This Month in Genetics

The infantile form of spinal muscular atrophy (SMA) is a devastating condition with a median life expectancy of 2  years without ventilation. Essentially, no effective interventions have been available, but in a recent issue of The New England Journal of Medicine, back-to-back publications report clinical trials of two completely different therapies that both show promise for treating SMA. Mendell et al. repo rt the use of single-dose intravenous gene therapy for infants with SMA, whereas Finkel et al.
Source: The American Journal of Human Genetics - Category: Genetics & Stem Cells Authors: Tags: Editors' Corner Source Type: research