CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues.

looked at a risk variant of AMD in the C3 locus (rs2230199) and showed a trend towards elevated levels of the complement activation marker C3a [ 53 ]. rs2230199 is a C>G transversion; if specific knockdown of the pathogenic (G) allele by random indels (generated by active Cas9 targeting) is possible, then indeed this could be an ideal strategy.

also in 53,322 other papers