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Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis.
J Cyst Fibros. 2023 Mar;22 Suppl 1(Suppl 1):S39-S44. doi: 10.1016/j.jcf.2022.12.016. Epub 2023 Jan 17.
J Cyst Fibros. 2023.
PMID: 36658041
Free PMC article.
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, t …
Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective C …
Progress in Respiratory Gene Therapy.
McLachlan G, Alton EWFW, Boyd AC, Clarke NK, Davies JC, Gill DR, Griesenbach U, Hickmott JW, Hyde SC, Miah KM, Molina CJ.
McLachlan G, et al. Among authors: griesenbach u.
Hum Gene Ther. 2022 Sep;33(17-18):893-912. doi: 10.1089/hum.2022.172.
Hum Gene Ther. 2022.
PMID: 36074947
Free PMC article.
Review.
In addition, the inclusion of features such as codon optimization and promoters providing long-term expression have improved the expression characteristics of therapeutic transgenes. ...
In addition, the inclusion of features such as codon optimization and promoters providing long-term expression have improved the expr …
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Ex Vivo and In Vivo Lentivirus-Mediated Transduction of Airway Epithelial Progenitor Cells.
Leoni G, Wasowicz MY, Chan M, Meng C, Farley R, Brody SL, Inoue M, Hasegawa M, Alton EW, Griesenbach U.
Leoni G, et al. Among authors: griesenbach u.
Curr Gene Ther. 2015;15(6):581-90. doi: 10.2174/1566523215666151016123625.
Curr Gene Ther. 2015.
PMID: 26471068
Free article.
A key challenge in pulmonary gene therapy for cystic fibrosis is to provide long-term correction of the genetic defect. This may be achievable by targeting airway epithelial stem/progenitor cells with an integrating vector. ...
A key challenge in pulmonary gene therapy for cystic fibrosis is to provide long-term correction of the genetic defect. This may be a …
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The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep.
Alton EW, Baker A, Baker E, Boyd AC, Cheng SH, Coles RL, Collie DD, Davidson H, Davies JC, Gill DR, Gordon C, Griesenbach U, Higgins T, Hyde SC, Innes JA, McCormick D, McGovern M, McLachlan G, Porteous DJ, Pringle I, Scheule RK, Shaw DJ, Smith S, Sumner-Jones SG, Tennant P, Vrettou C.
Alton EW, et al. Among authors: griesenbach u.
Biomaterials. 2013 Dec;34(38):10267-77. doi: 10.1016/j.biomaterials.2013.09.023. Epub 2013 Oct 3.
Biomaterials. 2013.
PMID: 24090839
The UK CF Gene Therapy Consortium is currently evaluating long-term repeated delivery of pGM169 complexed with the cationic lipid GL67A in a large Multidose Trial. ...
The UK CF Gene Therapy Consortium is currently evaluating long-term repeated delivery of pGM169 complexed with the cationic lipid GL6 …
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