Transplantation of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in Macular Degeneration.

ABSTRACT:

Purpose

Transplantation of human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells offers the potential for benefit in macular degeneration. Previous trials have reported improved visual acuity (VA), but lacked detailed analysis of retinal structure and function in the treated area.

Design

Phase 1/2 open-label dose-escalation trial to evaluate safety and potential efficacy (clinicaltrials.gov identifier, NCT01469832).

Participants

Twelve participants with advanced Stargardt disease (STGD1), the most common cause of macular degeneration in children and young adults.

Methods

Subretinal transplantation of up to 200 000 hESC-derived RPE cells with systemic immunosuppressive therapy for 13 weeks.

Main outcome measures

The primary end p

SUBMITTER: Mehat MS 

PROVIDER: S-EPMC6195794 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature