Exon skipping oligomer conjugates for muscular dystrophy

Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described in Formula (I). The antisense oligomer conjugate has addition of an R6-G-peptide group with the terminal acetyl group at the 3' end.

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Bibliographic Details
Main Authors Passini, Marco A, Hanson, Gunnar J
Format Patent
LanguageEnglish
Published 26.05.2023
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 53 skipping are described in Formula (I). The antisense oligomer conjugate has addition of an R6-G-peptide group with the terminal acetyl group at the 3' end.
Bibliography:Application Number: NZ20170755438