OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION

The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RN...

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Main Authors Jensen Mads Aaboe, Jagasia Ravi, Rasmussen Søren Vestergaard, HEDTJÄRN Maj, Hoener Marius, Patsch Christoph, Costa Veronica, Pedersen Lykke
Format Patent
LanguageEnglish
Published 06.07.2017
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Abstract The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.
AbstractList The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.
Author Patsch Christoph
Pedersen Lykke
Jensen Mads Aaboe
Hoener Marius
Rasmussen Søren Vestergaard
Jagasia Ravi
HEDTJÄRN Maj
Costa Veronica
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– fullname: Patsch Christoph
– fullname: Costa Veronica
– fullname: Pedersen Lykke
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HOFFMANN-LA ROCHE INC
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Snippet The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in...
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SubjectTerms BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
Title OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
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