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Viral vector platforms within the gene therapy landscape.
Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Bulcha JT, et al. Signal Transduct Target Ther. 2021 Feb 8;6(1):53. doi: 10.1038/s41392-021-00487-6. Signal Transduct Target Ther. 2021. PMID: 33558455 Free PMC article. Review.
Immune Responses to Viral Gene Therapy Vectors.
Shirley JL, de Jong YP, Terhorst C, Herzog RW. Shirley JL, et al. Mol Ther. 2020 Mar 4;28(3):709-722. doi: 10.1016/j.ymthe.2020.01.001. Epub 2020 Jan 10. Mol Ther. 2020. PMID: 31968213 Free PMC article. Review.
Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes.
Namkung S, Tran NT, Manokaran S, He R, Su Q, Xie J, Gao G, Tai PWL. Namkung S, et al. Hum Gene Ther. 2022 Nov;33(21-22):1187-1196. doi: 10.1089/hum.2022.143. Hum Gene Ther. 2022. PMID: 36178359 Free PMC article.
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice.
Wang J, Zhang Y, Mendonca CA, Yukselen O, Muneeruddin K, Ren L, Liang J, Zhou C, Xie J, Li J, Jiang Z, Kucukural A, Shaffer SA, Gao G, Wang D. Wang J, et al. Nature. 2022 Apr;604(7905):343-348. doi: 10.1038/s41586-022-04533-3. Epub 2022 Mar 23. Nature. 2022. PMID: 35322228 Free PMC article.
A Molecular Revolution in the Treatment of Hemophilia.
Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. Butterfield JSS, et al. Mol Ther. 2020 Apr 8;28(4):997-1015. doi: 10.1016/j.ymthe.2019.11.006. Epub 2019 Nov 13. Mol Ther. 2020. PMID: 31843450 Free PMC article. Review.
104 results