Oligonucleotide (IMAGE)

Iowa State University

Oligonucleotide

Caption

A short and smart oligonucleotide: a short antisense oligonucleotide (3UP8) targeting a specific intronic sequence corrects aberrant splicing of Survival Motor Neuron 2 (SMN2) and restores high levels of functional SMN protein in patient cells of spinal muscular atrophy (SMA). Prominent green dots (see in "SMN section") represent well-organized SMN bodies (gems) in the nucleus of the 3UP8-treated cells (bottom two panels). Gem contains another protein ZPR1 (see red dots in "ZPR1 section") that co-localizes with SMN protein in the nucleus of the cell (see white dots in "Merge section"). The control oligonucleotide (F8) that targets an unrelated sequence had no effect on SMN or ZPR1 levels in patient cells (top two panels). Cell were transfected with the same amount of antisense oligonucleotide (compare the color intensity in "Oligo section").

Credit

Courtesy Ravindra Singh

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