Gene transfer technology offers new opportunities
Gene-based technology company Oxford BioMedica and researchers at Imperial College of Science and Technology, London, have announced that they have achieved very efficient gene transfer to mouse embryos in utero. If the technology is reproducible in man, it will create the potential to cure diseases such as Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF) that are incurable at present.
One in three thousand males inherit a non-functional dystrophin gene and suffer from DMD. The disease causes severe muscle weakness and affects every muscle in the body. In order to correct the disease in children or adults every muscle would need to receive a functional dystrophin gene. This is not feasible using many current gene therapy techniques because the tissue volume is too great.
A solution to this problem is to deliver the gene when the tissue volume is small, i.e. to the developing foetus in the womb. The Imperial College team, using BioMedica's LentiVector, has now shown that this is possible in animal models. Gene transfer has been shown to a wide range of tissues, including liver, brain and muscle, following administration of LentiVector to the foetal blood supply. If this were to be recapitulated in humans it would provide a potential route to treating several genetic deficiencies, according to Dr Mike Themis of the Gene Therapy Group, Division of Biomedical Sciences at Imperial College, who presented the developments to the American Society for Gene Therapy last month.
'In utero, gene therapy opens many exciting opportunities to treat people with these severely debilitating and fatal diseases before suffering occurs,' he said. 'We hope that one day this may be offered to parents as an alternative to termination of pregnancy. The efficiency of the gene transfer that we see in animals enables us to study the function of genes in a way not possible before. This may accelerate our understanding of a broad range of diseases and create new therapeutic strategies.'