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oligonucleotide

Linked experiment: EXP001579

Linked peptide: (RXRRBR)2XB

Linked paper: Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum (2011)

RNA overview

RNA Type

oligonucleotide

Payload/target

ATM splicing mutation c.7865C>T (TATC cells; exon 55 aberrant splicing)

Target gene/transcript

not reported

Sequence information

Sequence reported in source article

yes

RNA sequence

sense:TAAGCATCACAAAGTACCTCAACAC

Sense strand

TAAGCATCACAAAGTACCTCAACAC

Antisense strand

Chemical modifications

RNA modifications

Customized AMO sequence described previously (Du et al. 2007 PNAS, ref 10); AMCA-labeled CPP-AMO used for uptake imaging in some experiments