AI Article Synopsis

  • - Age-related macular degeneration (AMD) causes blindness primarily due to the dysfunction of retinal pigment epithelium (RPE), leading researchers to create an RPE patch from human embryonic stem cells (hESC).
  • - The patch was successfully delivered into the eyes of two patients with severe AMD, with the study focusing on any adverse events and significant improvements in their vision.
  • - Both patients experienced substantial visual acuity gains after 12 months, showing the potential safety and effectiveness of the hESC-RPE patch as a treatment option for AMD.

Article Abstract

Age-related macular degeneration (AMD) remains a major cause of blindness, with dysfunction and loss of retinal pigment epithelium (RPE) central to disease progression. We engineered an RPE patch comprising a fully differentiated, human embryonic stem cell (hESC)-derived RPE monolayer on a coated, synthetic basement membrane. We delivered the patch, using a purpose-designed microsurgical tool, into the subretinal space of one eye in each of two patients with severe exudative AMD. Primary endpoints were incidence and severity of adverse events and proportion of subjects with improved best-corrected visual acuity of 15 letters or more. We report successful delivery and survival of the RPE patch by biomicroscopy and optical coherence tomography, and a visual acuity gain of 29 and 21 letters in the two patients, respectively, over 12 months. Only local immunosuppression was used long-term. We also present the preclinical surgical, cell safety and tumorigenicity studies leading to trial approval. This work supports the feasibility and safety of hESC-RPE patch transplantation as a regenerative strategy for AMD.

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Source
http://dx.doi.org/10.1038/nbt.4114DOI Listing

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