Misfolding Proteins, MisLede-ing Headlines?

On March 14th, the Guardian reported on a recent advance using gene transfer against a degenerative brain condition called spinalcerebellar ataxia.  The condition belongs to a class of diseases that also includes Huntington's disease, and as the report indicates, the study could prove useful for many other degenerative brain disorders.

The lede begins "Scientists are a step closer to curing a severe inherited brain-wasting disease using gene therapy."  Without diminishing the value of the study, readers should interpret the story with caution.  Among the limitations of studies like these, two stand out.  First, perhaps more than any single class of diseases, those involving neurodegenerative diseases have the highest failure rate in terms of preclinical findings translating into clinical applications.  Second, most brain degenerative diseases involve multiple brain regions.  But most gene transfer strategies, while thinking globally, target locally.  As such, they seem unlikely to halt progression of disease in regions of the brain that are not specifically targeted. (photo credit: moon rhythm 2006)