COMPOSITIONS COMPRISING SYNTHETIC POLYNUCLEOTIDES ENCODING CRISPR RELATED PROTEINS AND SYNTHETIC SGRNAs AND METHODS OF USE

摘要

The present invention relates to compositions comprising and methods of using synthetic polynucleotides, e.g., modified mRNA, encoding CRISPR related proteins including dCAS9 and synthetic sgRNAs targeting a gene of interest.

主权项

1. A synthetic polynucleotide comprising:
(a) a first region of linked nucleosides, said first region encoding a CRISPR related protein selected from the group consisting of the CRISPR related proteins found in Table 6; (b) a first flanking region located at the 5′ terminus of said first region comprising a sequence of linked nucleosides selected from the group consisting of the 5′ untranslated region (UTR) found in Table 7; (c) a second flanking region located at the 3′ terminus of said first region comprising a sequence of linked nucleosides selected from the group consisting of the 3′ untranslated region (UTR) found in Table 8.