I believe the next step is optic discs in a dish( an organoid) , and our ASO FPP will be added around this time with the effects known soon for Retinitis Pigmentosa
The interesting thing is that these optic discs ( with all the layers of the retina) are being created ( takes a while and is tricky) by the individuals stem cells ( from fibroplasts) that have the actual disease the new drug is for, hence removing some of the issues that might have got in the way later.
I found this explanation re organoids in general ...
Imagine the possibility of creating individualized, complex collections of cells that bear a resemblance to a patient’s own tissues. That technology—the ability to grow ‘organoids’—is becoming a reality and is finding new uses every day, in part thanks to the work of scientists at the Harvard Stem Cell Institute.
Organoids are tiny, self-organized three-dimensional tissue cultures that are derived from stem cells. Such cultures can be crafted to replicate much of the complexity of an organ, or to express selected aspects of it like producing only certain types of cells.
Organoids grow from stem cells—cells that can divide indefinitely and produce different types of cells as part of their progeny. Scientists have learned how to create the right environment for the stem cells so they can follow their own genetic instructions to self-organize, forming tiny structures that resemble miniature organs composed of many cell types. Organoids can range in size from less than the width of a hair to five millimeters.
Certainly sounding very hopeful, no missteps to date. Chart looks good too. ( can some one please chart one?)
(20min delay)