A phase I clinical trial of human embryonic stem cell-derived retinal pigment epithelial cells for early-stage Stargardt macular degeneration: 5-years' follow-up.

ABSTRACT:

Objectives

To evaluate the long-term biosafety and efficacy of transplantation of human embryonic stem cells-derived retinal pigment epithelial (hESC-RPE) cells in early-stage of Stargardt macular degeneration (STGD1).

Materials and methods

Seven patients participated in this prospective clinical study, where they underwent a single subretinal transplantation of 1 × 10⁵ hESC-RPE cells in one eye, whereas the fellow eye served as control. These patients were reassessed for a 60-month follow-up through systemic and ophthalmic examinations.

Results

None of the patients experienced adverse reactions systemically or locally, except for two who had transiently high intraocular pressure post-operation. Functional assessments demonstrated that all of the seven oper

SUBMITTER: Li SY 

PROVIDER: S-EPMC8450131 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature