Department of Microbiology and Immunology, McGill University, Montreal, QC H3A2B4, Canada.
Antimicrob Agents Chemother. 2011 Jun;55(6):2662-9. doi: 10.1128/AAC.01738-10. Epub 2011 Mar 14.
GS-9148 is an investigational phosphonate nucleotide analogue inhibitor of reverse transcriptase (RT) (NtRTI) of human immunodeficiency virus type 1 (HIV-1). This compound is an adenosine derivative with a 2',3'-dihydrofuran ring structure that contains a 2'-fluoro group. The resistance profile of GS-9148 is unique in that the inhibitor can select for the very rare Q151L mutation in HIV-1 RT as a pathway to resistance. Q151L is not stably selected by any of the approved nucleoside or nucleotide analogues; however, it may be a transient intermediate that leads to the related Q151M mutation, which confers resistance to multiple compounds that belong to this class of RT inhibitors. Here, we employed pre-steady-state kinetics to study the impact of Q151L on substrate and inhibitor binding and the catalytic rate of incorporation. Most importantly, we found that the Q151L mutant is unable to incorporate GS-9148 under single-turnover conditions. Interference experiments showed that the presence of GS-9148-diphosphate, i.e., the active form of the inhibitor, does not reduce the efficiency of incorporation for the natural counterpart. We therefore conclude that Q151L severely compromises binding of GS-9148-diphosphate to RT. This effect is highly specific, since we also demonstrate that another NtRTI, tenofovir, is incorporated with selectivity similar to that seen with wild-type RT. Incorporation assays with other related compounds and models based on the RT/DNA/GS-9148-diphosphate crystal structure suggest that the 2'-fluoro group of GS-9148 may cause steric hindrance with the side chain of the Q151L mutant.
GS-9148 是一种研究中的磷酸核苷酸类似物抑制剂,可抑制人类免疫缺陷病毒 1 型(HIV-1)的逆转录酶(RT)(NtRTI)。该化合物是一种腺苷衍生物,具有 2',3'-二氢呋喃环结构,其中包含一个 2'-氟基团。GS-9148 的耐药谱是独特的,因为抑制剂可以选择 HIV-1 RT 中非常罕见的 Q151L 突变作为耐药途径。Q151L 不能被任何已批准的核苷或核苷酸类似物稳定选择;然而,它可能是一种短暂的中间体,导致相关的 Q151M 突变,从而赋予对属于此类 RT 抑制剂的多种化合物的耐药性。在这里,我们采用预稳态动力学研究了 Q151L 对底物和抑制剂结合以及掺入的催化速率的影响。最重要的是,我们发现 Q151L 突变体在单轮条件下无法掺入 GS-9148。干扰实验表明,GS-9148-二磷酸的存在,即抑制剂的活性形式,不会降低天然对应物掺入的效率。因此,我们得出结论,Q151L 严重损害了 GS-9148-二磷酸与 RT 的结合。这种效应是高度特异性的,因为我们还证明了另一种 NtRTI,替诺福韦,与野生型 RT 相似的选择性被掺入。与其他相关化合物和基于 RT/DNA/GS-9148-二磷酸晶体结构的模型的掺入测定表明,GS-9148 的 2'-氟基团可能会与 Q151L 突变体的侧链发生空间位阻。