This technology is a collection of optimized therapeutic prime-editing strategies targeting four common splicing mutations in the peripherin‐2 gene (PRPH2) that are associated with various retinal degenerations.
This technology is a gene therapy platform to reactivate two transcriptional factors, Islet1 and Lhx3, for increased resilience against ALS degeneration in motor neurons.
This technology is a modified CRISPR/Cas9 gene editing therapy that is mutation-independent for treating autosomal dominant disease-related genes, including retinitis pigmentosa.