Wexner Medical Center among first in nation to administer new gene therapy for ALS

The Ohio State University Wexner Medical Center is one of the first nationwide to administer a targeted gene therapy for patients with a specific form of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

RNA modification ‘pivotal’ for protein linked to neurodegeneration in ALS

Scientists know that TDP-43, which helps regulate processing of RNA, may be responsible for the death of nerve cells in ALS and frontotemporal dementia. And a study suggests that a common modification to RNA, a methylation event known as m6A, plays a pivotal role in TDP-43-related neurodegeneration in ALS. Through sequencing analysis, investigators showed that methylation strongly influences the binding of TDP-43 to its RNA targets. They also observed highly abundant RNA methylation in the end-stage tissues of patients with ALS.