January 10, 2017

expert reaction to stem cell therapy in blind mice with end-stage retinal degeneration

Publishing in Stem Cell Reports, a group of researchers have reported that retinal tissue derived from mouse induced pluriopotent stem cells (iPSCs) established connections with neighbouring cells and responded to light stimulation, restoring visual fuction in half of mice with end-stage retinal degeneration.

Prof. Robert MacLaren, Professor of Ophthalmology, University of Oxford, said:

“This is a very nice study which shows how stem cells might in future be applied to treat blindness caused by retinal degeneration.  Here the researchers make stem cells, not from embryos, but from adult mice.  They then grow these stem cells into small patches of light-sensitive retina which are then transplanted into the eyes of mice which are blind from retinal degeneration.  The mice can see to a limited extent with these transplanted cells.

“This study is exciting because it shows that a complicated part of central nervous system, such as the retina, could potentially be regenerated from something as simple as a skin cell.  Furthermore, the engineered retinal tissue appears to be able to make connections to the brain after transplantation.  Clinical treatments are still a long way off, but for patients with retinal degeneration, this provides some hope for the future.”

Dr Dusko Ilic, Reader in Stem Cell Science, King’s College London, said:

“This beautiful and well executed proof of concept study demonstrates in a mouse model how the host cells interact directly with transplanted cells, forming synapses and transmitting signals.  This study strongly supports hESC/iPSC-based cellular therapy of retinal degeneration disorders.  There are ongoing clinical trials for age-related macular degeneration using both hESC (human embryonic stem cells) and iPSC (induced pluripotent stem cells) in the USA, UK, Japan and Israel – in those trials people use hESC/iPSC-derived retinal pigment epithelium; whereas in this new paper the authors used a different cell type present in retina.”

* ‘iPSC-derived retina transplants improve vision in rd1 end-stage retinal-degeneration mice’ by Michiko Mandai et al. will be published in Stem Cell Reports at 17:00 UK time on Tuesday 10 January 2017, which is also when the embargo will lift. 

Declared interests

Prof. Robert MacLaren: “I have no relevant conflicts of interest to declare.”

Dr Dusko Ilic: “I declare no conflict of interest.”