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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: alton ewfw. 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 …
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: alton ew. 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 …
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. 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 …
Clinical expert panel on monitoring potential lung toxicity of inhaled oligonucleotides: consensus points and recommendations.
Alton EW, Boushey HA, Garn H, Green FH, Hodges M, Martin RJ, Murdoch RD, Renz H, Shrewsbury SB, Seguin R, Johnson G, Parry JD, Tepper J, Renzi P, Cavagnaro J, Ferrari N. Alton EW, et al. Nucleic Acid Ther. 2012 Aug;22(4):246-54. doi: 10.1089/nat.2012.0345. Epub 2012 Jul 18. Nucleic Acid Ther. 2012. PMID: 22809313 Free PMC article.
To that end, identification of sensitive methods and biomarkers that can detect toxicity in humans before any long term and/or irreversible side effects occur would be helpful. In light of the public's greater interests, the Inhalation Subcommittee of the Oligonucleotide S …
To that end, identification of sensitive methods and biomarkers that can detect toxicity in humans before any long term and/or irreve …
Cystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator function.
Simmonds NJ, D'Souza L, Roughton M, Alton EW, Davies JC, Hodson ME. Simmonds NJ, et al. Among authors: alton ew. Eur Respir J. 2011 May;37(5):1076-82. doi: 10.1183/09031936.00079010. Epub 2010 Sep 16. Eur Respir J. 2011. PMID: 20847077 Free article.
Our aim was to determine whether residual function of the cystic fibrosis transmembrane conductance regulator (CFTR) is present in long-term survivors with severe mutations. Nasal potential difference (PD) and sweat chloride were measured in 34 long-term survivors ( …
Our aim was to determine whether residual function of the cystic fibrosis transmembrane conductance regulator (CFTR) is present in long-t …
Poly (D, L-lactide-co-glycolide)/DNA microspheres to facilitate prolonged transgene expression in airway epithelium in vitro, ex vivo and in vivo.
Stern M, Ulrich K, Geddes DM, Alton EW. Stern M, et al. Among authors: alton ew. Gene Ther. 2003 Aug;10(16):1282-8. doi: 10.1038/sj.gt.3301994. Gene Ther. 2003. PMID: 12883524
Repeat administration of gene therapy for cystic fibrosis is likely to be essential for long-term clinical efficacy. This may be minimized by the use of slow-release gene transfer preparations with more prolonged expression and longer dosing intervals for the patient. ...
Repeat administration of gene therapy for cystic fibrosis is likely to be essential for long-term clinical efficacy. This may be mini …
Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis.
Ferrari S, Geddes DM, Alton EW. Ferrari S, et al. Among authors: alton ew. Adv Drug Deliv Rev. 2002 Dec 5;54(11):1373-93. doi: 10.1016/s0169-409x(02)00145-x. Adv Drug Deliv Rev. 2002. PMID: 12458150 Free PMC article. Review.
In addition, once in the cell, further hurdles have to be overcome, including DNA degradation, nuclear import and the ability to maintain long-term transgene expression. Strategies to overcome these barriers will be addressed in this review and include the use of: (i) clin …
In addition, once in the cell, further hurdles have to be overcome, including DNA degradation, nuclear import and the ability to maintain lo …
Characterisation of LMD virus-like nanoparticles self-assembled from cationic liposomes, adenovirus core peptide mu and plasmid DNA.
Tagawa T, Manvell M, Brown N, Keller M, Perouzel E, Murray KD, Harbottle RP, Tecle M, Booy F, Brahimi-Horn MC, Coutelle C, Lemoine NR, Alton EW, Miller AD. Tagawa T, et al. Among authors: alton ew. Gene Ther. 2002 May;9(9):564-76. doi: 10.1038/sj.gt.3301686. Gene Ther. 2002. PMID: 11973632
LMD particles can be formulated reproducibly, they are amenable to long-term storage (>1 month) at -80 degrees C and are stable to aggregation at a plasmid DNA concentration up to 5 mg/ml (15 mM nucleotide concentration). ...
LMD particles can be formulated reproducibly, they are amenable to long-term storage (>1 month) at -80 degrees C and are stable to …
Cystic fibrosis mice carrying the missense mutation G551D replicate human genotype-phenotype correlations.
Delaney SJ, Alton EW, Smith SN, Lunn DP, Farley R, Lovelock PK, Thomson SA, Hume DA, Lamb D, Porteous DJ, Dorin JR, Wainwright BJ. Delaney SJ, et al. Among authors: alton ew. EMBO J. 1996 Mar 1;15(5):955-63. EMBO J. 1996. PMID: 8605891 Free PMC article.
The G551D mutant mice show greatly reduced CFTR-related chloride transport, displaying activity intermediate between that of cftr(mlUNC) replacement ('null') and cftr(mlHGU) insertional (residual activity) mutants and equivalent to approximately 4% of wild-type CFTR activity. The …
The G551D mutant mice show greatly reduced CFTR-related chloride transport, displaying activity intermediate between that of cftr(mlUNC) rep …
Long-term survival of the exon 10 insertional cystic fibrosis mutant mouse is a consequence of low level residual wild-type Cftr gene expression.
Dorin JR, Stevenson BJ, Fleming S, Alton EW, Dickinson P, Porteous DJ. Dorin JR, et al. Among authors: alton ew. Mamm Genome. 1994 Aug;5(8):465-72. doi: 10.1007/BF00369314. Mamm Genome. 1994. PMID: 7949729 Free article.
We conclude that (i) residual wild-type mRNA in the exon 10 insertional mutant mouse ameliorates the severity of the intestinal phenotype observed in the absolute "null" CF mice, (ii) the presence of low-level residual wild-type Cftr mRNA does not correct the CF ion transport def …
We conclude that (i) residual wild-type mRNA in the exon 10 insertional mutant mouse ameliorates the severity of the intestinal phenotype ob …