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

Posted: July 24, 2013 at 2:42 pm

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

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Oklahoma Veterinarian Offering Stem Cell Therapy For Aging Pets

Posted: July 24, 2013 at 2:42 pm

TULSA, Oklahoma -

A controversial therapy that's not available for humans is being used on their pets. Stem cells are helping dogs and cats heal faster and get rid of pain as the animals get older.

Mandy Clinton loves her dog and she'd do just about anything for her.

"I've had her for 14 years. She's my homegirl," Clinton said about Libbi.

But Libbi has come up with a hitch in her giddy up. She tripped while walking up the stairs and tore her ACL.

Clinton was looking into surgery when she learned about stem cell therapy and its ability to help dogs heal faster and reduce pain.

"I had no idea. I had no idea. It blew my mind, but I've had Libbi for 14 years, and so I was ready to do it if it was possible," she said.

Dr. Joe Landers at Heritage Veterinary Hospital will be performing Libbi's surgery on Wednesday.

They'll harvest stem cells from Libbi's stomach, process and stimulate them, then implant the stem cells on her leg joint.

"If you've ever cut your skin, deeply with a knife or something, you've had stem cell repair," said Dr. Joe Landers, DVM.

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UCLA Researcher Calls for Easing of Restrictions on Stem Cell Lines Derived from Eggs From Paid Providers

Posted: July 24, 2013 at 10:07 am

A UCLA researcher has spoken out in
support of a proposal to allow use of California stem cell agency
funds to purchase stem cell lines derived from eggs provided by women
who have been paid for the service.
Kathrin Plath, an associate professor, said in a letter to the agency that she and her colleagues would like to use a line from the Oregon SCNT
experiment by Shoukhrat Mitalipov in which human stem cells were cloned. Currently agency funds cannot be used for that purpose as
a result of regulations that are the extension of a state law that
bars use of agency funds for payment for eggs.
The agency's standards group meets later today to consider changing those regulations. The proposal will
then go before the full board tomorrow.
Plath, who has received $5 million from CIRM, said,

“In my lab, we are ... interested in
understanding what happens to the somatically silenced X chromosome
when differentiated cells are reprogrammed by SCNT. The key question
is: are these SCNT-ESCs more similar to iPSCs or
fertilization-derived ESCs with respect to the epigenetic state of
the X chromosome. Furthermore, it has been shown in mouse
reprogramming that the active X chromosome becomes deregulated during
SCNT-based reprogramming, and we would like to address this problem
in the human system as well.

“We believe that the comparison of
the epigenetic states between fertilization-derived ESCs, SCNT-ESCs
and human iPSCs is important for a better characterization of these
cells and understanding of their epigenetic nature.”

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USING STEM CELLS FROM YOUR OWN BODY – Video

Posted: July 23, 2013 at 2:41 pm


USING STEM CELLS FROM YOUR OWN BODY
USING STEM CELLS FROM YOUR OWN BODY.

By: Anthony Frederick

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USING STEM CELLS FROM YOUR OWN BODY - Video

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Speech 1010- Persuasive Speech- Stem Cell Research – Video

Posted: July 23, 2013 at 2:41 pm


Speech 1010- Persuasive Speech- Stem Cell Research
Angad SPeech.

By: MrAngaddeep

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Speech 1010- Persuasive Speech- Stem Cell Research - Video

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Stem cells turned into photoreceptors could treat vision loss

Posted: July 23, 2013 at 4:49 am

LONDON, July 22 (UPI) -- British scientist say embryonic stem cells turned into photoreceptors can integrate into a live retina, possibly promising new treatments for eye diseases.

Writing in the journal Nature Biotechnology, researchers from University College London report producing rod-like photoreceptors from embryonic stem cells and successfully transplanting them into the retinas of mice.

The result suggests embryonic stem cells may one day lead to treatment for eye diseases sush retinitis pigmentosa, macular degeneration or other degenerative conditions that can cause loss of vision.

