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On- and Off-Target Activity of M2PCSK9 in NHPs
Rhesus macaques received AAV at the indicated doses. We performed liver biopsies at 18 and 128 days (d18 and d128) post-injection. (A) Indel% in M2PCSK9 target region in the rhesus PCSK9 gene calculated by AMP-seq. (B) Number of ITR-seq-identified off-targets. Results for d18 (gray bars) and d128 (black bars) liver biopsies are indicated for each NHP.
