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US 2022/0090073 A1Published application
Methods and compositions for the treatment of neurodegenerative diseases
OwnerAlexandra PERLEGOSinventor
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Abstract
Recombinant constructs and cells, as well as methods, for modulating mRNA translation by targeting YTHDF proteins, which play a role in the recognition of m6A methylation of mRNA transcripts, are provided.
Claims20
A nucleic acid molecule, encoding a YTHDF1, YHTDF2, or YTHDF3 protein, operably linked to a heterologous promoter and/or an upstream activation sequence (UAS).
A method of treating a neurodegenerative disease in a subject in need thereof, comprising:
- a) administering a therapeutically-effective amount of a YTHDF protein comprising
- i) YTHDF1, YTHDF2, or YTHDF3;
- or
- ii) a protein that is at least 95% identical to the sequence of SEQ ID NO:
- 2, 3, or 4, wherein the YTHDF protein is capable of specifically binding to N6-methyladenosine (m6A)-modified mRNA, when expressed in a human cell;
- and
- b) reducing or eliminating at least one symptom of the neurodegenerative disease.
4 more elements
Dependencies are inferred from the claim text.