLaren said. But rather than deliver the viruss DNA, weve taken out most of the viral DNA and instead put in the missing gene. So it releases the DNA into the nucleus its a single stranded DNA with the missing [CHM] gene.

The researchers injected their engineered virus into the retinas of six patients between the ages of 35 and 63, all of whom were experiencing different stages of choroideremia. Four of the patients still had good eyesight, though they had almost no peripheral vision, and the other two patients had already started to experience vision loss.

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Gene therapy trial shows promise in treating rare form of blindness

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Thursday 16 January 2014 11.33

Pioneering gene therapy has restored vision to two men with a rare inherited eye disease who were told to expect to go blind.

Scientists hope early intervention with the surgical treatment will halt progression of the devastating disorder, choroideremia, before patients are robbed of their sight.

It is the first time gene therapy has successfully been applied to the light-sensitive photoreceptors of the retina, the digital camera at the back of the eye.

Preliminary results from the first six patients taking part in a Phase One trial surprised and delighted the Oxford University team.

Although the trial was only designed to test safety and dosages, two men with relatively advanced disease experienced dramatic improvements to their eyesight.

The researchers are now planning a larger Phase II trial that will focus on the therapy's effectiveness.

Professor Robert MacLaren, who led the gene therapy operations at Oxford Eye Hospital, said: "We're absolutely delighted with the results so far.

"It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained for as long as we have been following up the patients, which is two years in one case.

"In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients' home opticians to get current and historical data on their vision in former years, long before the gene therapy trial started.

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Gene therapy breakthrough in blindness research

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By Thomas Moore, Health and Science Correspondent

Patients suffering from an inherited form of blindness have for the first time had their vision dramatically improved by gene therapy.

The first six patients to be given the experimental injections into the back of the eye were all able to see better in dim light.

And two of them were able to read more lines on an eye chart.

The patients were born with a genetic form of blindness called choroideremia, which affects 1 in 50,000 people, most of them boys, who start to lose their vision in late childhood.

But in a groundbreaking clinical trial, doctors at the Oxford Eye Hospital injected a harmless virus that had been engineered to carry a working copy of the gene that sufferers lack.

The trial was intended to confirm that the injections did not damage the delicate light-sensing cells in the retina.

But the gene therapy had an unexpected therapeutic benefit - and three more patients have now been treated with a higher dose.

Professor Robert MacLaren of Oxford University, who led the trial, said: "In truth we did not expect to see such dramatic improvements in visual acuity.

"It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained as long as we have been following up the patients, which is two years in one case."

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Gene Therapy Breakthrough Could 'Cure' Blindness

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Is this the Omics-to-Clinic Site We #39;ve All Been Waiting For? with Jonathan Hirsch, Syapse
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Walking at Project Walk Atlanta Spinal Cord injury Recovery
Joe walking with lift walker. T-level injury.

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BRUSSELS - Belgian medical researchers have succeeded in repairing bones using stem cells from fatty tissue, with a new technique they believe could become a benchmark for treating a range of bone disorders.

The team at the Saint Luc university clinic hospital in Brussels have treated 11 patients, eight of them children, with fractures or bone defects that their bodies could not repair, and a spin-off is seeking investors to commercialise the discovery.

Doctors have for years harvested stem cells from bone marrow at the top of the pelvis and injected them back into the body to repair bone.

The ground-breaking technique of Saint Luc's centre for tissue and cellular therapy is to remove a sugar cube sized piece of fatty tissue from the patient, a less invasive process than pushing a needle into the pelvis and with a stem cell concentration they say is some 500 times higher.

The stem cells are then isolated and used to grow bone in the laboratory. Unlike some technologies, they are also not attached to a solid and separate 'scaffold'.

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Stem cells from fatty tissue show potential for bone repair

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A piece of a three-dimensional bone structure obtained from the own adipose stem cells of a patient is seen at Brussels' Saint Luc Hospital January 14, 2014. Belgian medical researchers have succeeded in repairing bones using stem cells from fatty tissue, with a new technique they believe could become a benchmark for treating a range of bone disorders. REUTERS

BRUSSELS -- Belgian medical researchers have succeeded in repairing bones using stem cells from fatty tissue, with a new technique they believe could become a benchmark for treating a range of bone disorders.

