The long noncoding RNA MINCR, implicated in ALS and Alzheimer’s disease as well as several types of cancer, appears to function differently when present at high versus low levels.
After six months, patients with fast-progressing amyotrophic lateral sclerosis who had received the experimental treatment had less loss of function than those who received a placebo.
Researchers slowed disease progression in the mice by injecting two different viral vectors, each containing one part of the DNA encoding the Cas9 protein, to edit the causative gene.
Flexible proteins appear to protect molecules from becoming denatured in extreme conditions such as heat and from clumping up, as happens in some neurodegenerative diseases.
Boosting the levels of Akkermansia muciniphila in mouse guts slowed the progression of an ALS-like disease, while two other microbiome members were associated with more severe symptoms.
He developed an MRI-based map of the human cortex, discovered genetic risk factors for neurodegenerative diseases, and wrote about his struggles with ALS.
Human endogenous retroviruses that colonized vertebrate DNA millions of years ago have long been dismissed as junk DNA, but researchers now know that they may play important roles in cancer, neurodegeneration, and other ailments.
Clinical trials that target human endogenous retroviruses to treat multiple sclerosis, ALS, and other ailments are underway, but many questions remain about how these sequences may disrupt our biology.
At first blush, do-it-yourself clinical trials seem pointless and reckless. But a deeper truth pervades the research and the patients who drive it forward.
