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PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs

Effective systemic delivery of small interfering RNAs (siRNAs) to tissues other than liver remains a challenge. siRNAs are small (∼15 kDa) and therefore rapidly cleared by the kidneys, resulting in limited blood residence times and tissue exposure. Current strategies to improve the unfavorable pharm...

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Автори: Bruno M.D.C. Godinho, Emily G. Knox, Samuel Hildebrand, James W. Gilbert, Dimas Echeverria, Zachary Kennedy, Reka A. Haraszti, Chantal M. Ferguson, Andrew H. Coles, Annabelle Biscans, Jillian Caiazzi, Julia F. Alterman, Matthew R. Hassler, Anastasia Khvorova
Формат: Artigo
Мова:Inglês
Опубліковано: Elsevier 2022-09-01
Серія:Molecular Therapy: Nucleic Acids
Предмети:
oligonucleotide
PEGylation
extrahepatic
GalNAc
siRNA conjugates
RNA interference
Онлайн доступ:http://www.sciencedirect.com/science/article/pii/S2162253122001573
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