The researchers say they've developed a new method for growing embryonic stem cells that allows them to become immature eye cells and self-organize into three-dimensional structures similar to those seen in a developing retina.

In laboratory tests, the structures were transplanted into the retinas of night-blind mice where they integrated with the natural cells of the eye and formed synaptic connections, the MIT Technology Review reported Monday.

While the technique is probably years away from human trials, embryonic stem cells are already being tested in clinical trials in Japan for macular degeneration.

Researchers there say an alternative source of stem cells, dubbed induced pluripotent stem cells, will soon move into human trials as a treatment for eye disease.

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Stem cells turned into photoreceptors could treat vision loss

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Magnetised carriers help steer stem cells to therapy sites

Posted: July 23, 2013 at 4:49 am

23 July 2013

Magnets could be a tool for directing stem cells to treat conditions such as heart or vascular disease.

By feeding stem cells particles made of magnetised iron oxide, scientists at Emory University and the Georgia Institute of Technology believe they can then use magnets to attract the cells to a particular location in the body after intravenous injection.

The results are published online in the journal Small and will appear in an upcoming issue. The paper was a result of collaboration between the laboratories of W. Robert Taylor of Emory, and Gang Bao of Georgia Tech. Taylor is professor of medicine and biomedical engineering and Bao is professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University.

The study used mesenchymal stem cells that can be obtained from adult tissues such as bone marrow or fat and are capable of becoming bone, fat and cartilage cells, but not other types of cell such as muscle or brain. They secrete a variety of nourishing and anti-inflammatory factors, which could make them valuable tools for treating conditions such as cardiovascular disease or autoimmune disorders.

Magnetized iron oxide nanoparticles are already US Food and Drug Administration (FDA) approved for diagnostic purposes with magnetic resonance imaging (MRI). Other scientists have tried to load stem cells with similar particles, but found that the coating on the particles was toxic or changed the cells properties.

The nanoparticles used in this study have a polyethylene glycol coating that protects the cell from damage. Another feature is that the Emory/Georgia Tech team used a magnetic field to push the particles into the cells, rather than chemical agents used previously.

We were able to load the cells with a lot of these nanoparticles and we showed clearly that the cells were not harmed, Taylor said in a statement. The coating is unique and thus there was no change in viability and perhaps even more importantly, we didnt see any change in the characteristics of the stem cells, such as their capacity to differentiate. This was essentially a proof of principle experiment. Ultimately, we would target these to a particular limb, an abnormal blood vessel or even the heart.

The particles are coated with the non-toxic polymer polyethylene glycol, and have an iron oxide core that is about 15 nanometres across. For comparison, a DNA molecule is two nanometres wide and a single influenza virus is at least 100 nanometres wide.

The particles appear to become stuck in cells lysosomes, which are parts of the cell that break down waste. The particles stay in one place for at least a week and leakage cannot be detected. The scientists measured the iron content in the cells once they were loaded up and determined that each cell absorbed roughly 1.5 million particles.

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Stem cells could help reverse some blindness

Posted: July 23, 2013 at 4:49 am

Scientists in Britain have taken a significant step towards the possibility of reversing certain forms of blindness using stem cells.

For the first time, researchers have successfully grown light sensitive mouse retina cells from embryonic stem cells in the lab and transplanted them into night-blind mice.

Following the transplantation, the cells appeared to develop normally, by integrating into the existing retina and forming nerve connections needed to transmit visual information to the brain.

The findings are published in the latest edition of Nature Biotechnology.

It is not yet clear how the technique would work in humans.

There are two types of photoreceptors in the eyes, known as rods and cones.

Rods are important for night vision.

Using a new technique involving 3D culture and differentiation of mouse stem cells recently developed in Japan, the team grew retinas containing all the different nerve cells needed for sight.

They then transplanted 200,000 of the artificially grown photoreceptor cells into retina of the night blind mice.

Three weeks later a much smaller number of the cells had integrated into the mouse retina and had begun looking like normal mature rod cells.

