COMPOUNDS AND METHODS FOR SKIPPING EXON 44 IN DUCHENNE MUSCULAR DYSTROPHY

Described herein in various embodiments are compositions comprising (a) a cyclic peptide; and (b) an antisense compound, wherein the antisense compound targets exon 44 of the DMD gene in a pre-mRNA sequence.

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Bibliographic Details
Main Authors KHEIRABADI, Mahboubeh, LI, Xiang, QIAN, Ziqing, WYSK, Mark, GIRGENRATH, Mahsweta, LIAN, Wenlong, ESTRELLA, Nelsa, SETHURAMAN, Natarajan
Format Patent
LanguageEnglish
French
German
Published 10.07.2024
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary:Described herein in various embodiments are compositions comprising (a) a cyclic peptide; and (b) an antisense compound, wherein the antisense compound targets exon 44 of the DMD gene in a pre-mRNA sequence.
Bibliography:Application Number: EP20220777539