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Gene Therapy

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NEWS
March 16, 2011 | By Melissa Healy, Los Angeles Times
A small-scale gene therapy trial conducted at seven U.S. medical centers has found that a single infusion of a specialized gene, piggybacked onto a virus and fed directly into the brain, can safely lessen the severity of symptoms and improve response to medication in patients with advanced Parkinson's page NIH" href="http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001762/" target="_blank"> Parkinson's disease . The clinical trial --...
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SCIENCE
April 23, 2014 | By Monte Morin
A procedure that uses a series of electric jolts to inject lab-designed DNA molecules into cells of the inner ear may help to regrow auditory nerves in people with profound hearing loss, according to researchers. In a paper published Wednesday in Science Translational Medicine , Australian researchers said they used tiny electrodes and gene therapy to regenerate nerve cells in chemically deafened guinea pigs. The procedure, they said, may one day improve the functioning of human cochlear implants -- electronic devices that provide hearing sensations to the deaf.
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OPINION
December 12, 1999
Re using biotech to plump up hogs (Dec. 8): To heck with farm animals! They need to find a way of reversing that technique. What we really need is gene therapy to slim down fat humans. ALICE L. RAMIREZ Los Angeles
SCIENCE
July 11, 2013 | By Melissa Pandika
Italian researchers have used a defanged version of HIV to replace faulty genes - and eliminate devastating symptoms - in children suffering two rare and fatal genetic diseases. Improved gene therapy techniques prevented the onset of metachromatic leukodystrophy in three young children and halted the progression of Wiskott-Aldrich syndrome in three others. The advance represents a major stride for a field that has struggled to translate experimental successes in lab animals into safe and effective treatments for people, experts said.
NEWS
October 24, 2012 | By Karen Kaplan, Los Angeles Times
Scientists have demonstrated a new type of gene therapy that would - in principle - allow mothers to avoid saddling their children with rare diseases that could result in heart problems, dementia, diabetes, deafness and other significant health issues. The disorders in question are all due to mutations in one of the 37 genes in our mitochondrial DNA. “Mitochondria are structures within cells that convert the energy from food into a form that cells can use,” according to this explainer from the NIH's National Library of Medicine.
SCIENCE
April 23, 2014 | By Monte Morin
A procedure that uses a series of electric jolts to inject lab-designed DNA molecules into cells of the inner ear may help to regrow auditory nerves in people with profound hearing loss, according to researchers. In a paper published Wednesday in Science Translational Medicine , Australian researchers said they used tiny electrodes and gene therapy to regenerate nerve cells in chemically deafened guinea pigs. The procedure, they said, may one day improve the functioning of human cochlear implants -- electronic devices that provide hearing sensations to the deaf.
NEWS
December 3, 2010 | By Shari Roan, Los Angeles Times
Disease-modifying treatments for Alzheimer's disease have proved elusive. However, a study in mice published this week suggests that a treatment strategy relying on gene therapy may be worth pursuing. Researchers at the Gladstone Institute of Neurological Disease in San Francisco found that mice and humans with Alzheimer's disease have unusually low levels of an enzyme called EphB2 in the parts of the brain that control memory. EphB2 plays an important role in fostering communication between brain cells.
NEWS
June 29, 2012 | By Jon Bardin, Los Angeles Times / For the Booster Shots blog
Can't kick cigarettes? A vaccine may one day help by preventing nicotine from reaching its target in the brain, according to research published this week. Most smoking therapies do a poor job of stopping the habit - 70% to 80% of smokers who use an approved drug therapy to quit relapse. Scientists say this is because the targets of existing therapies are imperfect, only slightly weakening nicotine's ability to find its target in the brain. So some scientists have been trying a different approach - creation of a vaccine.
SCIENCE
November 6, 2009 | Thomas H. Maugh II
In the third gene-therapy success of recent weeks, French researchers have arrested the progression of the rare and fatal degenerative disorder adrenoleukodystrophy, which was at the heart of the popular movie "Lorenzo's Oil." The disease has stabilized in two boys who were 7 years old when the therapy was performed two years ago, the team reported today in the journal Science. "This is a disease that never, ever stabilizes" on its own, said Dr. Katherine High of the Children's Hospital of Philadelphia, who was not involved in the research.
OPINION
September 29, 1985
The approval of federal guidelines for gene-transplant experiments on humans is an important step in realizing the potential of genetic research. Under the guidelines, researchers who receive federal funds must submit a detailed proposal for each genetic therapy that they want to undertake, and must receive approval from a local committee and from the Recombinant DNA Advisory Committee of the National Institutes of Health in Washington.
