MRNA modulator therapy for treatment of haploid insufficiency

Described herein are compositions and methods for the treatment of haploid insufficiency by mRNA modulation. For example, provided herein are fusion proteins comprising an RNA effector protein that targets one or more mRNAs of an active allele of a gene associated with haploid insufficiency, and a m...

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Bibliographic Details
Main Author KOHLER JEFFREY
Format Patent
LanguageChinese
English
Published 09.07.2024
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 are compositions and methods for the treatment of haploid insufficiency by mRNA modulation. For example, provided herein are fusion proteins comprising an RNA effector protein that targets one or more mRNAs of an active allele of a gene associated with haploid insufficiency, and a modulator moiety; the modulator moiety stimulates and/or stabilizes the one or more mRNAs. 本文描述了用于通过mRNA调节治疗单倍体机能不全症的组合物和方法。例如，本文提供了包括RNA效应蛋白和调节子部分的融合蛋白，所述RNA效应蛋白靶向与单倍体机能不全症相关基因的活性等位基因的一种或多种mRNA；所述调节子部分刺激和/或稳定所述一种或多种mRNA。
Bibliography:Application Number: CN202280077046