CF Rx: Exercise

We can’t change our genes – yet – but we can alter our environments to ease life with an inherited illness. Meet two young people whose love of dance, running, yoga, and more is helping

When a Bad Animal Model is Good: Cystic Fibrosis

mice-DNA-690x320A “good” animal model is one that has the same symptoms of a disease that we do, right? Not always. Sometimes we can actually learn more when an animal is not a perfect model; their

New Miracle Drugs: What Would You Pay?

Levi Collazo, a biology major at Southwestern Oklahoma State University, has cystic fibrosis (CF). “In the first photo, I was making fun of my own weight. I’ve always used humor as a defense mechanism. I weighed 110 pounds. I’m 5’10″. … Continue reading »

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When Mutation Counters Infection: From Sickle Cell to Ebola

 While pharmaceutical companies focus on drug discovery for Ebola virus disease, a powerful clue is coming from a rare “Jewish genetic disease” that destroys the brain. People with Niemann-Pick C1 disease can’t get Ebola, adding to the list of disease … Continue reading »

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A Checklist for Gene Therapy From the UK Cystic Fibrosis Trial

Washington, D.C.           I’m at the American Society of Gene and Cell Therapy annual meeting, one of my favorite conferences. The very first talk provided a great example of why it is taking gene therapy so long to reach the …

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