Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia.

Retinal gene therapy is increasingly recognized as a novel molecular intervention that has huge potential in treating common causes of blindness, the majority of which have a genetic aetiology. Choroideremia is a chronic X-linked retinal degeneration that was first described in 1872. It leads to progressive blindness due to deficiency of Rab-escort protein 1 (REP1).

Vector Shedding and Immunogenicity Sampling for Retinal Gene Therapy.

There has been recent growth in the number and magnitude of clinical trials for various forms of retinal gene therapy. Because of regulatory requirements, and to better understand vector safety profiles, there is a need for standardised and effective methods to collect, process, and store biological samples taken from trial patients that can be used to assess the dissemination of the vector within bodily fluids and any systemic cellular and humoral immune responses.

Intravitreal pegaptanib for the treatment of ischemic diabetic macular edema.

Purpose: Pegaptanib has been shown to be effective in treating diabetic macular edema (DME). In the original Phase II/III trial, however, patients with macular ischemia were excluded. In this study, we treated patients with ischemic DME.