Robin R. Ali

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: It is shown that donor cells can integrate into the adult or degenerating retina if they are taken from the developing retina at a time coincident with the peak of rod genesis, and the ontogenetic stage of donor cells for successful rod photoreceptor transplantation is defined.

TL;DR: Gene therapy for SCID-X1 is a highly effective strategy for restoration of functional cellular and humoral immunity in patients with X-linked severe combined immunodeficiency.

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.