COMPOSITIONS AND METHODS FOR TREATING TARDBP ASSOCIATED DISEASES

The present invention relates to the field of antisense oligonucleotides used to reduce expression of the transactive response DNA binding protein 43 (TARDBP) gene which encodes the protein TAR DNA-binding protein 43 (TDP43). The present invention also relates to the field of antisense oligonucleoti...

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Bibliographic Details
Main Authors FLYNN, Loren Louise, WILTON, Stephen, AKKARI, Patrick Anthony, MEJZINI, Rita, FLETCHER, Sue
Format Patent
LanguageEnglish
Published 22.06.2023
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
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary:The present invention relates to the field of antisense oligonucleotides used to reduce expression of the transactive response DNA binding protein 43 (TARDBP) gene which encodes the protein TAR DNA-binding protein 43 (TDP43). The present invention also relates to the field of antisense oligonucleotides used to prevent the subsequent downregulation of stathmin-2. The invention also provides pharmaceutical compositions and methods to treat the effects of a disease associated with TDP43 proteinopathy by administration of antisense oligonucleotides and therapeutic compositions comprising AONs targeted to TARDBP and AONs targeted to STMN2.
Bibliography:Application Number: AU20210394117