conclusion and impact of the study

The conclusion of the study is that the method of AAV9-PHPB delivery and CRISPR/Cas9 treatment combination resulted in the most effective disruption of the mutant gene, indicating that this method of the treatment may be the most effective and is likely to be successful in human studies. The study also indicates that the treatment was done successfully in a human cell, with high efficiency using the CRISPR/CAS9 treatment. The researchers also tested the method in mice, which indicates that not only does the treatment works on human cells, but it also works at a larger scale and is unlikely to affect other organ systems.  Specifically, of all of the treatments and delivery, it has been noted by the scientists that the mice that were given treatment through an injection into the eye, had a higher level of damage compared to the untreated eye, but had less damage compared to other methods of delivering the AAV virus. By indicating that a method of treatment that has worked in the human cell and mice. If the treatment has worked in both human cells and mice it is an indication that it is also likely to work in a human patient. Using data that is published in this paper it would be possible to go to human trial, which if it works, will be able to have permanent treatment for the retinitis prigmentosa, which takes away the sight of those that are affected. 

https://www.ncbi.nlm.nih.gov/pubmed/29281027