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.

Today, thanks to the groundbreaking research led by U-M’s Dr. Michelle Hastings, Lori Isom, Arul Chinnaiyan and the nearly 170 RNA scientists and clinicians of the Center for RNA Biomedicine, we are rewriting the outcomes for children like these girls.

Amelia and Makenzie began treatment in 2024 with an antisense oligonucleotide (ASO) therapeutic developed by Dr. Hastings and her team, 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. 

And what’s more, this type of therapy can be used to treat a wide range of genetic diseases. 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. Join us in taking the next life-changing steps to bring RNA Therapeutics to Michigan. Your involvement can make all the difference.

Visit the link below and help turn the dream into a reality for so many.

For more information visit: rna.umich.edu

rna.umich.edu