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Posts Tagged ‘Therapy’


U.S. stem cell experts have produced a library of the powerful cells using ordinary skin and bone marrow cells from patients, and will share them freely with other researchers. 

How scientists can derive stem cells from ordinary skin cells.

How scientists can derive stem cells from ordinary skin cells.

They used a new method to genetically re-program ordinary cells in order to revert them back into embryonic stem cells — the master cells of the body with the ability to produce any type of tissue or blood cell.  The new cells are called induced pluripotent stem cells, or iPS cells, and are made using four genes that reprogram an ordinary adult cell into a primitive stage resembling the first days of a human embryo — a method pioneered by Shinya Yamanaka of Kyoto University in Japan.  

The new cells come from patients with 10 incurable genetic diseases and conditions, including Parkinson’s, sclerosis, juvenile diabetes,and Down’s Syndrome.  The new stem cells are not at the point yet that they can treat anyone, but this allows many researchers to experiment with these cells in lab dishes to better understand the diseases.

This new method could allow scientists to overcome the morality barrier of stem cell research, that is destroying viable eggs and embryos.

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French researchers say that gene therapy showed great promise in combating the tragic neurogenerative disease: Huntington’s Disease.

The Process Behind Huntington’s Disease

The disease strikes around one person in 10,000, with the problems mainly showing up between the early 30s and the age of 50.  Its symptoms are uncontrolled, jerky movement, leading to personality change, dementia and death, which occurs around 15 years after the first signs emerge.  Huntington’s is caused by a flaw in a single gene (IT15) on Chromosome 4.  This genetic defect causes a mutated form of a protein called huntingtin to kill cells in the brain.  It is a dominant trait, so if one parent has it, offspring have a 50% chance of getting the disease, and if both have it, the child will certainly get the problem gene.

Scientists at the Institute of Biomedical Imaging and Molecular Imaging Research Centre were experimenting with a modified virus to deliver a corrective gene into brain cells that boosts a natural shield against the problems caused by huntingtin’s.  The protection comes in the form of a ciliary neurotrophic factor, or CNTF.  When the brain is cut or smashed, the synthesis of CNTF is increased as it heps neurons to survive the onslaught.  Like a Trojan horse, the virus is injected into the affected part of the brain, where it infects cells, inserting the gene for CNTF.

Experiments on lab rats, followed by primates, show that the technique does protect striatum cells, and the team is now moving towards a clinical trial on humans.  Other genes, like the glial-derived neurotrophic factor have been inserted into brain tissue as well via virus, and produced similar effects.

Gene therapy shows prospect of being able to slow, stop or even reverse a disease by correcting a flawed gene.  However, many experiments that did well on lab animals performed poorly on humans.

British researchers have also said that they had found that an inflammation-causing protein called IL-6 could serve as an early warning sign of Huntington’s.  People with high levels of this protein went on to develop symptoms of the disease more than a decade later.  This early warning system, combined with the new gene therapy, might help combat Huntington’s.

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Scientists have discovered that using tiny magnets to deliver anti-cancer gene thereapy could make the therapy more effective.  Gene therapy replaces faulty genes, which would possibly code for the regulatory proteins that cause cells to die or stop replicating.  IF one of these genes is damaged through mutation, the cell divisions become very rapid and uncontrolled, causing cancer.  The problem has been getting the genes to the right part of the body to treat the damaged DNA.

Gene Therapy, but Instead of Using Viruses, Scientists Want to Use WBCs and Magnets

Now, by inserting magnetic nanoparticles into white blood cells and injecting them into the bloodstream, doctors have been able to guide the genes attached by using an external magnet.  Thus, the therapy has actually reached its target and in great numbers causing the treatment to become much more powerful.  The new method might also be used to send genes to treat other conditions like arthritis or heart disease.

Tests on humans are still not quite ready.  As of now, the tests have only treated tumors just under the skin of mice.  The real test will be treating tumors deep inside the body, where cancer normally strikes organisms.  “We would hope that this will be safer because we are using a natural mechanism in the body and patients’ own white blood cells to deliver the gene therapy,” says Dr. Clair Lewis, the head researcher.

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