Gene Correction as Alternative to Gene Therapy

Researchers have demonstrated a new way to correct disease-causing mutations in DNA. Researchers have struggled to perfect gene therapy, in which they try to compensate for defective genes by inserting a new working copy into cells. Gene correction takes a different approach: repairing the mistakes in DNA that cripple the gene.

The team designed a zinc-finger nuclease that recognizes the mutation in SCID, the "bubble boy" syndrome, and showed that the nuclease prompted correction of the defect in a high frequency of T cells - the cells affected by the disorder.

The study offers the prospect that zinc-finger nucleases could be engineered against any disease-causing mutation in the human genome, and used to treat the disease.