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Full-Text Articles in Biochemistry
Stabilin-Mediated Cellular Internalization Of Phosphorothioate-Modified Antisense Oligonucleotides (Asos), Colton M. Miller, Aaron J. Donner, Emma K. Blank, Andrew W. Egger, Brianna M. Kellar, Punit P. Seth, Edward N. Harris
Stabilin-Mediated Cellular Internalization Of Phosphorothioate-Modified Antisense Oligonucleotides (Asos), Colton M. Miller, Aaron J. Donner, Emma K. Blank, Andrew W. Egger, Brianna M. Kellar, Punit P. Seth, Edward N. Harris
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Introduction: Antisense oligonucleotides (ASOs) are short chemically modified oligonucleotides (5-7.4 kDa) that can produce a pharmacological effect by binding to RNA and affecting intermediary metabolism. Over 35 phosphorothioate (PS) ASOs are at various stages of clinical development for use as therapeutic agents and pharmacological tools. Antisense therapy is a progressing area of research, as these small strands of nucleotide oligomers can be produced to silence genes that aggravate chronic disorders or infections. An important distinction for ASOs compared to DNA is the substitution of the phosphodiester (PO) backbone with the PS modification. This sulfur substitution allows for these polar polyanionic …