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The use of BMSC in the Stem Cell Ophthalmology Treatment Study achieved meaningful visual improvements in a significant percentage of the NAION patients reported. Improvements typically manifested no later than 6 months post-procedure. Duration of visual loss did not appear to affect the ability of the eyes to respond to treatment. Possible mechanisms by which visual improvement occurred may include BMSC paracrine secretion of proteins and hormones, transfer of mitochondria, release of messenger RNA or other compounds via exosomes or microvesicles and neuronal transdifferentiation of the stem cells.

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The BMSC protocols of the SCOTS achieved meaningful visual acuity improvements or stability in RP that were of statistical significance. Duration of disease did not appear to affect the ability of eyes to respond. Safety was confirmed. Possible mechanisms by which improvement occurred may include transdifferentiation of BMSC into Neuronal Nuclei (NeuN) positive cells, BMSC paracrine secretions or neurotrophic factors and hormones, transfer of mitochondria, release of messenger RNA or other compounds via exosomes or microvesicles. Given the successful outcome in this otherwise progressive condition, consideration should be given to providing this treatment option.