Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier

Effective transvascular delivery of therapeutic oligonucleotides to the brain presents a major hurdle to the development of gene silencing technologies for treatment of genetically defined neurological disorders. Distribution to the brain after systemic administrations is hampered by the low permeab...

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Vydáno v:Mol Ther
Hlavní autoři: Godinho, Bruno M.D.C., Henninger, Nils, Bouley, James, Alterman, Julia F., Haraszti, Reka A., Gilbert, James W., Sapp, Ellen, Coles, Andrew H., Biscans, Annabelle, Nikan, Mehran, Echeverria, Dimas, DiFiglia, Marian, Aronin, Neil, Khvorova, Anastasia
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6225091/
https://ncbi.nlm.nih.gov/pubmed/30143435
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.08.005
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