Stem Cell Success: One Couple's Effort to Protect Their Son From Fatal Nerve Disease Will Help Other Boys Too

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Newswise ANN ARBOR, Mich. University of Michigan alumna Brooke Kendrick and her husband Stephen were ready to start a family.

But a devastating inherited nerve disease runs in her family, affecting her brother and threatening to kill or cripple any male child she has. So, the couple chose to conceive via in vitro fertilization, to have their embryos tested for the genetic defect, and to implant only disease-free ones.

Now, as they get ready to celebrate the first birthday of their healthy son Gus, and the arrival of his sibling conceived the same way, they know that theyve stopped adrenoleukodystrophy, or ALD, from traveling further down their family line.

But at the same time, theyve done something extraordinary for all families whose boys have ALD, whose men (like Brookes brother) have a less-severe form called AMN, or whose women and girls carry the genetic trait and might pass it on.

By donating the disease-affected embryos that they didnt want to a U-M Medical School lab, theyve made it possible for scientists to study ALD in its earliest stages.

The lab, called the MStem Cell Laboratories, derived embryonic stem cells from the embryo, and coaxed them to grow into nerve cells.

When genetically abnormal embryos would otherwise be discarded, families may donate them to research toward cures for diseases affecting their loved ones, says stem cell scientist Gary Smith, Ph.D., who directs the lab. Disease-specific human embryonic stem cells are the gold standard for research the purest pathway to understanding disease establishment and progression, and to discovering ways to prevent or alleviate pain and suffering caused by these diseases.

Scientists at U-M and around the world are now using the nerve cells developed from the ALD-carrying stem cells. Each nerve cell, or neuron, carries the ALD gene defect which lets scientists like U-M neurologist John Fink, M.D., study how the genetic abnormality affects nerve cells as they grow.

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Stem Cell Success: One Couple's Effort to Protect Their Son From Fatal Nerve Disease Will Help Other Boys Too