CRISPR Breakthrough Could Rewrite Future of Genetic Disease Treatment

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly developed form of CRISPR at UNSW Sydney points to a safer way of treating ...
eLife

Translational reading frame determines the pathogenicity of C-terminal frameshift deletions in MeCP2: an alternative therapeutic approach

This study offers important insight into the pathogenic basis of intragenic frameshift deletions in the carboxy-terminal domain of MECP2, which account for some Rett syndrome cases, yet similar ...