COMPOSITIONS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY

The present disclosure relates compositions and methods to treat neuromuscular diseases and disorders, e.g., spinal muscular atrophy (SMA), characterized by the presence of a splicing silencer located in SMN2 intron 7 pre-mRNA, comprising coadministering a therapeutically effective amount of an anti...

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Bibliographic Details
Main Authors MARASCO, Luciano E, KORNBLIHTT, Alberto R, KRAINER, Adrian, STIGLIANO, Jose
Format Patent
LanguageEnglish
Published 28.09.2023
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 disclosure relates compositions and methods to treat neuromuscular diseases and disorders, e.g., spinal muscular atrophy (SMA), characterized by the presence of a splicing silencer located in SMN2 intron 7 pre-mRNA, comprising coadministering a therapeutically effective amount of an antisense oligonucleotide (ASO) complementary to a nucleotide sequence within intron 7 of human SMN2 pre-mRNA; and a subclinical dose of a histone deacetylate inhibitor, e.g., valproic acid, trichostatin A, or a combination thereof.
Bibliography:Application Number: US202118005514