Center for RNA Biomedicine

CENTER FOR RNA BIOMEDICINE - GIFT ACCOUNT-330716

Meet Amelia and Makenzie, twin girls who were once full of energy and living normal lives. After several years of normal development, their world began to change drastically—they experienced vision loss, movement difficulties, and seizures. Doctors pinpointed the culprit: juvenile Batten disease, a fatal genetic disorder caused by a rare mutation.

A mere five years ago, this would have been a tragic story without hope. Doctors would have faced devastated parents with the unfortunate news that nothing more could be done.

Today, thanks to the groundbreaking research led by U-M’s Dr. Michelle Hastings, we are rewriting the outcomes for children like these girls. With the power of antisense oligonucleotides (ASOs), we can now target and correct the specific genetic mutation responsible for their disorder, offering renewed hope and a chance at a brighter future. 

And what’s more, this type of therapy can be used to treat a wide range of genetic diseases. 

Amelia and Makenzie began treatment in 2024, and the results have been promising. “We are enthusiastic about the trail we are blazing, which unlocks new possibilities for personalized RNA-based treatments,” says Dr. Hastings. 

Bringing this groundbreaking technology to the experts at Michigan is the next step in transforming hope into reality for as many people as possible. Your support will enable us to acquire cutting-edge equipment that is crucial in developing therapies for both rare and chronic diseases. And where better than at a premier research and medical institution like Michigan?

We have the right people, the expertise, and an urgent need. Join us in taking the next life-changing steps to bring RNA Therapeutics to Michigan. Your involvement can make all the difference.

For more information visit: rna.umich.edu