Targeted gene repair – in the arena

Targeted gene repair -- in the arena.

The development of targeted gene repair is under way and, despite some setbacks, shows promise as an alternative form of gene therapy. This approach uses synthetic DNA molecules to activate and direct the cell's inherent DNA repair systems to correct inborn errors. The progress of this technique and its therapeutic potential are discussed in relation to the treatment of genetic diseases.

The Role of Gene Therapy in Cartilage Repair

The key principle of gene delivery to articulations by direct intra-articular injection is to release complementary DNA(cDNA)-encoding medical products that will lead to maintained, endogenous production of the gene products withinthe articulation. In fact, this has been accomplished for both in vivo and ex vivo gene delivery, using several vectors,genes, and cells in some animal models. Some c...

Epigenetic Modification of the Repair Donor Regulates Targeted Gene Correction

Optimizing design of vectors is critical to effective gene therapy. In targeted gene correction (TGC), cleavage of chromosomal DNA near a mutation stimulates homology-directed repair of a target gene using a donor provided in trans. We have systematically addressed epigenetic parameters of donor design, using a flow-based assay to quantify correction frequencies and expression levels of a green...

The Arena: December 1966: Arena

The Arena: December 1965: Arena