UIC Applied Health Sciences MAGAZINE
100 years of biomedical visualization
The UIC Program in Biomedical Visualization celebrates a century at the intersection of art, life science, Huntingtin-lowering therapy medicine and technology
IONIS-HTTRx
Huntington’s disease is a hereditary neurodegenerative disease that impacts the striatum, or motor center, of the brains of individuals with a mutation in the Huntingtin gene. Currently, there is no cure. Ionis Pharmaceutical’s antisense oligonucleotide therapy, by the name of Ionis-HTTRx, is the first targeted HTT-lowering drug tested in humans. Phase 1/2 results recently demonstrated safety and tolerability of the drug in humans and proved successful in lowering the amount of HTT protein. The next step is to prove a reduction in disease symptoms.
RNase is an enzyme that binds to and cleaves mRNA in RNA-ASO hybrids. Shown here is the RNase H1 domain responsible for this action. By initiating degradation of Huntingtin mRNA, HTTRx is capable over lowering the amount of mutant Huntingtin protein.
The mutant Huntingtin protein is responsible for damage to certain motor centers in the brain. This occurs because of an expanded CAG repeat region in the Huntingtin gene, which is carried through to mutant RNA and, finally, to mutant protein.
1. HTTRx ASO binds HTT RNA
SUMMER
2021
2. RNase binds RNA-ASO hybrid
Elizabeth Papautsky centers the patient experience and voice, including her own
In-kind gift from pioneering biomedical artist helps students learn from the past
