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Category Archives: Gene therapy

The Puzzle of Pancreatic Cancer: How Steve Jobs Did Not Beat the Odds—but Nobel Winner Ralph Steinman Did

Steve Jobs was a rare case, right down to his death. Announced Wednesday, Jobs's death from "complications of pancreatic cancer" only hints at the vast complexity of the disease to which he succumbed at the age of 56. [More]

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Studying Mental Illness in a Dish

No organ in the human body is as resistant to study as the brain. Whereas researchers can examine living cells from the liver, lung and heart, taking a biopsy of the brain is, for many reasons, more problematic.

The inability to watch living human brain cells in action has hampered scientists in their efforts to understand psychiatric disorders. But researchers have identified a promising new approach that may revolutionize the study and treatment of conditions such as schizophrenia, autism and bipolar dis­order. A team led by researchers at the Salk Institute for Biological Studies in La Jolla, Calif., took skin cells from a patient with schizophrenia, turned them into adult stem cells and then grew those stem cells into neurons. The resulting tangle of brain cells gave neuroscientists their first real-time glimpse of human schizophrenia at the cellular level. Another team, from Stanford University, converted human skin cells directly into neurons without first stopping at the stem cell stage, potentially making the process more efficient. The groups published their results recently in Nature ( Scientific American is part of Nature Publishing Group).

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A Breath of Fresh Air: New Hope for Cystic Fibrosis Treatment (preview)

In 1989 when scientists discovered the defective gene that causes cystic fibrosis, a serious hereditary disorder that primarily strikes children of European descent, it seemed as though a long-hoped-for cure might soon follow. After all, tests in many laboratories showed that providing normal copies of the gene should enable patients to make healthy copies of the protein specified by the gene. If successful, that feat would go a long way toward restoring health in the tens of thousands of people around the world who suffered from cystic fibrosis and typically died in their late 20s. (Half of all patients now live to their late 30s or beyond.) The question was whether researchers would be able to reliably insert the correct gene into the proper tissues in patients’ bodies to rid them of the illness forever.

That task proved harder than anyone had believed. Although scientists successfully engineered viruses to ferry copies of the correct gene into patients’ cells, the viruses did not do the job well. By the late 1990s additional unexpected complications made it increasingly obvious that another approach to addressing the fundamental problem in cystic fibrosis would need to be found.

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New Report Details Uphill Battle to Solve the U.S.’s Pain Problem

Chronic pain affects at least one in three adults in the U.S., which is more than the sum total of those with heart disease, cancer and diabetes combined. For many of these 116 million Americans, their pain is severe and eludes available treatments. In addition to the human suffering, the monetary cost of medical treatment and lost productivity has reached $635 billion a year. [More]

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New Report Details Uphill Battle to Solve the U.S.'s Pain Problem

Chronic pain affects at least one in three adults in the U.S., which is more than the sum total of those with heart disease, cancer and diabetes combined. For many of these 116 million Americans, their pain is severe and eludes available treatments. In addition to the human suffering, the monetary cost of medical treatment and lost productivity has reached $635 billion a year. [More]

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Close Encounters of Science and Medicine

From medicine to science

When I was about 3 or 4 years old, I got very sick. I stayed in bed for many weeks and every day a nurse would come to give me a penicillin shot. The pain from shots turned into fear, in time fear turned into a plan for revenge. When I got better I demanded to have my own syringe and cruelly treated all teddy bears and dolls. If they didn’t look sick I made them sick, just to perform surgeries, sew wounds and give shots. I even offered my service to family members; unfortunately, they stubbornly kept on being healthy.

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