Structure-Guided Chemical Modification of Guide RNA and its Applications

The disclosure relates to compositions comprising and methods for chemical modification of single guide RNA (sgRNA), tracrRNA and/or crRNA used individually or in combination with one another or Cas system components. Compositions comprising modified ribonucleic acids have been designed with chemical modification for even higher efficiency as unmodified native strand of sgRNA. Administration of modified ribonucleic acids will allow decreased immune response when administered to a subject, increased stability, increased editing efficiency and facilitated in vivo delivery of sgRNA via various delivery platforms. The disclosure also relates to methods of decreasing off-target effect of CRISPR and a CRISPR complex.

Researchers

Departments: Office of the Institute Professors, Department of Chemical Engineering
Technology Areas: Biotechnology: DNA & RNA Editing / Chemicals & Materials: Polymers / Drug Discovery and Research Tools: Genomics & Proteomics / Therapeutics: Nucleic Acids
Impact Areas: Healthy Living

  • structure-guided chemical modification of guide rna and its applications
    United States of America | Granted | 11,845,933

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