Under the control of MRI
Early-stage Gene therapy may Slow down Huntington's Disease
Margarita Romanova, 1nsk.ru
In a new study in mice, Johns Hopkins Medicine researchers report that using MRI scans to measure blood volume in the brain could serve as a non-invasive way to potentially track progress in gene editing therapy for early-stage Huntington's disease, a neurodegenerative disorder that attacks brain cells. The researchers say that identifying and treating the mutation known to cause Huntington's disease with this type of gene therapy, before the patient begins to show symptoms, can slow down the progression of the disease.
Article by Liu et al. Huntingtin silencing delays onset and slows progression of Huntington's disease: a biomarker study published in the journal Brain – VM.
"What's exciting about this study is the ability to identify a reliable biomarker that can track the potential success of genetic therapy before patients start showing symptoms," says Wenzhen Duan, MD, director of the translational neuroscience laboratory and professor of psychiatry and behavioral sciences at Johns Hopkins University School of Medicine. "Such a biomarker can facilitate the development of new treatments and help us determine the best time to start them."
For the study, Duan and her team collaborated with colleagues from the Kennedy Krieger Institute in Baltimore, Maryland, who developed a new method for more accurate measurement of blood volume in the brain using advanced functional MRI scanning. When scanned, they can display the trajectory of blood flow in small blood vessels, so-called arterioles, in the brains of mice designed to carry mutations of the human huntingtin gene that mimic the early stages of Huntington's disease in humans.
Typical maps of cerebral blood volume in the brains of wild-type mice (control) and three different models of Huntington's disease at the age of 3 months. The top row shows raw images – red areas indicate a quantitatively defined part of the brain. The bottom row shows typical maps of cerebral blood volume after gene therapy. Scale rulers are shown on the right. A warmer color means higher values of cerebral blood volume. Figure from the press release of Gene Editing Therapy in Early Stages of Huntington's Disease May Slow Down Symptom Progression, Mouse Study Shows – VM.
Duan notes that there are many known metabolic changes in the brains of people with Huntington's disease, and these changes initiate a blood volume response in the brain in the early stages of the disease. Blood volume is a key marker of the supply of oxygen to brain cells, which, in turn, supplies neurons with energy for their functioning. But with Huntington's disease, the volume of arteriolar blood of the brain decreases sharply, which leads to a deterioration of the state of neurons due to lack of oxygen as the disease progresses.
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