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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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The US federal government has lifted an enforced moratorium on funding research into how to make viruses deadlier and more transmissible.

The moratorium, which was imposed three years ago, froze funding for what’s called “gain of function” research: controversial experiments seeking to alter pathogens and make them even more dangerous. Now, the money is back on the table, giving those trials the green light once more.

The director of the National Institutes of Health (NIH), Francis S. Collins, announced the lifting of the moratorium on Tuesday, saying gain of function (GOF) research with viruses like influenza, MERS, and SARS could help us “identify, understand, and develop strategies and effective countermeasures against rapidly evolving pathogens that pose a threat to public health”.

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