Category Archives: Genetic Therapy

Welcomeat the website of the DMD Genetic Therapy Group The primary mission of the DMD Genetic Therapy Group at the Department of Human Genetics (chaired by Prof. Dr. Silvre van der Maarel) of the Leiden University Medical Center is the development of a genetic therapy for Duchenne Muscular Dystrophy (DMD). The project started in 1998 and is currently supervised by Dr. Annemieke Aartsma-Rus, after Dr. Judith C.T. van Deutekom left in February 2007 to work as Head of research for the biotech company Prosensa B.V. (Leiden, the Netherlands). In a joint effort the DMD Genetic Therapy Group and Prosensa are developing antisense oligonucleotides (AONs) as small synthetic molecule drugs for DMD. The Department of Human Genetics has an excellent track record in the field of duchenne muscular dystrophy. It set up the first DNA based carrier detection and prenatal diagnosis worldwide, performed etiological DNA, RNA and protein research, and developed diagnostics, for DMD and other muscular diseases, in an international framework. The DMD Genetic Therapy Group was awarded for its research by the Princess Beatrix Fund in 2001 and by the LUMC (C.J. Kok Award) in 2003. In 2007, the first clinical trial on antisense-mediated exon skipping was successfully completed … Continue reading →

A new genetic therapy that helped blind mice and dogs respond to light stimulus could restore sight to people who suffer from diseases such as retinitis pigmentosa (a gradual loss of vision from periphery inwards). The therapy uses chemicals known as photoswitches, which change shape when hit with light, to open the channels that activate retinal cells. Treated mice can distinguish between steady and flashing light, while dogs with late-stage retinal degeneration also regain some sensitivity to light. The procedure starts with an adeno-associated virus. Some retina cells in blind mice survive after disease kills the rod and cone photoreceptors, but they won't work on their own. The virus inserts a gene that instructs the cells to produce a modified version of a common glutamate receptor ion channel. Then photoswitches are attached to the newly-formed ion channels, akin to a glutamate amino acid dangling on a light-sensitive string. When light hits a photoswitch, it forces an ion channel open, thereby turning the retinal neurons on and off many times a second. The researchers are able to insert the gene into almost all of the million or so retinal ganglion cells, which should restore useful vision. The success of this therapy … Continue reading →

I recently had the pleasure of hearing futurist Raymond Kurzweil speak at an SFU Public Square event in Vancouver. I have a big soft spot for him, ever since he invented the Kurzweil Reading Machine in the 1970s. It was a boon to the blind and people with poor eyesight, and opened the door for many other technologies. One of the first machines was installed at the New York Public Library, and I was sent to interview its keeper. What do people bring to read on it? I asked. Mostly pornography, he replied. I thought I heard him wrong until he explained that there were readers for the blind who could tackle The History of the American Civil War,soamachine was being used for racier content. I use this story in my book Technocreep, to illustrate the unintended consequences of technology. Now, Kurzweil is at it again, suggesting that if we make it through the next 10 or 15 years, some of us, at least, may become immortal. He predicts that, by then, we will be adding more than a year to the human lifespan each year, and not just for babies. He likens our bodies to computer software that has … Continue reading →

Top Healthcare Stocks JNJ -1.53% PFE -0.55% ABT -1.06% MRK -1.24% AMGN -0.36% Health care stocks were lower this afternoon, with the NYSE Health Care Sector Index falling about 1.1% and shares of health care companies in the S&P 500 retreating about 0.9% as a group. In company news, RXi Pharmaceuticals Corp ( RXII ) advanced Tuesday after the genetic therapy company said photographs of the first patients enrolled in Phase IIb testing of its RXI-109 drug candidate indicate it may be effective in suppressing recurrence of hypertrophic scars through the first three months of treatment. The patients will continue to be monitored for another six months to determine whether the results persist over time. Complete three-month results from the current trial are expected in early 2015. The company was planning to discuss the early test results for RXI-109 this afternoon at the BIO Investor Forum running now through Wednesday in San Francisco. The RXII presentation was scheduled to begin at 1 p.m. ET. Here is the original post: Sector Update: Health Care Stocks Falling; RXII Pharmaceuticals Climbs After Genetic Drug Appears to Hinder Scarring … Continue reading →

Genetic therapy This is a video talking about genetic therapy. I put a real life story through a comic so you can watch and listen in the same time. By: af5aristoto … Continue reading →

Damage to the central nervous system, the brain and spinal cord, is currently irreparable. But this may change soon as researchers have now discovered that genetic and chemical treatment could help regenerate damaged nerves. Future therapies could help repair nerve damage after people suffer spinal cord injury or brain trauma, said the study. "Due to the complexity of the structure of the central nervous system, regrowth leads most often to incorrect rewiring, such as pain," said Simone Di Giovanni, a neuroscientist and neurologist from Imperial College London. "The peripheral nervous system is much more simple and has effective, although partial, regeneration," noted the researchers. Most spinal cord injuries are caused by damage to axons, the long extensions of neurons that send messages around inside the nervous system, the study pointed out. The researchers found that when nerves are damaged in the peripheral nervous system, they emit signals to switch on a program to initiate nerve growth. This program is "epigenetic", meaning that it can activate or deactivate genes without altering DNA. They also identified a protein, called P300/CBP-associated factor (PCAF), as being central to initiating nerve regrowth. The researchers found that when this protein was injected into mice that had … Continue reading →

Home > News > health-news London, April 16 : Damage to the central nervous system, the brain and spinal cord, is currently irreparable. But this may change soon as researchers have now discovered that genetic and chemical treatment could help regenerate damaged nerves. Future therapies could help repair nerve damage after people suffer spinal cord injury or brain trauma, said the study. "Due to the complexity of the structure of the central nervous system, regrowth leads most often to incorrect rewiring, such as pain," said Simone Di Giovanni, a neuroscientist and neurologist from Imperial College London. "The peripheral nervous system is much more simple and has effective, although partial, regeneration," noted the researchers. Most spinal cord injuries are caused by damage to axons, the long extensions of neurons that send messages around inside the nervous system, the study pointed out. The researchers found that when nerves are damaged in the peripheral nervous system, they emit signals to switch on a program to initiate nerve growth. This program is "epigenetic", meaning that it can activate or deactivate genes without altering DNA. They also identified a protein, called P300/CBP-associated factor (PCAF), as being central to initiating nerve regrowth. The researchers found that … Continue reading →

Genetic therapy attempts to remedy genetic disorders through the application of new genes. If you're interested in pursuing a career in medical or biological research, continue reading about this field to determine if it's a good fit for you. Genetic therapists find ways to cure harmful genetic flaws by replacing a non-functioning or mutated gene with a normal gene. These scientists use vectors, which are often viruses, to transfer a replacement gene into the appropriate cell. Genetic therapy techniques may eventually cure genetic disorders that currently affect thousands of people around the world, but the field is still in the early stages of development. Therefore, very little specific information about gene therapy is available. However, Education-Portal.com has some information about related academic programs and career opportunities that you could find useful if you're interested in learning more about this field. Educational programs in gene therapy are only available at the doctoral degree level, but undergraduate programs in biology and molecular biology may explore topics related to the field, including gene expression, cell structure and genetic mutations. Entry into doctoral programs is highly competitive and primarily offered by some of the most prestigious universities in the United States. The rigorous curriculum … Continue reading →