Tag Archives: Barth

Harvard stem cell study promises personalized treatments

Personalized Medicine
Personalized Medicine (Photo credit: Wikipedia)

Scientists have for some time been able to reproduce pieces of human organs on chips. Now in a new study they’ve been able to use a patient’s stem cells to reproduce in the lab chunks of functioning tissue from a human being with a specific disease.

In this case, a team of experts from multiple disciplines “modeled the cardiovascular disease Barth syndrome, a rare X-linked cardiac disorder caused by mutation of a single gene called Tafazzin, or TAZ. The disorder, which is currently untreatable, primarily appears in boys, and is associated with a number of symptoms affecting heart and skeletal muscle function.”

The disease in this case results in very weak contractions of the heart muscle. The hope is that they may also eventually be able to model functioning tissue from patients with other diseases that produce other functional problems.

Why would they want to create functioning yet diseased human tissue outside a human being? The answer is that they can then experiment with and test all kinds of drugs and other treatments that they might not want to use directly on an actual living, breathing human being. In this scenario they were able to inject a genetic product that corrected the contractile problem right there in the lab.

While the article doesn’t say this, I’m thinking it could also mean in the long run fewer animals used for experimentation. And it could lead to shorter times before promising therapies can get to clinical trials.

A brave new world, indeed.

 

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