Date Posted: 10 September 2009
Recent advances in retinal gene therapy, notably in three concurrent human trials addressing LCA (Leber's congenital amurosis), have focused significant attention on the wider potential to apply such technologies to other diseases, both within and beyond the field of ophthalmology. The use of modified viruses to act as the delivery vector for a variety of gene therapies continues to represent one of the most efficient means of transferring DNA into fully differentiated neurons.
While the field has weathered several storms over the last decade the travails should hopefully yield a safer and more efficacious strategy to treating a range of neurological diseases. Recent electron microscopy and protein studies in dogs and primates that had received injections of the adeno-associated viral vector (AAV) have shown that the viral coat appears to persist for up to 6 years in the retina. The study, led by Prof. Fabienne Rolling of INSERM in Nantes, showed the presence of intact viral particles up to 6 years after a single sub-retinal injection. The authors argue that the finding is in "striking contrast" to the presumed degradation of capsid proteins by the cell's proteasomal machinery.
In discussing their report the authors highlight that their findings represent the first such identification of AAV persistence following efficient gene transfer and suggest that the discovery may have important consequences for current clinical trials. Prof. Rolling concludes by advising that the current UK and US trials in LCA should consider evaluating the findings within their experimental protocols and follow-up studies. The research will likely attract significant attention from professionals involved in the regulation of such novel therapeutic advances. [Steiger et al, Molecular Therapy, 2009, Vol. 17, No. 3, 516-523].
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