Stem-cell and genetic therapies make a healthy marriage

From Nature, 15 May 2019:

Aside from a 20-second exposure to the outside world at birth, David Vetter spent his entire life cocooned in plastic. Afflicted by severe combined immunodeficiency or SCID, a hereditary disease that severely compromises or destroys the immune system, the ‘boy in the bubble’ was exquisitely vulnerable to infection. Eventually, a latent virus hidden in a bone-marrow transplant killed him in 1984 at the age of 12.

Until a few years ago, a bone-marrow transplant from a matched donor was the only hope that children such as David had of achieving a normal life. Now, two rapidly advancing areas of medicine — stem-cell and gene therapies — are combining to offer what amounts to a cure: a complete reboot of the immune system, free from the genetic mutations that cause diseases such as SCID.

And it’s not just immunodeficiency disorders, or even genetic disorders, that could benefit from the combination of stem-cell and gene therapies. This scientific partnership could fight everything from blood diseases to HIV. Read more.

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