This month, there was fanfare about the first case of usingCRISPR invivo to treat Leber’s congenital amaurosis 10(LCA10) which is a leading cause of blindness in childhood. This isdue to mutations in a large gene,CEP290.
The article comments that:
“For the latest trial, the components of the gene-editingsystem – encoded in the genome of a virus — are injected directlyinto the eye, near photoreceptor cells.â€
A. Specifically, what components are the authors referring towhen they say “the components of the gene-editing system – encodedin the genome of a virus�
B. How is the zinc finger technology similar to the CRISPRapproach?
C. What is a potential risk of using the CRISPR technologyinvivo?
D. If there was a suitable safe harbor, or the possibility touse a AAV virus to replace the mutant CEP290 gene in thephotoreceptor cells (Similar to replacing the CNG3B channel usingAAV5). What would be the potential difficulties compared to fixingthe gene with CRISPR?
E. If the mutation in the CEP290 gene was known to be apremature stop codon, would the gene therapy be the safest approachto treat this disease?
