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Stem Cell Treatment for Progeria, The Fast-Aging Disease

Progeria
The researchers at the Salk Institute in California have come up with a stem cell technique that could not only help in finding a cure for Progeria, but also help scientists understand the process of normal ageing in a better way.

Progeria (Prematurely Old) or Hutchinson-Gilford Progeria Syndrome is a very rare genetic disease, where the victims (children) age rapidly and do not live to become teenagers.

Most of them die from complications related to hardening of the arteries and blood vessels.

So far only 64 Progeria cases have been recorded throughout the world.

A mutation (change in the DNA sequence) on the LMNA gene, results in the defective formation of protein Lamin A. This protein plays a key role in building the nuclear membrane (protective layer surrounding the cell’s genetic material).

Scientists have developed a technique which alters the DNA in such a way that it instructs specific types of cells, such as skin and muscle cells to convert to Induced pluripotent stem (IPS) cells. IPS cells, just like embryonic stem cells have the capacity to turn into the type of tissue into which they are transplanted.

The above mentioned technique was used on the damaged skin cells taken from Progeria patients, by the research team led by Juan Carlos Izpisua Belmonte and Guanghui Liu in California. Interestingly, they found no defects in the nuclei of the damaged cells that were reprogrammed to become IPS cells.

In other words the reprogrammed cells resembled normal, healthy IPS cells.

But these cells when transplanted into the muscle tissue in the laboratory started behaving like Progeria cells, resuming the defective functioning.

Though the results did not turn out to be completely positive, the technique has paved a path to study the onset and progression of the disease.

Also, Liu and his team members are working on developing a technique that can fix the mutation responsible for Progeria in its stem cell stage.

1 response to Stem Cell Treatment for Progeria, The Fast-Aging Disease

  1. This is an excellent news. The rarity of the disorder makes lesser people know about it and it also causes people to not understand the pain the affected person goes through. More and more research has to be done to help the affected people and more awareness programs have to be conducted for people to be aware of it

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