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CRISPR/Cas9 gene editing therapies for cystic fibrosis.
Graham C, Hart S. Graham C, et al. Among authors: hart s. Expert Opin Biol Ther. 2021 Jun;21(6):767-780. doi: 10.1080/14712598.2021.1869208. Epub 2021 Feb 2. Expert Opin Biol Ther. 2021. PMID: 33412935
Genetic therapies for cystic fibrosis lung disease.
Hart SL, Harrison PT. Hart SL, et al. Curr Opin Pharmacol. 2017 Jun;34:119-124. doi: 10.1016/j.coph.2017.10.006. Epub 2017 Nov 5. Curr Opin Pharmacol. 2017. PMID: 29107808 Review.
New approaches to genetic therapies for cystic fibrosis.
Christopher Boyd A, Guo S, Huang L, Kerem B, Oren YS, Walker AJ, Hart SL. Christopher Boyd A, et al. Among authors: hart sl. J Cyst Fibros. 2020 Mar;19 Suppl 1:S54-S59. doi: 10.1016/j.jcf.2019.12.012. Epub 2020 Jan 13. J Cyst Fibros. 2020. PMID: 31948871 Free article. Review.
Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing.
Walker AJ, Graham C, Greenwood M, Woodall M, Maeshima R, O'Hara-Wright M, Sanz DJ, Guerrini I, Aldossary AM, O'Callaghan C, Baines DL, Harrison PT, Hart SL. Walker AJ, et al. Mol Ther Methods Clin Dev. 2023 Oct 18;31:101140. doi: 10.1016/j.omtm.2023.101140. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027060 Free PMC article.
Lipid Nanoparticles Efficiently Deliver the Base Editor ABE8e for COL7A1 Correction in Dystrophic Epidermolysis Bullosa Fibroblasts In Vitro.
Guri-Lamce I, Alrokh Y, Graham C, Maeshima R, Rognoni E, Caley M, Łaczmański Ł, Hart SL, McGrath JA, Jacków-Malinowska J. Guri-Lamce I, et al. J Invest Dermatol. 2024 Apr 5:S0022-202X(24)00269-0. doi: 10.1016/j.jid.2024.03.027. Online ahead of print. J Invest Dermatol. 2024. PMID: 38583743 Free article. No abstract available.
A beginner's guide to gene editing.
Harrison PT, Hart S. Harrison PT, et al. Among authors: hart s. Exp Physiol. 2018 Apr 1;103(4):439-448. doi: 10.1113/EP086047. Epub 2018 Jan 25. Exp Physiol. 2018. PMID: 29282799 Free article. Review.
Effective silencing of ENaC by siRNA delivered with epithelial-targeted nanocomplexes in human cystic fibrosis cells and in mouse lung.
Tagalakis AD, Munye MM, Ivanova R, Chen H, Smith CM, Aldossary AM, Rosa LZ, Moulding D, Barnes JL, Kafetzis KN, Jones SA, Baines DL, Moss GWJ, O'Callaghan C, McAnulty RJ, Hart SL. Tagalakis AD, et al. Thorax. 2018 Sep;73(9):847-856. doi: 10.1136/thoraxjnl-2017-210670. Epub 2018 May 10. Thorax. 2018. PMID: 29748250 Free PMC article.
1,607 results