The team at the Saint Luc university clinic hospital in Brussels have treated 11 patients, eight of them children, with fractures or bone defects that their bodies could not repair, and a spin-off is seeking investors to commercialize the discovery.

Doctors have for years harvested stem cells from bone marrow at the top of the pelvis and injected them back into the body to repair bone.

The ground-breaking technique of Saint Luc's centre for tissue and cellular therapy is to remove a sugar cube sized piece of fatty tissue from the patient, a less invasive process than pushing a needle into the pelvis and with a stem cell concentration they say is some 500 times higher.

The stem cells are then isolated and used to grow bone in the laboratory. Unlike some technologies, they are also not attached to a solid and separate 'scaffold'.

"Normally you transplant only cells and you cross your fingers that it functions," the centre's coordinator Denis Dufrane told Reuters television.

His work has been published in Biomaterials journal and was presented at an annual meeting of the International Federation for Adipose Therapeutics and Science (IFATS) in New York in November.

Belgian Professor Denis Defrane, coordinator of the centre of tissue and cellular therapy of Brussels' Saint Luc Hospital, shows how a hole in the tibia of a patient suffering from a disease was treated on an x-ray, in Belgium January 14, 2014.

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Belgian scientists repair bones with new stem cell technique

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Cofounder of Stem Cell Theranostics and StartX Med Divya Nag is attacking one of medicine's biggest problems: the fact that most types of human cellslike those in the heart or liverdie when you keep them in a petri dish. This makes testing new drugs a risky, costly and time-consuming business: 90% of medicines that start clinical trials turn out to be too unsafe or ineffective to market. But a new technology, the induced pluripotent stem cell, may help. Nag's company, Stem Cell Theranostics, was created from technology funded by a $20 million grant from the California Institute of Regenerative Medicine and is closing a venture round. It turns cellsusually from a piece of skininto embryonic-like stem cells, then uses them to create heart cells. These cells can live in petri dishes and be used to test new drugs. Someday they might even replace heart tissue that dies during a heart attack. Three large pharmaceutical companies are customers, though revenues are small. Nag, who was already publishing in prestigious scientific journals when she was an undergraduate, dropped out of Stanford to pursue her dream. No regrets: "Our technology was so promising and I was so passionate about it that nothing else made sense to me," she says. "It was very clear this was what I wanted to do."

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2014 30 Under 30: Science & Healthcare

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Goodie Mob - Cell Therapy (O.N.P. Instrumental) (1995) [HQ]
Producer: Organized Noize Productions Released: 1995 : LaFace Records Official Facebook Page: https://www.facebook.com/BeatJunKie82 --- Original Copyright O...

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By MARIA CHENG AP Medical Writer

LONDON (AP) - A small, preliminary study using gene therapy to treat a rare form of blindness is promising and could trigger similar efforts for other causes of vision loss, British doctors say.

They studied just six patients. Of those, two have had dramatic improvements in their vision and none has reported any serious side effects. The study was only designed to test the treatment's safety, not its effectiveness.

Gene therapy -inserting copies of a normal gene into a patient who has a faulty or missing gene - has previously been tried for other rare types of blindness with limited success.

All of the patients in the new trial were men with choroidermia, an inherited and untreatable form of progressive blindness. The disease is the result of a protein deficiency that kills eye cells that detect light.

"We're trying to rescue cells that would otherwise have died," said Dr. Robert MacLaren of the University of Oxford, who led the research. He and colleagues injected a harmless virus carrying the missing protein into the thinnest part of the patients' retinas. The study was paid for by the Wellcome Trust and Britain's Department of Health, and was published online Thursday in the journal, Lancet.

Dr. Ian MacDonald, chairman of ophthalmology and visual sciences at the University of Alberta in Canada, who was not part of the study but is planning a similar trial, called the new work "very promising because there is really no other way to deliver this protein."

Since the paper only had data up to six months after the patients' surgeries, MacDonald said it was unclear if the treatment would last forever or if the men's eyesight would continue to worsen.

