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Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.
Mol Ther Methods Clin Dev. 2018 Nov 1;12:71-84. doi: 10.1016/j.omtm.2018.10.016. eCollection 2019 Mar 15.
Mol Ther Methods Clin Dev. 2018.
PMID: 30534580
Free PMC article.
Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease.
McNamara EL, Taylor RL, Clayton JS, Goullee H, Dilworth KL, Pinós T, Brull A, Alexander IE, Lisowski L, Ravenscroft G, Laing NG, Nowak KJ.
McNamara EL, et al. Among authors: dilworth kl.
Hum Mol Genet. 2020 Jan 1;29(1):20-30. doi: 10.1093/hmg/ddz214.
Hum Mol Genet. 2020.
PMID: 31511858
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Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes.
Ginn SL, Amaya AK, Liao SHY, Zhu E, Cunningham SC, Lee M, Hallwirth CV, Logan GJ, Tay SS, Cesare AJ, Pickett HA, Grompe M, Dilworth K, Lisowski L, Alexander IE.
Ginn SL, et al.
JHEP Rep. 2019 Dec 27;2(1):100065. doi: 10.1016/j.jhepr.2019.100065. eCollection 2020 Feb.
JHEP Rep. 2019.
PMID: 32039406
Free PMC article.
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Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.
Cabanes-Creus M, Westhaus A, Navarro RG, Baltazar G, Zhu E, Amaya AK, Liao SHY, Scott S, Sallard E, Dilworth KL, Rybicki A, Drouyer M, Hallwirth CV, Bennett A, Santilli G, Thrasher AJ, Agbandje-McKenna M, Alexander IE, Lisowski L.
Cabanes-Creus M, et al. Among authors: dilworth kl.
Mol Ther Methods Clin Dev. 2020 May 13;17:1139-1154. doi: 10.1016/j.omtm.2020.05.004. eCollection 2020 Jun 12.
Mol Ther Methods Clin Dev. 2020.
PMID: 32490035
Free PMC article.
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Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing.
Nguyen Tran MT, Mohd Khalid MKN, Wang Q, Walker JKR, Lidgerwood GE, Dilworth KL, Lisowski L, Pébay A, Hewitt AW.
Nguyen Tran MT, et al. Among authors: dilworth kl.
Nat Commun. 2020 Sep 25;11(1):4871. doi: 10.1038/s41467-020-18715-y.
Nat Commun. 2020.
PMID: 32978399
Free PMC article.
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Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.
Westhaus A, Cabanes-Creus M, Dilworth KL, Zhu E, Salas Gómez D, Navarro RG, Amaya AK, Scott S, Kwiatek M, McCorkindale AL, Hayman TE, Frahm S, Perocheau DP, Tran BM, Vincan E, Wong SL, Waters SA, Riddiough GE, Perini MV, Wilson LOW, Baruteau J, Diecke S, González-Aseguinolaza G, Santilli G, Thrasher AJ, Alexander IE, Lisowski L.
Westhaus A, et al. Among authors: dilworth kl.
Hum Gene Ther. 2023 Apr;34(7-8):273-288. doi: 10.1089/hum.2022.188.
Hum Gene Ther. 2023.
PMID: 36927149
Free PMC article.
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