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2020 1
2021 5
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2023 4
2024 0

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Page 1
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: mandwie m. 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
Gain-of-function factor H-related 5 protein impairs glomerular complement regulation resulting in kidney damage.
Malik TH, Gitterman DP, Lavin DP, Lomax-Browne HJ, Hiemeyer EC, Moran LB, Boroviak K, Cook HT, Gilmore AC, Mandwie M, Ahmad A, Alexander IE, Logan GJ, Marchbank KJ, Bradley A, Pickering MC. Malik TH, et al. Among authors: mandwie m. Proc Natl Acad Sci U S A. 2021 Mar 30;118(13):e2022722118. doi: 10.1073/pnas.2022722118. Proc Natl Acad Sci U S A. 2021. PMID: 33753502 Free PMC article.
Conversion of the Liver into a Biofactory for DNaseI Using Adeno-Associated Virus Vector Gene Transfer Reduces Neutrophil Extracellular Traps in a Model of Systemic Lupus Erythematosus.
Ahmad A, Mandwie M, O'Sullivan KM, Smyth C, York J, Doyle H, Holdsworth SR, Pickering MC, Lachmann PJ, Alexander IE, Logan GJ. Ahmad A, et al. Among authors: mandwie m. Hum Gene Ther. 2022 May;33(9-10):560-571. doi: 10.1089/hum.2021.264. Hum Gene Ther. 2022. PMID: 35293226
Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
Cunningham SC, van Dijk EB, Zhu E, Sugden M, Mandwie M, Siew S, Devanapalli B, Tolun AA, Klein A, Wilson L, Aryamanesh N, Gissen P, Baruteau J, Bhattacharya K, Alexander IE. Cunningham SC, et al. Among authors: mandwie m. Hum Gene Ther. 2023 Sep;34(17-18):917-926. doi: 10.1089/hum.2023.011. Epub 2023 Sep 11. Hum Gene Ther. 2023. PMID: 37350098
11 results