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Hope for Huntington’s Sufferers
SOURCE: TEEN.GWNEWS.COM
JAN 11, 2025
Gene therapies can help alleviate or even cure physical conditions such as sickle cell disease, leukemia, and others. But until recently, no effective treatment existed for Huntington’s disease, a grim and eventually fatal genetic disorder. Now a new therapy shows hope for slowing the ravages of Huntington’s.
When God created the world, He gave all living things a genetic code. Genes contain instructions for building proteins. In turn, those proteins form muscle fibers, hair, skin, and more. They also control body functions. Today, when problems occur in a person’s genetic code, scientists can sometimes add to, silence, or edit that code to address the problems. The ability to alter the genetic code in a living human has been possible only since the early 1990s. (Read about another gene editing breakthrough here.)
Huntington’s disease (HD) is a rare, inherited brain disorder caused by a mutation in the genetic code. Over time, the mutation produces a toxic protein that causes the brain’s nerve cells to stop working. HD sufferers have difficulty controlling muscle movements. They may also experience emotional, personality, and mental problems. Symptoms worsen over time as brain cells keep dying.
Researchers have worked on HD treatments for years. The National Library of Medicine estimates scientists have conducted more than 100 trials. Certain drug treatments have shown promise in easing some HD symptoms. However, none has seemed to affect the disease itself.
Until now.
Researchers from gene-therapy company uniQure developed an experimental medication called AMT-130. The treatment involves a “safe virus” injected into a subject’s brain. The virus helps cells stop making the toxic protein. Trial patients who received AMT-130 experienced a significant slowdown of Huntington’s.
Professor Sarah Tabrizi is Director of the University College London Huntington’s Disease Center. She worked on the AMT-130 trial. She says data from early testing seems to show “the potential to meaningfully slow disease progression.”
The new therapy doesn’t claim to cure HD—yet. But humanly speaking, AMT-130 could give people with HD extra years with much better quality of life. Plus, there is hope that, given to those with the inherited gene for HD, treatment might delay—or even stop—Huntington’s before symptoms arise.
God graciously allows humans to help alleviate suffering for other humans. But He alone determines the length of a life. “In your book were written, . . . the days that were formed for me, when as yet there was none of them.” (Psalm 139:16)
Professor Ed Wild was also involved in the AMT-130 trial. He told medical internet site HDBuzz, “Today we get to move Huntington’s disease into the column headed ‘treatable.’”
Thank you, gracious God!
Why? God makes no mistakes. It’s often hard to see purpose in suffering, but we can trust that the all-wise, all-powerful, and merciful Creator is working all things for our good and His glory.
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