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Gene therapy for RPE65‐related Leber congenital amaurosis
Author(s) -
LEROY BP
Publication year - 2014
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2014.3762.x
Subject(s) - rpe65 , medicine , genetic enhancement , cis trans isomerases , clinical trial , disease , amaurosis , retinal , pediatrics , ophthalmology , optometry , gene , genetics , retinal pigment epithelium , biology , peptidylprolyl isomerase , isomerase
Purpose The talk will focus on an overview of gene therapy trials for RPE65‐related Leber congenital amaurosis (LCA), which are currently ongoing or have been finalised. Methods Systematic review of trials. Results Currently there are 6 trials around the World focusing on gene therapy for RPE65‐related Leber congenital amaurosis, with good safety outcomes and considerable success in restoring some visual function. So far, no safety issues have been encountered in these trials, whereas some improvement and/or stabilization of retinal function is seen. However, progression of disease is not halted entirely. Conclusions Gene therapy trials for RPE65‐related Leber congenital amaurosis are safe and somewhat successful in restoring and/or stabilizing retinal function, despite early indications of further progression of disease.