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In an animal study, Mayo clinic researchers identify ways to delay and increase survival from Lou Gehrig’s disease

Researchers discovered a modified antioxidant enzyme that delayed onset and increased survival of mice with familial amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The study found that the polyamine-modified catalase better penetrated the blood-brain barrier, reducing levels of hydrogen peroxide and nitric oxide.

SourceMayo Clinic·JournalAnnals of Neurology·DateDec 17, 2000

Gene discovery provides link between neurological disorders

Researchers discovered a new gene, neurofilament light, associated with Charcot-Marie-Tooth disease, which affects peripheral nerves and leads to progressive weakness. The defect is linked to demyelination, resulting in axonal loss and muscle denervation, also seen in other neurological disorders like Parkinson's and Alzheimer's diseases.

SourceThe American Journal of Human Genetics·JournalAmerican Journal of Human Genetics·DateJun 12, 2000

Duke scientists reverse a rare form of muscular dystrophy in mice using gene therapy

Researchers have successfully reversed damage caused by Pompe disease, a rare inherited muscle-wasting disorder, in laboratory mice using a modified virus to deliver a therapeutic gene. The study's findings show promise for treating dozens of forms of muscular dystrophy and may lead to a continuous supply of enzyme production.

SourceDuke University Medical Center·JournalProceedings of the National Academy of Sciences·DateAug 2, 1999

Dietary Supplements Could Put A Stop To A Royal Curse

Researchers have discovered naturally occurring enzymes that can detoxify porphyrins, building blocks of haemoglobin, in a potential breakthrough for treating variegate porphyria. This rare disorder was passed down through the royal family, including Queen Elizabeth II's first cousin Prince William of Gloucester.

SourceNew Scientist·JournalThe New Scientist·DateMay 19, 1999

New Gene Therapy Strategy Keeps Muscles Strong In Old Age: Possibilities Seen For Disease Treatment, But Also For Athletic Or Cosmetic Enhancements

Scientists have developed a novel gene therapy treatment that permanently blocks age-related loss of muscle size and strength in mice. The treatment increases muscle strength by up to 27% in older mice, fully restoring their strength to young adulthood.

SourceUniversity of Pennsylvania School of Medicine·JournalProceedings of the National Academy of Sciences·DateDec 14, 1998

The Best Molecular Snapshot Yet Of Cells Affected By Alzheimer's Disease

A University of Rochester team has developed a technology that provides the most thorough information yet on cells from the brains of deceased Alzheimer's patients, enabling comparison with healthy and sick cells. The study identified five genes whose expression differed significantly between healthy and Alzheimer's brains.

SourceUniversity of Rochester·JournalProceedings of the National Academy of Sciences·DateAug 3, 1998

Gene-Altered Mice Boost Studies of Cardiomyopathy: Enables Research Into Causes And Treatment Of Heart Failure

Scientists have created gene-altered mice that closely reproduce the clinical features of dilated cardiomyopathy, allowing for unprecedented study of early changes and development of new therapies. The model enables researchers to understand the causes and mechanism of the disease, ultimately aiming to develop more effective treatments.

SourceUniversity of Chicago Medical Center·JournalJournal of Clinical Investigation·DateJun 1, 1998

Multiple Genes Lead To Same Problem In Enlarged-Heart Disease Linked To Sudden Death In Athletes, New Study Shows

A new study has identified a common functional problem in enlarged heart disease that causes sudden death in athletes, regardless of the genetic cause. This finding suggests that a single therapeutic approach may be effective for all victims of hypertrophic cardiomyopathy (HCM), a leading cause of sudden death in young adults.