Thursday, October 13, 2011

Gene therapy and stem cells unite

Imagine the people with fatal genetic liver disease (which is caused by a gene mutation of only two base pairs of DNA) being able to recieve treatment or even cure without the need for liver transplant?

Researchers are now working on harvesting patient's own stem cells and using molecular scalpel  scientists were able to cut out the mutated gene and insert a correct one in its place. Then the stem cell with a corrected gene sequence is used to treat the organ. This means the patient will not need a transplant and immunouppressant drugs.

Such a leap of personalized medicine and treatment is a fork in the road, are we going to be able to decrease the technology costs so drastically for this to be even an option? or only very few select individuals will be able to afford this?

Will the companies weather out the financial hardship and be able to create the technologies needed to make personalized gene/stem cell therapy as a template for all genetic disorders and diseases? 

Aside: Maybe the plot of sci-fi movie Repo men is no longer our future :)

For more info read the full article Gene therapy and stem cells unite at BBC Health

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

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