Receptor-based gene delivery

A crucial problem for nucleic acid-based therapeutics is delivering a gene to the nucleus of a nondividing cell, where the cell's machinery can transcribe and translate its message into a protein, the step essential for the gene to have any therapeutic effect.

Viruses have mechanisms for accomplishing this, and many have been engineered as gene therapy vectors. However, as noted by Mark Cooper, senior vice president of science and medical affairs at Copernicus Therapeutics Inc., viral delivery has drawbacks, such as the potential for an engineered virus to become replication competent and, thereby, infectious. Other issues include manufacturing and the size of the gene that can be delivered...