Dr. Jean Bennett of the University of Pennsylvania, who conducted earlier work for a different eye disease, called the new study results exciting and said the same approach might be useful for other causes of blindness, including macular degeneration.

But she also said scientists should be cautious in trying future gene therapy treatments. "We can do as much work as we can in the laboratory and try to sort out all the variables, but there are always surprises," she said.

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Early gene therapy trial for blindness promising - Quincy Herald-Whig | Illinois & Missouri News, Sports

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Genetics How I am is How I am- E.V
Have you ever wondered why your ears are so big? Or why your nose is so long? Genetics is the answer, and when you find out how it all works, you can finally blame your dad for his ears.

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Genetics- Intro to Genetics

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Scrog; Bubba Kush genetics: plants under scrog for 17 days
Scrog @ 17 days.

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Scrog Bubba Kush genetics; plants 14 days old right before the scrog
Scrog Bubba Kush.

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Table One: Genetics and Heredity
For Mr. Kennedy #39;s class. Made by: Courtney Wright, Lindsey Blizzard, Laken Hatcher, D #39;Andre Johnson, and Drake Lanier.

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click image to zoom The future of dairy cattle breeding will be among the key topics as eight breeders from coast to coast voice their perspectives at the workshop Advancing Dairy Cattle Genetics: Genomics and Beyond in Tempe, Ariz., from February 17 to 19.

For more workshop details, go to http://www.ans.iastate.edu/events/dairygenomics/ to complete your registration.

In the meantime, here is more insight on the two breeder panels that will hone in on the future of cattle breeding:

Traits of the Future:

Dana Allen Tully, Gar-Lin Dairy Farms, Eyota, Minn. Dana manages an 1,855 cow dairy of which two-thirds of the cows are Holsteins and one-third are crossbred. A Ph.D. nutritionist by training, Dana places a strong focus on health traits such somatic cell score, daughter pregnancy rate, and productive life when making mating decisions. She also is a member of the Innovation Center for U.S. Dairys Cow of the Future Committee.

Alan Andersen, Seagull-Bay, American Falls, Idaho In recent years, the Andersens 600 cow Holstein herd has routinely placed bulls in the top 100 of the DNA or genomic-tested list. The most recognizable bull in that group is Seagull-Bay Supersire. Alan and his son, Greg, focus on high production and fitness traits. The family also has another 1,500 cow dairy at Declo, Idaho, managed by Alan's son Ben. This herd has utilized a 3-way crossbreeding system since 2005.

Alan Chittenden, Dutch Hollow Farm, Schodack Landing, N.Y. These Jersey breeders have been leaders in developing polled genetics that also rank high among all animals in the breed. Today, the herd consists of 600 Jersey cows. Their incredible track record of breeding top-notch cattle earned them the prestigious Master Breeder award from the American Jersey Cattle Association in 2012.

Jonathan Lamb, Oakfield Corners Dairy, Oakfield, N.Y. Lamb regularly uses embryo transplant and in-vitro fertilization on his 6,000 cow dairy to improve the genetics of his herds. Respected among his peers, Lamb serves as chairman of the genetic advancement committee for Holstein Association USA. Lamb was also named the Holstein USA Associations Distinguished Young Breeders in 2012.

On-farm Genomic Applications:

John Andersen, Double A Dairy, Jerome, Idaho Perhaps one of the largest users of genomic tests on a commercial scale, Andersen and his team routinely run genomic tests to sort out high-end and low-end genetic animals at their 13,000 cow dairy. Additionally, John stays involved of high-end genetics through breeding cattle under the Triple Crown Genetics prefix.

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Rethinking dairy genetics: Eight breeders to share needs in cows

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A small, preliminary study using gene therapy to treat a rare form of blindness is promising and could trigger similar efforts for other causes of vision loss, British doctors say.

They studied just six patients. Of those, two have had dramatic improvements in their vision and none has reported any serious side effects. The study was only designed to test the treatment's safety, not its effectiveness.

Gene therapy inserting copies of a normal gene into a patient who has a faulty or missing gene has previously been tried for other rare types of blindness with limited success.

