Back to results
US 2025/0145976 A1Published application
Messenger rna encoding cas9 for use in genome-editing systems
AssigneeCrispr Therapeutics AG
InventorChristian Cobaugh
Life of this patent
Estimating the term…
Abstract
The present disclosure provides optimized mRNAs encoding a site-directed endonuclease for use in a CRISPR/Cas system. Also provided herein are delivery systems for use of the CRISPR/Cas system in methods of in vivo and ex vivo genome editing.
Claims31
An mRNA comprising:
- a 5′ untranslated region (UTR);
- an open reading frame (ORF) comprising a nucleotide sequence that encodes a site-directed endonuclease, wherein the nucleotide sequence is at least 85% identical to a nucleotide sequence of SEQ ID NO:
- 4;
- and
- a 3′ untranslated region (UTR).
2 more elements
An mRNA comprising:
- a 5′ UTR;
- an ORF comprising a nucleotide sequence that encodes a site-directed endonuclease, wherein the nucleotide sequence is at least 85% identical to the nucleotide sequence of SEQ ID NO:
- 4;
- and
- a 3′ UTR,
- wherein 100% of the uridines of the mRNA are modified and/or replaced with N1-methylpseudouridine.
3 more elements
An mRNA comprising a nucleotide sequence of SEQ ID NO:
- 2 or SEQ ID NO:
- 14, wherein 100% of the uridines of the mRNA are modified and/or replaced with N1-methylpseudouridine.
Dependencies are inferred from the claim text.