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ASO

Linked experiment: EXP000608

Linked peptide: P1-9R

Linked paper: Erythroid-Progenitor-Targeted Gene Therapy Using Bifunctional TFR1 Ligand-Peptides in Human Erythropoietic Protoporphyria (2019)

RNA overview

RNA Type

ASO

Payload/target

AON-V1 (FECH intron 3 splice correction)

Target gene/transcript

not reported

Sequence information

Sequence reported in source article

no

RNA sequence

not reported

Sense strand

Antisense strand

Chemical modifications

RNA modifications

Phosphorothioate AON (as previously reported)