A list of gene editing trial(s) currently underway that may potentially benefit patients with Usher syndrome. Please refer to the link under each trial for more information on that trial. Please be aware that all information provided are for informational purposes only, and are in no way endorsed or approved by Usher Syndrome Ireland.
Pre-Clinical Development Stage
Editas Medicine recently made headlines around the world with their first ever in-human gene editing for a different inherited retinal disease, LCA. They are currently developing several programs, one of which is the EDIT-102 for Usher syndrome 2a (USH2A).
“Mutations in exon 13 of the USH2A gene are most often responsible for the development of USH2A. This information supports the design of a CRISPR medicine that edits exon 13 to restore healthy USH2A protein expression, consequently restoring the function of photoreceptors.”
More information can be found here: https://www.editasmedicine.com/gene-editing-pipeline/