Long-term safety of human retinal progenitor cell transplantation in retinitis pigmentosa patients.

ABSTRACT:

Background

Retinitis pigmentosa is a common genetic disease that causes retinal degeneration and blindness for which there is currently no curable treatment available. Vision preservation was observed in retinitis pigmentosa animal models after retinal stem cell transplantation. However, long-term safety studies and visual assessment have not been thoroughly tested in retinitis pigmentosa patients.

Methods

In our pre-clinical study, purified human fetal-derived retinal progenitor cells (RPCs) were transplanted into the diseased retina of Royal College of Surgeons (RCS) rats, a model of retinal degeneration. Based on these results, we conducted a phase I clinical trial to establish the safety and tolerability of transplantation of RPCs in eight patients with advanced retiniti

SUBMITTER: Liu Y 

PROVIDER: S-EPMC5622579 | biostudies-literature | 2017 Sep

REPOSITORIES: biostudies-literature