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https://www.sciencedirect.com/science/article/pii/S1569199319309920
We are building on this breakthrough to develop siRNA-mediated therapy for cystic fibrosis by silencing of the αENaC chain with a specific siRNA (siENaCα), delivered with a novel targeted lipid (L)/ peptide (P)/siRNA (R) nanoparticle.Author: A. Christopher Boyd, Shuling Guo, Lulu Huang, Batsheva Kerem, Yifat S. Oren, Amy J. Walker, Stephen ...
https://www.nature.com/articles/s41598-017-00662-2
Apr 06, 2017 · Receptor-targeted liposome-peptide-siRNA nanoparticles represent an efficient and safe non-viral siRNA delivery system that could be used to modulate sodium hyperabsorption, thus helping to restore the correct volume of surface liquid, mucus hydration and mucociliary clearance in the airways, in cystic fibrosis.Author: Maria D. I. Manunta, Aristides D. Tagalakis, Martin Attwood, Ahmad M. Aldossary, Josephine L. Barnes...
https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(16)36437-1
May 01, 2012 · SiRNA silencing of ENaC has been proposed as a CF therapeutic by preventing lung dehydration and the subsequent events of lung pathogenesis. ENaC is a multimeric sodium ion channel localised in the apical membrane of epithelia usually composed of three homologous subunits: α, β, γ but α is the major structural subunit and so was selected for silencing.
https://www.frontiersin.org/articles/10.3389/fphar.2018.01113/full
Introduction. Cystic fibrosis (CF) is the most common lethal genetic disease in the Caucasian population. Since the gene responsible for the disease was identified in 1989 (Riordan et al., 1989), numerous discoveries have led to an improvement in patients’ longevity and quality of life.It is a complex disease, and many advances are still needed to understand the mechanisms of the pathology ...Author: Pauline Bardin, Florence Sonneville, Harriet Corvol, Olivier Tabary
https://discovery.ucl.ac.uk/1551443/1/Manunta_Delivery_ENaC_siRNA.pdf
Delivery of ENaC siRNA to epithelial cells mediated by a targeted nanocomplex: a therapeutic strategy for cystic fibrosis Maria D. I. Manunta 1, Aristides D. Tagalakis1, Martin Attwood1,3, Ahmad M. Aldossary 1, Josephine L. Barnes 2, Mustafa M. Munye1, Alexander Weng1,4, Robin J McAnulty 2 & Stephen L. Hart 1
https://thorax.bmj.com/content/73/9/847
Sep 01, 2018 · ENaC regulation is thus a promising target for CF therapy. Our aim was to develop siRNA nanocomplexes that mediate effective silencing of airway epithelial ENaC in vitro and in vivo with functional correction of epithelial ion and fluid transport.Author: Aristides D Tagalakis, Mustafa M Munye, Rositsa Ivanova, Hanpeng Chen, Claire M Smith, Ahmad M Aldos...
https://www.researchgate.net/publication/315836588_Delivery_of_ENaC_siRNA_to_epithelial_cells_mediated_by_a_targeted_nanocomplex_A_therapeutic_strategy_for_cystic_fibrosis
Delivery of ENaC siRNA to epithelial cells mediated by a targeted nanocomplex: A therapeutic strategy for cystic fibrosis. ... the potential of LPRs as tools f or siRNA delivery to the airways and ...
https://www.idtdna.com/pages/education/decoded/article/small-rna-therapies-for-cystic-fibrosis
Small RNA therapies for cystic fibrosis The McCray Lab (Univ IA, USA) used DsiRNAs to develop a protocol for efficient small RNA delivery into airway epithelia. The resulting method was then used to study miRNA regulation of gene products involved in airway fluid and electrolyte transport, cellular mechanisms linked to cystic fibrosis.
https://www.ncbi.nlm.nih.gov/pubmed/28386087
Apr 06, 2017 · The inhibition of ENaC may have therapeutic potential in CF airways by reducing sodium hyperabsorption, restoring lung epithelial surface fluid levels, airway hydration and mucociliary function. The challenge has been to deliver siRNA to the lung with sufficient efficacy for …Author: Maria D. I. Manunta, Aristides D. Tagalakis, Martin Attwood, Ahmad M. Aldossary, Josephine L. Barnes...
https://academic.oup.com/hmg/article/28/R1/R88/5537028
Jul 23, 2019 · Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung, since it manifests the most significant life-threatening disease.Author: Ziying Yan, Paul B McCray Jr, John F Engelhardt
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