Structural and mechanistic bases for resistance of the M66I capsid variant to lenacapavir.

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Tác giả: Jonathan R Andino-Moncada, Arun S Annamalai, Stephanie M Bester, Lorenzo Briganti, Alan N Engelman, Ashwanth C Francis, Eric O Freed, Juliet Greenwood, Reed Haney, Szu-Wei Huang, Alex B Kleinpeter, Mamuka Kvaratskhelia, Binh Nguyen, Rajalingam Radhakrishnan, Lauren I Schope, Parmit K Singh, Satya P Singh, Meghna Tripathi, Guochao Wei

Ngôn ngữ: eng

Ký hiệu phân loại: 725.7 *Refreshment facilities and park structures

Thông tin xuất bản: United States : mBio , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 743042

Lenacapavir (LEN) is the first-in-class viral capsid protein (CA) targeting antiretroviral for treating multi-drug-resistant HIV-1 infection. Clinical trials and cell culture experiments have identified resistance-associated mutations (RAMs) in the vicinity of the hydrophobic CA pocket targeted by LEN. The M66I substitution conferred by far the highest level of resistance to the inhibitor compared to other RAMs. Here we investigated structural and mechanistic bases for how the M66I change affects LEN binding to CA and viral replication. The high-resolution X-ray structure of the CA(M66I) hexamer revealed that the β-branched side chain of Ile66 induces steric hindrance specifically to LEN, thereby markedly reducing the inhibitor binding affinity. By contrast, the M66I substitution did not affect the binding of Phe-Gly (FG)-motif-containing cellular cofactors CPSF6, NUP153, or SEC24C, which engage the same hydrophobic pocket of CA. In cell culture, the M66I variant did not acquire compensatory mutations. Analysis of viral replication intermediates revealed that HIV-1
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