Pyruvate kinase deficiency (PKD) gene editing treatment method
AssigneesCentro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A, M.P., Consorcio Centro De InvestigacióN BioméDica En Red, Fundación Instituto de Investigación Sanitaria Fundación Jiménez Díaz
InventorsJosé Carlos Segovia Sanz, Óscar Quintana Bustamante, Sara Fañanas Baquero
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Abstract
The present invention relates to the treatment of Pyruvate Kinase Deficiency (PKD) using the Clustered-Regularly Interspaced Short Palindromic Repeats (CRISPR) system. This technology offers the possibility to design an improved single guide RNA (sgRNA), in particular, an improved crRNA to be associated with tracrRNA, which is incorporated into a CRISPR-associated protein (Cas9) to recognize and induce DNA double-strand breaks at a specific target location. DNA double-strand breaks will be repaired by homologous recombination (HR) in the presence of a donor sequence for PKLR gene repair.
Claims21
A single guide sgRNA comprising a crRNA sequence chemically bound to a tracrRNA nucleotide sequence that interacts with a CRISPR-associated protein (Cas) polypeptide, wherein the sgRNA comprises SEQ ID NO 12.
Dependencies are inferred from the claim text.