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. Dans divers modes de réalisation, l'invention concerne des compositions comprenant (a)...

Full description

Saved in:
Bibliographic Details
Main Authors KHEIRABADI, Mahboubeh, LI, Xiang, QIAN, Ziqing, WYSK, Mark, GIRGENRATH, Mahsweta, LIAN, Wenlong, ESTRELLA, Nelsa, SETHURAMAN, Natarajan
Format Patent
LanguageEnglish
French
Published 13.04.2023
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
Online AccessGet full text

Cover

More Information
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. Dans divers modes de réalisation, l'invention concerne des compositions comprenant (a) un peptide cyclique ; et (b) un composé antisens, le composé antisens ciblant l'exon 44 du gène DMD dans une séquence de pré-ARNm.
Bibliography:Application Number: WO2022US75691