The Stem Cell Medicine team at Children’s Medical Research Institute (CMRI) have been involved in a new publication in the latest edition of Cell Reports which gives great hope of a new therapy that could restore vision to patients with some genetic eye diseases.
Stem Cell Medicine Group Leader, Dr Anai Gonzalez Cordero, was involved in the research along with two of her team members, Michelle O’Hara-Wright, and Milan Fernando. It was led by Professor Robin Ali from the University College London Institute of Ophthalmology, where Dr Gonzalez Cordero worked before moving to CMRI to establish her own lab.
“After years of research and proof-of-concept studies, this important study shows that cell transplantation of human stem cell-derived photoreceptor cells is indeed a feasible treatment option to rescue vision,’’ Dr Gonzalez Cordero said.
Macular disease and degeneration results in the loss of light-sensing cone photoreceptors, causing irreversible sight impairment. Current approaches are limited to slowing the progression of the disease, which means that many patients with advanced disease symptoms have no treatment options.
Photoreceptor replacement may restore vision by transplanting healthy cells, which must form new synaptic connections with the recipient retina.
Despite recent advances, convincing evidence of functional connectivity arising from transplanted human cone photoreceptors in advanced retinal degeneration is lacking. In this publication, the authors show restoration of visual function after transplantation of purified human pluripotent stem cell (hPSC)-derived cones into models with advanced degeneration.
“Of particular importance is the ability to transplant human cone photoreceptors to treat the macula, the region we are most dependent upon for cone-mediated, high-acuity vision,’’ the authors wrote. “Here, we provide definitive evidence of rescue of both retinal and visual function following the transplantation of a purified population of hPSC-derived cone photoreceptors into a model of advanced retinal degeneration.
“Electrophysiological and behavioural assessments demonstrate restoration of surprisingly complex light-evoked retinal ganglion cell responses and improved light-evoked behaviours.
“These data provide crucial validation for photoreceptor replacement therapy and for the potential to rescue cone-mediated vision.’’
Dr Gonzalez Cordero and her team have been responsible for developing protocols for generating large numbers of purified human pluripotent stem cells that can be used for this research.