NEWS
October 24, 2012 | By Karen Kaplan, Los Angeles Times
Scientists have demonstrated a new type of gene therapy that would - in principle - allow mothers to avoid saddling their children with rare diseases that could result in heart problems, dementia, diabetes, deafness and other significant health issues. The disorders in question are all due to mutations in one of the 37 genes in our mitochondrial DNA. “Mitochondria are structures within cells that convert the energy from food into a form that cells can use,” according to this explainer from the NIH's National Library of Medicine.
HEALTH
September 13, 2012 | By Elaine Herscher
Genes make us who we are - in sickness and in health. We get our genetic makeup from our parents, of course, but in the future, we might be getting genes from our doctors too. Imagine your doctor promising to cure your cancer or heart disease by prescribing some new snippets of DNA. For some diseases, gene therapy is already a reality. In other cases, genetic cures are still years away. Despite many challenges and setbacks - including some that are surely yet to come - experts predict that gene therapy will eventually become a crucial and even common part of healthcare.
SCIENCE
August 15, 2012 | By Rosie Mestel, Los Angeles Times
Dog lovers may be interested in an article published this week in the New England Journal of Medicine: It highlights the discoveries scientists are making about diseases that various dog breeds are prone to -- and how those findings can benefit human health as well as that of canines. It's written by longtime dog genetics researcher Elaine Ostrander of the National Human Genome Research Institute. The discoveries are possible because of several things: First off, both the human genome and dog genomes have been sequenced.
SCIENCE
July 20, 2012 | By Thomas H. Maugh II, Los Angeles Times
The long-frustrated field of gene therapy is about to reach a major milestone: the first regulatory approval of a gene therapy treatment for disease in the West. The European Medicine Agency's Committee for Medicinal Products for Human Use said Friday that it is recommending approval of Glybera, a treatment for lipoprotein lipase deficiency manufactured by uniQure of Amsterdam. The European Commission generally follows the recommendations of the agency, and if it does so this time, the product could be available in all 27 members of the European Union by the end of the year.
SCIENCE
July 18, 2012 | By Jon Bardin, Los Angeles Times
We like to think of the Olympics as a level playing field - that's why doping is banned. But scientific research complicates this view: There are numerous genetic factors known to confer advantages in athletic contests, from mutations that increase the oxygen carrying capacity of blood to gene variants that confer an incredible increase in endurance, and these mutations appear to be especially common in Olympic athletes. In other words, we may want an egalitarian Olympic games, but it probably isn't in the cards.
NEWS
June 29, 2012 | By Jon Bardin, Los Angeles Times / For the Booster Shots blog
Can't kick cigarettes? A vaccine may one day help by preventing nicotine from reaching its target in the brain, according to research published this week. Most smoking therapies do a poor job of stopping the habit - 70% to 80% of smokers who use an approved drug therapy to quit relapse. Scientists say this is because the targets of existing therapies are imperfect, only slightly weakening nicotine's ability to find its target in the brain. So some scientists have been trying a different approach - creation of a vaccine.
SCIENCE
July 20, 2012 | By Thomas H. Maugh II, Los Angeles Times
The long-frustrated field of gene therapy is about to reach a major milestone: the first regulatory approval of a gene therapy treatment for disease in the West. The European Medicine Agency's Committee for Medicinal Products for Human Use said Friday that it is recommending approval of Glybera, a treatment for lipoprotein lipase deficiency manufactured by uniQure of Amsterdam. The European Commission generally follows the recommendations of the agency, and if it does so this time, the product could be available in all 27 members of the European Union by the end of the year.
OPINION
June 9, 2011
There is a dramatic arc to the three-decade history of AIDS as an epidemic and social phenomenon. Since it was first reported 30 years ago this month, the disease has evolved from an unnamed and mysterious illness to a diabolical killing machine to a chronic condition that can be managed with drugs. Overall, AIDS has killed 25 million people worldwide. But the successful campaign to transform it — and HIV, the virus that causes it — from a sentence of sure death to a diagnosis that comes with a treatment plan is a medical triumph.
NEWS
March 16, 2011 | By Melissa Healy, Los Angeles Times
A small-scale gene therapy trial conducted at seven U.S. medical centers has found that a single infusion of a specialized gene, piggybacked onto a virus and fed directly into the brain, can safely lessen the severity of symptoms and improve response to medication in patients with advanced Parkinson's page NIH" href="http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001762/" target="_blank"> Parkinson's disease . The clinical trial --...
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