efficient allele-specific targeting of lrrk2 r1441 mutations mediated by rnai高效allele-specific体内基因lrrk2的目标由rnai r1441突变.pdfVIP

Efficient Allele-Specific Targeting of LRRK2 R1441 Mutations Mediated by RNAi ˜ ´ ´ Laura de Ynigo-Mojado, Itziar Martın-Ruız, James D. Sutherland* Proteomics Unit, CIC bioGUNE, Derio, Bizkaia, Spain Abstract Since RNA interference (RNAi) has the potential to discriminate between single nucleotide changes, there is growing interest in the use of RNAi as a promising therapeutical approach to target dominant disease-associated alleles. Mutations in th