Self-assembling materials with the potential to become safe and efficient gene delivery agents have been designed by French scientists. 

In gene therapy, a normal gene is inserted into the genome to replace an abnormal disease-causing gene - a process called transfection.  A carrier molecule called a vector delivers the therapeutic gene to the target cells. So far the most efficient vectors are viruses, which have an innate ability to introduce their genetic material into host cells.  But the potential difficulties in using viruses in this way, such as toxicity and unwanted immune responses, have hindered progress.

Synthetic gene delivery systems may offer a safer alternative.  Bernard Pucci and Ange Polidori at Université D'Avignon and colleagues have made new amphiphilic molecules known as bolaamphiphiles that have two polar head groups linked together by hydrocarbon and fluorocarbon segments.  One of the head groups on the molecule can interact with DNA to form complexes dubbed bolaplexes.  

The researchers have shown that these bolaplexes present significant in vitro transfection efficiency combined with low cytotoxicity, indicating a strong potential for application in DNA delivery systems.

Pucci emphasised the importance of research into synthetic vectors for gene delivery. 'Gene therapy will be able to be developed only if the pharmacologists can have a good DNA carrier' he said.

Nicola E Nugent