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 | , , , , , , , |
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Format | Patent |
Language | English |
Published |
06.07.2017
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Subjects | |
Online Access | Get full text |
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Summary: | 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. |
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Bibliography: | Application Number: US201615351113 |