Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration.
Level 4 - case-series / case-control
Case report describing autologous stem-cell transplantation in a single patient
PubMed 28296613 · doi:10.1056/NEJMoa1608368
What was done
Researchers assessed the feasibility of transplanting an autologous induced pluripotent stem cell (iPSC)-derived retinal pigment epithelial (RPE) cell sheet in a patient with advanced neovascular age-related macular degeneration. Skin fibroblasts from two patients were reprogrammed into iPSCs and differentiated into RPE cells. One patient underwent subretinal transplantation of the autologous RPE sheet following surgical excision of a neovascular membrane.
What was found
At 1 year post-transplantation, the graft remained intact. Best corrected visual acuity neither improved nor worsened, and cystoid macular edema was present. No other quantitative clinical metrics were reported in the abstract.
Why it matters
This report demonstrates the initial clinical feasibility and 1-year graft survival of an autologous iPSC-derived tissue sheet in human ocular disease.
Limits
This is a single-patient report (n=1) with no comparator or control. Visual acuity did not improve, cystoid macular edema persisted postoperatively, and safety or efficacy cannot be generalized.
Cited by
- supports Clinical trials in Japan have started using induced pluripotent stem cell technology derived from Yamanaka factors in human patients.