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Stem cells could help reverse some blindness

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Using Stem Cells To Treat Blindness

Posted: July 23, 2013 at 4:49 am

July 22, 2013

A new study published in the journal Nature Biotechnology represents a major step forward in curing certain types of blindness.

Described to the BBC News as a significant breakthrough, the UK-based study outlined a groundbreaking technique developed by doctors at Moorfields Eye Hospital and University College London that uses stem cell therapy to replace photoreceptors that may have died as a result of Stargardts disease or age-related macular degeneration.

While previous efforts have successfully focused on keeping the photoreceptors support cells alive, the new treatment aims to replace the retinas key cells directly. These cells sense light and are connected to other cells that relay the visual information to the brain.

The new technique expands on work done by Japanese researchers who used mice stem cells to fashion new retinas. The London team created photoreceptor cells and placed them in the eyes of blind mice.

After being injected, the new cells were able to connect with the mices existing framework albeit at a relatively low level of effectiveness. Approximately 1,000 of the 200,000 transplanted cells were able to connect with the rest of the eye.

This is a real proof of concept that photoreceptors can be transplanted from an embryonic stem cells source and it give us a route map to now do this in humans, said lead researcher Robin Ali, a professor of Human Molecular Genetics at the University College of London. Thats why were so excited, five years is now a realistic aim for starting a clinical trial.

The eye is one of the most promising candidates for stem cell therapy because there is only one type of intermediary cell between photoreceptors and the brain, unlike other organs which require far more connections between newly generated cells. A relatively small number tens of thousands of stem cells could improve vision, a number that would not make much of a difference in the liver or brain.

The eyes relatively weak local immune system also makes it an ideal candidate for stem cell therapy. A weak immune system means a lower chance of transplanted cells being rejected.

While experts described the study as a huge leap forward, some expressed concerns about the stem cells relatively low connection rate.

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Light-Detecting Retina Cells Made from Stem Cells

Posted: July 23, 2013 at 4:49 am

Scientists turn embryonic stem cells into photoreceptors that can integrate into a live retina.

Transplanted photoreceptors derived from embryonic stem cells (green) integrate into the damaged retina of an adult mouse and touch the next neuron in the retinal circuit (red).

Scientists in the U.K. have produced rod-like photoreceptors from embryonic stem cells, and successfully transplanted them into the retinas of mice. The work suggests that embryonic stem cells could perhaps one day be used as a treatment for patients who have lost their vision to retinitis pigmentosa, macular degeneration, or other degenerative conditions in which the light-detecting rods and cones of the retina die over time.

Currently, there are few treatment options for these conditions; electronic implanted devices are available for some patients in some countries, but their efficacy is limited (see A Second Artificial Retina Option for the E.U. and What Its Like to See Again with an Artificial Retina).The new work,reported in Nature Biotechnology on Sunday, offers hope for a more effective, comprehensive treatment.

The researchers used a new method for growing embryonic stem cells that enables them to turn into immature eye cells and self-organize into three-dimensional structures similar to those seen in a developing retina (see Growing Eyeballs). Immature light-detecting cells were harvested from this culture and transplanted into the retinas of night-blind mice. There, the cells integrated with the natural cells of the eye and formed synaptic connections. The work did not involve testing how well the mice could see after the cells were implanted.

While this particular technique is probably years away from human trials, embryonic stem cells are already being tested in clinical trials for macular degeneration and Stargardts macular dystrophy. Last week, in fact, Japanese authorities announced that an alternative source of stem cells will soon move into human trials as a treatment for eye disease.The BBC reported that Japan has approved the first clinical trial of induced pluripotent stem cells, or iPS cells. These stem cells are made by reprogramming normal adult cells so that they return to a more embryonic-like state so that they can then be converted into other cell types, such as retinal cells. In the clinical trial, doctors will collect a patients own cells, which will then be used in an experimental treatment for age-related macular degeneration. The trial will start with around six patients.

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Light-Detecting Retina Cells Made from Stem Cells

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