COMPOSITIONS FOR EDITING MECP2 TRANSCRIPTS AND METHODS THEREOF

Among other things, the present disclosure provides oligonucleotides, compositions and methods thereof that can bring about specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions and methods are useful to treat, prevent, or ameliorate MECP2 associated di...

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Main Authors LAMATTINA, Anthony, MARAPPAN, Subramanian, BUTLER, David Charles Donnell, SHIVALILA, Chikdu Shakti, MONIAN, Prashant, IWAMOTO, Naoki, KUMARASAMY, Jayakanthan, YU, Hui, BOULAY, David John, SHIMIZU, Mamoru, TURNER, Jesse, STANDLEY, Stephany Michelle, CHIVATAKARN, Onanong, GODFREY, Jack David, HARDING, Ian Chandler, ACKER, Christopher Michael, VARGEESE, Chandra, LU, Genliang, HOSS, Andrew Guzior, KANDASAMY, Pachamuthu, APPONI, Luciano Henrique, YANG, Hailin, DESAI, Jigar
Format Patent
LanguageEnglish
Published 02.05.2024
Subjects
CHEMISTRY
DERIVATIVES THEREOF
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SUGARS
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Summary:Among other things, the present disclosure provides oligonucleotides, compositions and methods thereof that can bring about specific editing of a target adenosine in a target RNA molecule. Such oligonucleotides, compositions and methods are useful to treat, prevent, or ameliorate MECP2 associated disorders, diseases and syndromes that can benefit from adenosine modification.
Bibliography:Application Number: AU20220349529