Gene Therapy


People from Mexico Show Vast Genetic Diversity

Mexico City, Mexico (Alex Torres/Flickr)

Mexico City, Mexico. (Alex Torres/Flickr)

The genetic diversity of the Mexican population is so vast that two people of Mexican descent can be as genetically different from each other as a European and a Chinese person. That’s the finding of researchers from UC San Francisco, Stanford, and the Mexican National Institute of Genomic Medicine. It’s considered the first, large-scale analysis of its kind,  and the study could change the way health care is delivered to Mexican-Americans. It helps drive forward the move toward personalized medicine.

KQED News anchor Mina Kim spoke with UCSF Professor Esteban Burchard, one of the co-authors of the study, during a Thursday evening newscast. Burchard described that because of “historical factors, geographical factors, linguistic factors,” the researchers identified that indigenous populations in Mexico are genetically distinct. The genetic ancestry mirrors the geography of Mexico.” Continue reading

“Landmark” in Gene Therapy Treatment

People with hemophilia lack a substance that helps blood clot.  (Shannon Muskopf: Flickr)

People with hemophilia lack a substance that helps blood clot. (Shannon Muskopf: Flickr)

In a preliminary study, six patients in England were successfully treated by gene therapy for the blood-clotting disease hemophilia B. Researchers injected each patient with the correct form of the gene that makes the needed substance that helps blood to clot, called a clotting factor.

“I think it’s a significant advancement in gene therapy and a treatment for hemophilia,” said Mark Kay, Professor at Stanford’s School of Medicine and a co-author of the study. “It’s been over a year for some of the patients and they are continuing to make the [clotting] factor. We have to start at low doses and work our way up. We have patients that are at 10 percent level for half a year.”

The current medication has a lifetime cost per adult patient of up to $20 million.

That 10 percent is remarkable. In hemophilia, people with a severe form of the disease have less than one percent of the clotting factor. Those with 10 percent are often able to live a normal life.

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