All of the patients in the new trial were men with choroidermia, an inherited and untreatable form of progressive blindness. The disease is the result of a protein deficiency that kills eye cells that detect light.

"We're trying to rescue cells that would otherwise have died," said Dr. Robert MacLaren of the University of Oxford, who led the research. He and colleagues injected a harmless virus carrying the missing protein into the thinnest part of the patients' retinas. The study was paid for by the Wellcome Trust and Britain's Department of Health, and was published online Thursday in the journal, Lancet.

Dr. Ian MacDonald, chairman of ophthalmology and visual sciences at the University of Alberta in Canada, who was not part of the study but is planning a similar trial, called the new work "very promising because there is really no other way to deliver this protein."

Since the paper only had data up to six months after the patients' surgeries, MacDonald said it was unclear if the treatment would last forever or if the men's eyesight would continue to worsen.

Dr. Jean Bennett of the University of Pennsylvania, who conducted earlier work for a different eye disease, called the new study results exciting and said the same approach might be useful for other causes of blindness, including macular degeneration.

But she also said scientists should be cautious in trying future gene therapy treatments. "We can do as much work as we can in the laboratory and try to sort out all the variables, but there are always surprises," she said.

Toby Stroh, 56, had the gene therapy surgery nearly two years ago and credits it with allowing him to keep reading and playing tennis.

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Early Gene Therapy Trial for Blindness Promising

Recommendation and review posted by Bethany Smith

"In truth, we did not expect to see such dramatic improvements

This has huge implications for anyone with a genetic retinal disease such as age-related macular degeneration or retinitis pigmentosa because it has, for the first time, shown, that gene therapy can be applied safely before the onset of vision loss.

The trial was carried out on patients suffering from choroideremia a rare inherited cause of blindness which affects around 1 in 50,000 people.

It is caused by a defective gene which fails to produce REP-1 a protein needed to keep pigment cells in the retina healthy. Without it the cells slowly stop working, switch off and die.

The first symptom of the condition is usually poor night vision which can occur in early childhood. Later, the field of vision progressively narrows to a "tunnel" until only a small central slit remains.

The purpose of our trial is to put this missing protein back into the retinal cells and prevent further degeneration, said Prof MacLaren.

We're not talking about treatment that needs to be repeated we're talking about a single one off replacement of the gene.

What was unique and exciting is we noticed visual improvements very early, which shows us that it is working.

"If we were able to treat people early, get them in their teens or late childhood, we'd be getting the virus in before their vision is lost. If the treatment works, we would be able to prevent them from going blind."

The scientists found that the protein could be replaced in the eye by inserting it into the DNA of a harmless virus which can be injected into cells beneath the retina. As the virus infects these retinal cells the missing protein is restored.

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Gene therapy heralds cure for blindness

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3 hours ago

Oxford Eye Hospital

Dr. Robert MacLaren and colleagues at Britain's Oxford Eye Hospital perform a gene therapy experiment. Patients who took part say the experimental, approach improved their vision

An experimental therapy for a blinding eye disease showed early and surprising promise when it improved the vision of patients in an early trial that was only supposed to test its safety, doctors reported Wednesday.

The experimental gene therapy not only stopped the steady degeneration of the patients vision, but appears to have reversed some of the damage. And the effects have lasted two years in one case, British researchers report in the Lancet medical journal.

Wayne Thompson of Staffordshire in Britain saw the stars for the first time in years after being treated in April.

One night in the summer, my wife called me outside as it was a particularly starry evening. As I looked up, I was amazed that I was able to see a few stars, Thompson, 43, said in a statement.

I hadnt seen stars for a long, long time, he added.

It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained for as long as we have been following up the patients, which is two years in one case, says Dr. Robert MacLaren of the Nuffield Laboratory of Ophthalmology at the University of Oxford, who leads the research team.

In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients home opticians to get current and historical data on their vision in former years, long before the gene therapy trial started. These readings confirmed exactly what we had seen, he added in a statement.

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In surprise result,gene therapy reverses blinding eye disease

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2014 Medical innovation Summit
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