Abstract
Retinal degenerations are a major cause of vision impairment and blindness. Neuroprotective therapy is a promising therapeutic strategy for retinal degenerative diseases. We investigated a novel neurotrophic factor mesencephalic astrocyte-derived neurotrophic factor (MANF) in the retina. MANF is expressed at a high level during postnatal development and the expression declines to a lower level as the retina matures. Müller cells are the major cells expressing MANF. It is also found in the retinal ganglion cells, in the inner nuclear layer (INL) neurons, and in retinal pigment epithelial (RPE) cells. Intravitreal injection of recombinant human (rh)MANF significantly protected rod and cone photoreceptors in rats carrying the rhodopsin S334ter mutation, and preserved electroretinograms (ERGs) in the rd10 (Pde6brd10/rd10) mice. These results indicate that MANF is a native protein in the retina and is a potent neurotrophic factor for photoreceptor protection.
Footnotes
R.W. has been named as the inventor in a patent application filed by the University of Miami that covers the intellectual property presented in this paper. All other authors declare no competing financial interests.
This work was supported by National Institutes of Health (NIH) Grants R01EY015289 and R01EY018586, Hope for Vision, the James and Esther King Biomedical Research Program of the State of Florida Grant 08KN-09, 2KF02, National Natural Science Foundation of China Grants 81560164 and 81300737, NIH Core Grants P30EY14801 and P30EY002162, and an unrestricted grant from Research to Prevent Blindness Inc. to Bascom Palmer Eye Institute. R.W. is the recipient of the 2015 Nelson Trust Award for Retinitis Pigmentosa from Research to Prevent Blindness, Inc.
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