Kamaljit S. Balaggan

TL;DR: Three young adult patients with early-onset, severe retinal dystrophy were administered subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the control of a human R PE65 promoter.

TL;DR: In dogs, RPE65 gene therapy with the same vector at lower doses improved vision-guided behavior, but only higher doses resulted in improvements in retinal function that were detectable with the use of ERG, and comparison with the results obtained in the dog model indicates that there is a species difference in the amount of R PE65 required to drive the visual cycle.

TL;DR: The high efficiency of gene transfer and expression mediated by lentiviruses can be harnessed in vivo without a requirement for vector integration and substantially reduces the risk of insertional mutagenesis.

TL;DR: A model to define genomic access to the viral integration site that provides optimal restriction motif combinations and minimizes the percentage of nonaccessible insertion loci is developed and a new nonrestrictive LAM-PCR approach is introduced.

TL;DR: Autologous RPE transplantation can in principle restore vision in neovascular AMD, but surgical complications remain high.