Figure 1.

Gene Therapy Strategies for Autosomal Dominant Diseases

Top: in autosomal dominant disorders such as RHO-adRP, one mutated copy of gene encodes the abnormal mRNA and protein (red) sufficient to cause disease. The wild-type copy is shown in green. Middle: allele-specific therapeutics target the mutated RHO gene or its mRNA product without affecting the wild-type copy. Bottom: mutation-independent therapeutics disrupt both the mutated and wild-type RHO genes or their RNA products and replace them with an exogenous copy of the codon-modified CRISPR or RNAi resistant RHO gene (blue).