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Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy.
Logan GJ, Mietzsch M, Khandekar N, D'Silva A, Anderson D, Mandwie M, Hsi J, Nelson AR, Chipman P, Jackson J, Schofield P, Christ D, Goodnow CC, Reed JH, Farrar MA, McKenna R, Alexander IE. Logan GJ, et al. Among authors: mckenna r. Mol Ther. 2023 Jul 5;31(7):1979-1993. doi: 10.1016/j.ymthe.2023.03.032. Epub 2023 Apr 3. Mol Ther. 2023. PMID: 37012705
Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.
Kok CY, Tsurusaki S, Cabanes-Creus M, Igoor S, Rao R, Skelton R, Liao SHY, Ginn SL, Knight M, Scott S, Mietzsch M, Fitzsimmons R, Miller J, Mohamed TMA, McKenna R, Chong JJH, Hill AP, Hudson JE, Alexander IE, Lisowski L, Kizana E. Kok CY, et al. Among authors: mckenna r. Mol Ther Methods Clin Dev. 2023 Aug 18;30:459-473. doi: 10.1016/j.omtm.2023.08.010. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37674904 Free PMC article.
First-in-class multifunctional TYMS nonclassical antifolate inhibitor with potent in vivo activity that prolongs survival.
Guijarro MV, Kellish PC, Dib PE, Paciaroni NG, Nawab A, Andring J, Kulemina L, Borrero NV, Modenutti C, Feely M, Nasri E, Seifert RP, Luo X, Bennett RL, Shabashvili D, Licht JD, McKenna R, Roitberg A, Huigens RW 3rd, Kaye FJ, Zajac-Kaye M. Guijarro MV, et al. Among authors: mckenna r. JCI Insight. 2023 Apr 25;8(10):e158798. doi: 10.1172/jci.insight.158798. JCI Insight. 2023. PMID: 37097751 Free PMC article.
Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing.
Mietzsch M, Nelson AR, Hsi J, Zachary J, Potts L, Chipman P, Ghanem M, Khandekar N, Alexander IE, Logan GJ, Huiskonen JT, McKenna R. Mietzsch M, et al. Among authors: mckenna r. bioRxiv [Preprint]. 2024 May 3:2024.05.01.590489. doi: 10.1101/2024.05.01.590489. bioRxiv. 2024. PMID: 38746165 Free PMC article. Preprint.
Sociodemographic Factors Predict Long-Term Attrition from Multidisciplinary Cleft Clinic.
Wagner CS, Barnett SL, Romeo D, Morales CZ, Salinero LK, Barrero CE, Pontell ME, McKenna RA, Folsom N, Taylor JA, Swanson JW. Wagner CS, et al. Among authors: mckenna ra. Cleft Palate Craniofac J. 2024 Feb 29:10556656241234804. doi: 10.1177/10556656241234804. Online ahead of print. Cleft Palate Craniofac J. 2024. PMID: 38425129
941 results