Title |
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients
|
---|---|
Published in |
BioMedical Engineering OnLine, July 2012
|
DOI | 10.1186/1475-925x-11-40 |
Pubmed ID | |
Authors |
Paolo Melillo, Leandro Pecchia, Francesco Testa, Settimio Rossi, Jean Bennett, Francesca Simonelli |
Abstract |
Objective techniques to assess the amelioration of vision in patients with impaired visual function are needed to standardize efficacy assessment in gene therapy trials for ocular diseases. Pupillometry has been investigated in several diseases in order to provide objective information about the visual reflex pathway and has been adopted to quantify visual impairment in patients with Leber Congenital Amaurosis (LCA). In this paper, we describe detailed methods of pupillometric analysis and a case study on three Italian patients affected by Leber Congenital Amaurosis (LCA) involved in a gene therapy clinical trial at two follow-up time-points: 1 year and 3 years after therapy administration. |
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