The effect of human gene therapy for RPE65-associated Leber's congenital amaurosis on visual function: a systematic review and meta-analysis.
Xue WangChaofeng YuRadouil T TzekovYihua ZhuWensheng LiPublished in: Orphanet journal of rare diseases (2020)
Human gene therapy is a pioneering treatment option for RPE65-LCA. Although its efficacy appears to be limited to less than 2 yrs after treatment, it carries the potential for further improvement and prolongation of efficacy.