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US 2024/0392271 A1Published application
Engineered casx repressor systems
AssigneeScribe Therapeutics Inc.
InventorsJason FERNANDES, Ross WHITE, Sean HIGGINS, Sarah DENNY, Benjamin OAKES, Emeric Jean Marius CHARLES
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Abstract
The disclosure relates to gene repressor systems comprising catalytically-dead Class 2 CRISPR proteins and one or more transcription repressor domains linked to the catalytically-dead Class 2 CRISPR protein as a fusion protein, as well as a guide ribonucleic acid (gRNA); and methods of making and using same.
Claims124
A gene repressor system comprising:
- a) a catalytically-dead Class 2 CRISPR protein;
- b) a transcription repressor domain;
- and
- c) a guide ribonucleic acid (gRNA)
- wherein:
- i) the transcription repressor domain is linked to the catalytically-dead Class 2 CRISPR protein as a fusion protein;
- ii) the gRNA comprises a targeting sequence complementary to a target nucleic acid sequence of a gene targeted for repression, silencing, or downregulation;
- iii) the fusion protein is capable of forming a ribonucleoprotein (RNP) with the gRNA;
- and
- iv) the RNP is capable of binding to the target nucleic acid.
7 more elements
A gene repressor system comprising:
- a) a catalytically-dead Class 2 CRISPR protein;
- b) a first transcription repressor domain;
- c) a second transcription repressor domain;
- d) a third transcription repressor domain;
- and
- e) a guide ribonucleic acid (gRNA)
- wherein:
- i) the catalytically-dead Class 2 CRISPR protein, the first transcription repressor domain, the second transcription repressor domain, and the third transcription repressor domain are linked as a fusion protein;
- ii) the gRNA comprises a targeting sequence complementary to a target nucleic acid sequence of a gene targeted for repression, silencing, or downregulation;
- iii) the fusion protein is capable of forming a ribonucleoprotein (RNP) with the gRNA;
- and
- iv) the RNP is capable of binding to the target nucleic acid of the gene.
9 more elements
Dependencies are inferred from the claim text.