Developing a treatment for vision loss through transplant of
photoreceptor precursors

Date:
October 18, 2021
Source:
National University of Singapore, Yong Loo Lin School of Medicine
Summary:
A recent study examining the therapeutic potential of photoreceptor
precursors, derived from clinically compliant induced pluripotent
stem cells (iPSC), has demonstrated the safety and therapeutic
potential of clinically compliant iPSC-derived photoreceptor
precursors as a cell replacement source for future clinical trials.



FULL STORY ========================================================================== Inherited retinal diseases (IRDs) are a group of genetically and
clinically homogeneous illnesses characterised by progressive retinal
damage leading to vision loss. The global incidence of IRDs is
approximately 1 in 2000 persons.

These disorders are amongst some of the leading causes of blindness
worldwide.


========================================================================== While the introduction of gene therapy has been a significant development,
its effectiveness has been blunted by the sheer extent of genetic heterogeneity, with more than 260 genes implicated in IRDs. This limits
the widespread application of gene therapy for all IRDs. Additionally,
gene therapy has limited efficacy in clinical cases of advanced retinal degeneration in which significant photoreceptor cell death has already occurred. Photoreceptor cells are found in the retina and respond to
light, converting it into electrical signals that activate physiological
chain reactions. These signals are sent through the optic nerve to the
brain for processing.

With the advent of induced pluripotent stem cell (iPSC) and embryonic stem
cell (ESC) technology, regenerative stem cell therapy has the potential
to be an alternative treatment for end-stage retinal degeneration,
independent of the underlying genetic defect. Retinal regenerative
therapies therefore hold great promise for the treatment of IRDs. Studies
in animal models of IRDs have suggested visual improvement following
retinal photoreceptor precursors transplantation, though there is limited evidence on the ability of these transplants to rescue retinal damage
in higher mammals.

A recently published study in the journal Stem Cell Research and Therapy
led by Assistant Professor Su Xinyi from the Department of Ophthalmology
at the NUS Yong Loo Lin School of Medicine examines the therapeutic
potential of photoreceptor precursors derived from clinically compliant
iPSCs. The study demonstrated the safety and therapeutic potential of clinically compliant iPSC- derived photoreceptor precursors as a cell replacement source for future clinical trials. These include performing
a first-in-man clinical trial for photoerecptor precursor transplant in Singapore, in collaboration with RxCELL, a biotechnology company focused
on therapeutic applications iPSCs.

========================================================================== Story Source: Materials provided by National_University_of_Singapore,_Yong_Loo_Lin_School_of Medicine. Note: Content may be edited for style and length.


========================================================================== Journal Reference:
1. Zengping Liu, Tanja Ilmarinen, Gavin S. W. Tan, Heidi Hongisto,
Edmund Y.

M. Wong, Andrew S. H. Tsai, Sami Al-Nawaiseh, Graham E. Holder,
Xinyi Su, Veluchamy Amutha Barathi, Heli Skottman, Boris
V. Stanzel. Submacular integration of hESC-RPE monolayer xenografts
in a surgical non-human primate model. Stem Cell Research & Therapy,
2021; 12 (1) DOI: 10.1186/ s13287-021-02395-6 ==========================================================================

Link to news story: https://www.sciencedaily.com/releases/2021/10/211018105930.htm

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