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US 12,644,123 B2Granted
Modulating expression of ALAS1 (5′-aminolevulinate synthase 1) gene
AssigneeCrispr Therapeutics AG
InventorsMary-Lee Dequeant, Heidi Heath, Rachel Yuen
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Abstract
The present disclosure relates to methods, compositions and kits for treating conditions that are related to the modulation of expression of 5′-Aminolevulinate Synthase 1 (ALAS1) by gene editing.
Claims8
A guide RNA (gRNA) for targeting a 5′-Aminolevulinate Synthase 1 (ALAS1) genomic locus, comprising a spacer sequence having at least 80% sequence identity to any one of the sequences of SEQ ID NOs:
- 25-47, 83-87, and 89-112, and wherein the gRNA is capable of inducing a cutting efficiency of at least 40% for targeting the ALAS1 genomic locus.
A composition, comprising:
- (a) a guide RNA (gRNA) that targets a 5′-Aminolevulinate Synthase 1 (ALAS1) genomic locus, or a nucleic acid encoding the gRNA, wherein the gRNA comprises a spacer sequence having at least 80% sequence identity to any one of the sequences of SEQ ID NOs:
- 25-47, 83-87, and 89-112, and is capable of inducing a cutting efficiency of at least 40% for targeting the ALAS1 genomic locus;
- and
- (b) an endonuclease or a nucleic acid encoding an endonuclease.
1 more element
Dependencies are inferred from the claim text.