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Gene therapy works in mice to prevent blindness that strikes boys (press release)

Sunday, August 07, 2005
by Mike Adams, the Health Ranger
Editor of NaturalNews.com (See all articles...)
Tags: health news, Natural News, nutrition


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Writing in the August issue of Molecular Therapy, scientists from the UF Genetics Institute describe how they successfully used gene therapy in mice to treat retinoschisis, a rare genetic disorder that is passed from mothers, who retain their sight, to their sons.

"Currently there is no treatment," said William Hauswirth, Ph.D., the Rybaczki-Bullard professor of ophthalmic molecular genetics. "These children lose their sight gradually, often with devastating results. What happens is the retina actually begins to split in the middle, causing loss of central vision - that's the vision that you need to be able to read or walk around."

Scientists say the gene transfer method eventually could be applied to many eye diseases caused by single gene defects, including a host of retinal disorders.

Retinoschisis is usually first detected in boys between 5 and 10 years of age when their vision problems cause reading difficulties. In a healthy eye, retinal cells secrete a protein called retinoschisin, or RS1, which acts like glue to connect the layers of the retina. Without it, the layers separate and tiny cysts form, devastating the vision and often leading to blindness in about 1 of every 5,000 boys.

UF researchers injected a healthy version of the human RS1 gene to the sub-retinal space of the right eyes of 15-day-old male mice, which, like boys with the disease, don't have the healthy gene to maintain the retina. In terms of disease development, the condition in the mice was roughly equivalent to retinoschisis in a 10-year-old boy.

Six months later, researchers looked at the interior of the eyes with a laser ophthalmoscope and found cyst formation was clearly evident in the untreated eyes, but the treated eyes appeared healthy. The eye's photoreceptor cells - the rods and cones that help the brain process light and color - were spared from the disease and the connections between the layers of the retinas were intact.

In addition, the protein appears capable of moving within the retina to its target sites and the beneficial changes appear to be long lasting, researchers said. Especially encouraging were signs the treatment may be able to repair retinal damage.

The treatment has promising implications for other genetic eye diseases that involve the eye's ability to process light, including retinitis pigmentosa, which affects about 200,000 people in the United States and is one of the most common inherited causes of blindness in people between the ages of 20 and 60.

"We've been very successful in curing a disease in mice that has a direct copy in humans," said Hauswirth, who, in conjunction with UF, has interest in a biotechnology company that may seek to market some of the research technology. "It may take two to five years before we try this in human patients because of the need for safety studies, but we feel based on success so far, we will be able to provide formal evidence for safety that will allow us to get treatment into the clinic."

UF researchers worked with Bernhard Weber, Ph.D., at the Institute of Human Genetics in Regensburg, Germany, and Robert Molday, Ph.D., director of the Center for Macular Research at the University of British Columbia in Vancouver.

The Foundation Fighting Blindness, the National Institutes of Health and the Macula Vision Research Foundation supported the research.

"We now have proof of principle that gene therapy can basically prevent retinoschisis," said Stephen Rose, Ph.D., chief research officer for the Maryland-based Foundation Fighting Blindness. "Furthermore, this therapy apparently demonstrates that even if disease has begun, there is a healing that takes place. That raises hope for suffering patients that we may be able to offer something that can improve the quality of their lives."


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About the author:Mike Adams (aka the "Health Ranger") is a best selling author (#1 best selling science book on Amazon.com) and a globally recognized scientific researcher in clean foods. He serves as the founding editor of NaturalNews.com and the lab science director of an internationally accredited (ISO 17025) analytical laboratory known as CWC Labs. There, he was awarded a Certificate of Excellence for achieving extremely high accuracy in the analysis of toxic elements in unknown water samples using ICP-MS instrumentation. Adams is also highly proficient in running liquid chromatography, ion chromatography and mass spectrometry time-of-flight analytical instrumentation.

Adams is a person of color whose ancestors include Africans and Native American Indians. He's also of Native American heritage, which he credits as inspiring his "Health Ranger" passion for protecting life and nature against the destruction caused by chemicals, heavy metals and other forms of pollution.

Adams is the founder and publisher of the open source science journal Natural Science Journal, the author of numerous peer-reviewed science papers published by the journal, and the author of the world's first book that published ICP-MS heavy metals analysis results for foods, dietary supplements, pet food, spices and fast food. The book is entitled Food Forensics and is published by BenBella Books.

In his laboratory research, Adams has made numerous food safety breakthroughs such as revealing rice protein products imported from Asia to be contaminated with toxic heavy metals like lead, cadmium and tungsten. Adams was the first food science researcher to document high levels of tungsten in superfoods. He also discovered over 11 ppm lead in imported mangosteen powder, and led an industry-wide voluntary agreement to limit heavy metals in rice protein products.

In addition to his lab work, Adams is also the (non-paid) executive director of the non-profit Consumer Wellness Center (CWC), an organization that redirects 100% of its donations receipts to grant programs that teach children and women how to grow their own food or vastly improve their nutrition. Through the non-profit CWC, Adams also launched Nutrition Rescue, a program that donates essential vitamins to people in need. Click here to see some of the CWC success stories.

With a background in science and software technology, Adams is the original founder of the email newsletter technology company known as Arial Software. Using his technical experience combined with his love for natural health, Adams developed and deployed the content management system currently driving NaturalNews.com. He also engineered the high-level statistical algorithms that power SCIENCE.naturalnews.com, a massive research resource featuring over 10 million scientific studies.

Adams is well known for his incredibly popular consumer activism video blowing the lid on fake blueberries used throughout the food supply. He has also exposed "strange fibers" found in Chicken McNuggets, fake academic credentials of so-called health "gurus," dangerous "detox" products imported as battery acid and sold for oral consumption, fake acai berry scams, the California raw milk raids, the vaccine research fraud revealed by industry whistleblowers and many other topics.

Adams has also helped defend the rights of home gardeners and protect the medical freedom rights of parents. Adams is widely recognized to have made a remarkable global impact on issues like GMOs, vaccines, nutrition therapies, human consciousness.

In addition to his activism, Adams is an accomplished musician who has released over a dozen popular songs covering a variety of activism topics.

Click here to read a more detailed bio on Mike Adams, the Health Ranger, at HealthRanger.com.

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