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Some 50,000 people in the U.S. are diagnosed with Parkinson’s disease (PD) every year. The American Institute of Neurology estimates there are one million people affected with this neurodegenerative disorder, with 60 years as average age of onset. Falls and fall-related injuries are a major issue for people with Parkinson’s?up to 70 percent of advanced PD patients fall at least once a year and two-thirds suffer recurring falls. These fall rates are twice as high as those of adults of comparable age, so improving balance in patients with Parkinson’s would provide a major health advantage.

Sunil Agrawal, professor of mechanical engineering and of rehabilitation and regenerative medicine at Columbia Engineering, along with Dario Martelli, a post-doctoral researcher in his group, have been working on this issue with Movement Disorders faculty from the department of neurology at Columbia University Medical Center?Stanley Fahn, a leading expert in Parkinson’s, and Un Jung Kang, division director, and Movement Disorder Fellow Lan Luo. In their latest study, published today in Scientific Reports, the team looked at whether or not Parkinson’s disease affects patients’ balance and diminishes their ability to react and adapt to walking with perturbations. The researchers found that the ability to adapt to multiple perturbations or to modify responses to changing amplitudes or directions was not affected by PD; both the Parkinson’s and the healthy subjects controlled their reactive strategies in the same way.

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A single infusion gene therapy treatment improved levels of the essential blood clotting protein Factor VIII, with 85 percent of patients achieving normal or near-normal levels of the blood clotting factor, even many months after treatment.


Summary: British doctors say they have achieved “mind-blowing” results using gene therapy to rid people of hemophilia A. [This article first appeared on LongevityFacts. Author: Brady Hartman.]

We are one step closer to a cure for hemophilia according to the results of a groundbreaking gene therapy trial published in the New England Journal of Medicine.

Clinical researchers at the Queen Mary University of London and Barts Health NHS Trust have found that over one year after a single gene therapy treatment, patients with hemophilia A are showing normal levels of the previously missing blood clotting factor, effectively curing them.

New view on mitochondrial DNA could put the brakes on mutations that drive diseases. Scientists perform landmark sequencing of mitochondrial DNA and discover surprising facts.


Summary: New view on mitochondrial DNA could help put the brakes on mutations that drive diseases. [Author: Brady Hartman. This article first appeared on LongevityFacts.]

DNA sequences between mitochondria inside a single cell are vastly different, reported scientists in the Perelman School of Medicine at the University of Pennsylvania. This discovery will help to illuminate the underlying mechanisms of diseases that start with mutations in mitochondrial DNA and provide clues about how patients might respond to specific treatments. The researchers published their findings in the journal Cell Reports this week.

Mutant Mitochondrial DNA

When Tina Gibson got married seven years ago, the 26-year-old knew it was unlikely that she would have children naturally. Her husband, 33-year-old Benjamin Gibson, had cystic fibrosis, a condition that can make men infertile, the couple told CNN.

The East Tennessee pair decided they would eventually adopt a child instead — and that they would foster several children in the meantime, until they were ready.

Then, last year, during a break between foster children, her father told them about something he’d heard on the news — embryo adoption, according to CNN. Gibson couldn’t get the idea out of her head.

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Gene therapy is the process of replacing defective genes with healthy ones, adding new genes to help the body fight or treat disease, or deactivating problem genes. It holds the promise to transform medicine and create options for patients who are living with difficult, and even incurable, diseases. Learn how this innovative therapy works.

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