Figure 4.
Enzyme, substrate, and ALT levels for subject 6 in study SB-913-1602 (MPS II)
Notes: ALT, alanine aminotransferase (normal: 7–55 U/L); ERT, enzyme replacement therapy; GAG, glycosaminoglycan; HS, heparan sulfate; I2S, iduronate-2-sulfatase; ZFN in vivo editing, zinc-finger nuclease-based in vivo genome editing. Numbers indicate days post-ERT administration. Maximum plasma ALT level is labeled relative to study day in red. Data on dermatan sulfate (DS) and total GAGs (T-GAG) are presented in Figure S3.